Cincinnati Children's

Setting Families on a Positive Path to Recovery After Pediatric TBI: Road-to-Recovery

Description: This study has two main goals: 1) to refine and enhance the R2R-TBI intervention; and 2) to examine the efficacy of the R2R-TBI intervention in a randomized control trial. To achieve the second goal, we will employ a between-groups randomized treatment design with repeated measures at baseline, one-month post-randomization, and at a six-month follow-up. The two conditions will be: a) usual medical care plus access to internet resources regarding pediatric brain injury (Internet Resources Comparison group, IRC), and b) usual medical care plus the R2R-TBI intervention (Road-to-Recovery group, R2R-TBI).

Contact(s):

Shari Wade - Shari.Wade@cchmc.org

Sex: ALL

Age: 18 years and over

Phase: NA

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05622461

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Interventions: BEHAVIORAL: Road-to-recovery

Conditions: Traumatic Brain Injury, Head Injury, Head Injuries, Closed, Brain Hemorrhage, Traumatic, Brain Edema, Intracranial Edema

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A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia

Description: This clinical trial will test the safety and efficacy of combining trametinib and azacitidine in patients with juvenile myelomonocytic leukemia (JMML). Newly diagnosed lower-risk JMML patients will receive trametinib and azacitidine. High-risk JMML patients will receive trametinib, azacitidine, fludarabine, and cytarabine.

Contact(s):

cancer@cchmc.org

Sex: ALL

Age: 1 month to 21 years old

Phase: PHASE1

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05849662

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Inclusion Criteria:

Age • Patients must be ≥ 1 month and ≤21 years of age at enrollment. Diagnosis • Patients must meet the 2022 International Consensus Classification criteria for JMML. The diagnosis is made based on the following criteria:. Clinical and hematologic features (the first 2 features are present in most cases; the last 2 are required): * Peripheral blood monocyte count ≥ 1 × 109/L\* * Splenomegaly† * Blast percentage in PB and BM \< 20% * Absence of BCR::ABL1 * This monocyte threshold is not reached in approximately 7% of cases. †Splenomegaly is absent in 3% of cases at presentation. II. Genetic studies (1 finding required): * Somatic mutation in PTPN11‡ or KRAS‡ or NRAS‡ or RRAS or RRAS2‡ * Clinical diagnosis of neurofibromatosis type 1 or germline NF1 mutation and loss of heterozygosity of NF1 or somatic biallelic loss of NF1 * Germline CBL mutation and loss of heterozygosity of CBL, or somatic mutation(s) in CBL§ * Germline mutations (indicating Noonan syndrome) need to be excluded. §Occasional cases with heterozygous splice site mutations. Performance Level * Karnofsky \> 50% for patients ≥ 16 years of age * Lansky \> 50% for patients \< 16 years of age. Prior Therapy * No prior leukemia directed therapy is permitted with the exception of:
• Cytoreduction with hydroxyurea can be initiated and continued for up to 24 hours prior to the start of trametinib.
• Cytoreduction with 6-mercaptopurine (6-MP) 6-MP can be initiated and continued for up to 72 hours prior to the start of trametinib.
• Intrathecal (IT) cytarabine, IT methotrexate or triple IT therapy (cytarabine, methotrexate and hydrocortisone) within 7 days of enrollment as part of a diagnostic evaluation. No prior hematopoietic stem cell transplant is permitted. Adequate Renal Function Defined as: * Patient must have a calculated creatinine clearance or radioisotope GFR ≥ 70ml/min/1.73m2 OR a normal serum creatinine based on age/gender in the chart below: Maximum Serum Creatinine (mg/dL): * 1 month to \< 6 months old - Male: 0.4, Female 0.4 * 6 months to \<1 year old - Male 0.5, Female 0.5 * 1 to \< 2 years old - Male: 0.6, Female: 0.6 * 2 to \< 6 years old - Male:0.8, Female: 0.8 * 6 to \< 10 years old - Male: 1, Female: 1 * 10 to \< 13 years old - Male: 1.2, Female: 1.2 * 13 to \< 16 years old - Male: 1.5, Female: 1.4 * ≥ 16 years old - Male: 1.7, Female: 1.4 The threshold creatinine values in this Table were derived from the Schwartz formula for estimating GFR (Schwartz et al. J. Peds, 106:522, 1985) utilizing child length and stature data published by the CDC. Adequate Liver Function Defined as: * Direct bilirubin \< 1.5 x upper limit of normal (ULN) for age or normal, AND alanine transaminase (ALT) \< 5 x ULN for age. * The hepatic requirements are waived for patients with known or suspected liver involvement by leukemia and will not be evaluable for hepatotoxicity. This must be reviewed and approved by the study chair or vice chair. Adequate Cardiac Function Defined as: * Ejection fraction of \> or = to 50% by echocardiogram, OR * Ejection fraction of \> or = to 50% by radionuclide angiogram (MUGA). Reproductive Function * Female patients of childbearing potential must have a negative urine or serum pregnancy test confirmed within 2 weeks prior to enrollment. * Female patients with infants must agree not to breastfeed their infants while on this study. * Male and female patients of child-bearing potential must agree to use an effective method of contraception approved by the investigator during the study and for a minimum of 6 months after study treatment.

Exclusion Criteria:

* Patients cannot have a known allergy to any of the drugs used in the study. * Patients cannot have a systemic fungal, bacterial, viral, or other infection that is exhibiting ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics or other treatment. The patient needs to be off pressors and have negative blood cultures for 48 hours. * Patients cannot have a plan to administer non-protocol chemotherapy, radiation therapy, or immunotherapy during the study period. * Patients cannot have significant concurrent disease, illness, psychiatric disorder or social issue that would compromise patient safety or compliance with the protocol treatment or procedures, interfere with consent, study participation, follow up, or interpretation of study results. * Patients cannot have a clinical or molecular diagnosis of Noonan syndrome. Note: patients with either neurofibromatosis type 1 or Casitas B-lineage lymphoma (CBL) syndrome (also known as Noonan-like syndrome), are eligible to enroll. Patients with Down syndrome are excluded from the study. * Patient cannot have had prior use of hematopoietic growth factors, biologics (anti-neoplastic agent), or XRT. * Patients cannot be taking any medications for treatment of left ventricular systolic dysfunction. * Patients cannot have a history of or current evidence of retinal vein occlusion (RVO) or central serous retinopathy (CSR). * Patients cannot have had prior use of any MEK inhibitor.

Interventions: DRUG: Trametinib, DRUG: Azacitidine, DRUG: Fludarabine, DRUG: Cytarabine

Conditions: Leukemia, Juvenile Myelomonocytic, JMML, JCML, Neurofibromatosis 1, CBL Syndrome

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Caloric Restriction and Activity to Reduce Chemoresistance in B-ALL (IDEAL2)

Description: This study is for older children, adolescents, and young adults with B-cell Acute Lymphoblastic Leukemia (B-ALL). Higher amounts of body fat is associated with resistance to chemotherapy in patients with B-ALL. Chemotherapy during the first month causes large gains in body fat in most people, even those who start chemotherapy at a healthy weight. This study is being done to find out if caloric restriction achieved by a personalized nutritional menu and exercise plan during routine chemotherapy can make the patient's ALL more sensitive to chemotherapy and also reduce the amount of body fat gained during treatment. The goals of this study are to help make chemotherapy more effective in treating the patient's leukemia as demonstrated by fewer patients with leukemia minimal residual disease (MRD) while also trying to reduce the amount of body fat that chemotherapy causes the patient to gain in the first month.

Contact(s):

cancer@cchmc.org

Sex: ALL

Age: 7 years to 25 years old

Phase: PHASE2

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05082519

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Inclusion Criteria:

* Patients must be ≥ 7.0 and \<26.0 years of age. * Patients must have a diagnosis of de novo B-ALL * Patients must have a M3 marrow (\>25% blasts by morphology) or at least 1,000/µL circulating leukemia cells in PB confirmed by Flow Cytometry (or other convincing evidence of a B-ALL diagnosis not meeting above criteria following central review by the Study Hematopathologist and Study Chair or Vice-Chair). * The treatment regimen must be the first treatment attempt for B-ALL- * Must be a multi-agent induction regimen inclusive of vincristine, glucocorticoid, pegaspargase/calaspargase, and daunorubicin or doxorubicin and with a planned duration \<35 days. * Organ function must meet that required for initiation of chemotherapy * Patients at diagnosis must meet Karnofsky \> 50% for patients \> 16 years of age and Lansky \> 50% for patients ≤ 16 years of age (or be expected to recover prior to Day 8) . * If the patient is a female of childbearing potential, a negative urine or serum pregnancy test is required within two weeks prior to enrollment.

Exclusion Criteria:

* Patient will be excluded if they are underweight at time of enrollment (BMI% \<5th percentile for age for patients age 10-19 years, BMI \<18.5 in patients 20-29 years). * Patients with Down syndrome or a DNA fragility syndrome (such as Fanconi anemia, Bloom syndrome) will be excluded. * Patient receiving a SJCRH-style "Total Therapy" regimen will be excluded. * Patients receiving anti-CD20 monoclonal antibody therapy during induction therapy. * Patients will be excluded if they received treatment for a previous malignancy. * Patient will be excluded if they are pregnant. * Patient will be excluded if they have a pre-diagnosis requirement for enteral or parenteral supplementation . * Patient will be excluded due to inability to perform the intervention (e.g., specific nutritional needs, severe developmental delay, paraplegia) * Patients will be excluded if they have significant concurrent disease, illness, psychiatric disorder or social issue that would compromise patient safety or compliance with the protocol treatment or procedures, interfere with consent, study participation, follow up, or interpretation of study results

Interventions: BEHAVIORAL: IDEAL2 Intervention

Conditions: B-cell Acute Lymphoblastic Leukemia, Obesity

Keywords: obesity, leukemia, B-cell leukemia, Pediatric obesity, Pediatric ALL

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Health-E You Efficacy Trial for Male Adolescents

Description: This study will involve evaluating Health-E You/Salud ìTu™, a web-based, pre-visit mobile app designed to support adolescent male youth and his clinicians in discussing sexual and reproductive health (SRH) topics and care. It will test its efficacy among male patients in clinical settings using a stepped wedge cluster randomized controlled trial design.

Contact(s):

Emmanuel Chandler - emmanuel.chandler@cchmc.org

Sex: MALE

Age: 13 years to 21 years old

Phase: NA

Healthy Volunteers:

This study is also accepting healthy volunteers

System ID: NCT06525064

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Interventions: OTHER: Health-E You app

Conditions: Sexually Transmitted Diseases, Sexual Health, Reproductive Health

Keywords: male adolescent, condom use, mhealth

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A Study of Treatment for Medulloblastoma Using Sodium Thiosulfate to Reduce Hearing Loss

Description: This phase III trial tests two hypotheses in patients with low-risk and average-risk medulloblastoma. Medulloblastoma is a type of cancer that occurs in the back of the brain. The term, risk, refers to the chance of the cancer coming back after treatment. Subjects with low-risk medulloblastoma typically have a lower chance of the cancer coming back than subjects with average-risk medulloblastoma. Although treatment for newly diagnosed average-risk and low-risk medulloblastoma is generally effective at treating the cancer, there are still concerns about the side effects of such treatment. Side effects or unintended health conditions that arise due to treatment include learning difficulties, hearing loss or other issues in performing daily activities. Standard therapy for newly diagnosed average-risk or low-risk medulloblastoma includes surgery, radiation therapy, and chemotherapy (including cisplatin). Cisplatin may cause hearing loss as a side effect. In the average-risk medulloblastoma patients, this trial tests whether the addition of sodium thiosulfate (STS) to standard of care chemotherapy and radiation therapy reduces hearing loss. Previous studies with STS have shown that it may help reduce or prevent hearing loss caused by cisplatin. In the low-risk medulloblastoma patients, the study tests whether a less intense therapy (reduced radiation) can provide the same benefits as the more intense therapy. The less intense therapy may cause fewer side effects. Radiation therapy uses high energy x-rays to kill tumor cells and shrink tumors. Cisplatin is in a class of medications known as platinum-containing compounds. It works by killing, stopping or slowing the growth of cancer cells. The overall goals of this study are to see if giving STS along with standard treatment (radiation therapy and chemotherapy) will reduce hearing loss in medulloblastoma patients and to compare the overall outcome of patients with medulloblastoma treated with STS to patients treated without STS on a previous study in order to make sure that survival and recurrence of tumor is not worsened.

Contact(s):

- cancer@cchmc.org

Sex: ALL

Age: 4 years to 21 years old

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05382338

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Inclusion Criteria:

* PRE-ENROLLMENT: Patients must be ≥ 4 years and ≤ 21 years of age at the time of enrollment * PRE-ENROLLMENT: Patient is suspected to have newly-diagnosed medulloblastoma by institutional diagnosis * Please note: Patients with a pending result of CSF cytology tests are eligible for NCI-2014-02057 (APEC14B1-Central Nervous System \[CNS\]) and CNS/Medulloblastoma Pre Enrollment Eligibility Screening * PRE-ENROLLMENT: The patient and/or their parents or legal guardians must have signed informed consent for APEC14B1 Part A - Eligibility Screening and consent for the Molecular Characterization Initiative (MCI) * PRE-ENROLLMENT: The required specimens are projected to be submitted under APEC14B1-CNS as soon as possible, preferably within 5 days of definitive surgery * PRE-ENROLLMENT: All patients must have rapid central pathology review under APEC14B1-CNS prior to study enrollment on ACNS2031 step 1 in order to avoid discordant diagnoses and to verify diagnosis criterion for treatment on ACNS2031. * Note: Patients with a pending result of CSF cytology tests are eligible for the rapid central pathology screening review. Confirmation of CSF negativity is needed for enrollment on the ACNS2031 protocol * PRE-ENROLLMENT: All patients must have rapid central molecular screening review under APEC14B1-CNS prior to study enrollment on ACNS2031 step 1, in order to avoid discordant diagnoses and to verify diagnosis criterion for treatment on ACNS2031 * PRE-ENROLLMENT: All patients who have histopathology confirmed must have rapid central imaging screening review under APEC14B1 prior to study enrollment on ACNS2031 step 1 * Note: Patients must not have metastatic disease on cranial or spinal MRI. Patients with \> 1.5 cm\^2 residual tumor after initial surgical resection may undergo a second surgical resection prior to subsequent therapy to render them eligible for this study. The day of the second resection to remove residual tumor will be regarded as the day of definitive surgery (Day 0) and must be within a month (31 days) of the initial resection * PRE-ENROLLMENT: All patients who have histopathology confirmed must have rapid central audiology review under APEC14B1-CNS prior to study enrollment on ACNS2031 step 1 * Patients must be \>= 4 years and =\< 21 years of age at the time of enrollment * Patients must be newly diagnosed and have eligibility confirmed by rapid central pathology and molecular screening reviews performed on APEC14B1 and via the Molecular Characterization Initiative * Average-risk cohort * Clinico-pathologic criteria: * M0 disease * No diffuse anaplastic histology AND * Molecular criteria: * SHH, p53wt, GLI2 normal, MYCN normal, no chromosome 14q loss * Group 3, MYC normal, no isochromosome 17q * Group 4, no chromosome 11 loss * Low-risk features cohort * Clinico-pathologic criteria: * M0 disease * No diffuse anaplastic histology AND * Molecular criteria: * Group 4, chromosome 11 loss * Patients must have negative lumbar CSF cytology * Note: CSF cytology for staging should be performed no sooner than 14 days post operatively to avoid false positive CSF. Ideally, CSF should be obtained between day 14 and day 21 to allow for final staging status before enrollment onto the study. Patients with positive CSF cytology obtained 0 to 14 days after surgery should have cytology repeated to determine eligibility and final CSF status. Patients with negative CSF cytology from lumbar puncture obtained 0 to 14 days after surgery do not need cytology repeated. Patients with negative CSF cytology from lumbar puncture obtained prior to surgery do not need cytology repeated post-operatively * Patients must have eligibility confirmed by Rapid Central Imaging Review performed on APEC14B1. Patients must have =\< 1.5 cm\^2 cross-sectional area of residual tumor. Whole brain MRI with and without gadolinium and spine MRI with gadolinium must be performed * Patients must weigh \> 10 kg * Patients must be enrolled, and protocol therapy must be projected to begin, no later than 31 days after definitive diagnostic surgery (day 0) * Peripheral absolute neutrophil count (ANC) \>= 1000/uL (within 7 days prior to enrollment) * Platelet count \>= 100,000/uL (transfusion independent) (within 7 days prior to enrollment) * Hemoglobin \>= 8.0 g/dL (may receive red blood cell count \[RBC\] transfusions) (within 7 days prior to enrollment) * A serum creatinine (within 7 days prior to enrollment) based on age/sex as follows: * 4 to \< 6 years (age); 0.8 mg/dL (male) 0.8 mg/dL (female) * 6 to \< 10 years (age); 1 mg/dL (male) 1 mg/dL (female) * 10 to \< 13 years (age); 1.2 mg/dL (male) 1.2 mg/dL (female) * 13 to \< 16 years (age); 1.5 mg/dL (male) 1.4 mg/dL (female) * \>= 16 years (age); 1.7 mg/dL (male) 1.4 mg/dL (female) OR a 24 hour urine Creatinine clearance \>= 70 mL/min/1.73 m\^2 (within 7 days prior to enrollment) OR a glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 (within 7 days prior to enrollment). GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard) * Note: Estimated GFR (eGFR) from serum creatinine, cystatin C or other estimates are not acceptable for determining eligibility * Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment) * Serum glutamic pyruvic transaminase (SGPT) (alanine aminotransferase \[ALT\]) =\< 135 U/L (within 7 days prior to enrollment) * Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U/L * Central nervous system function defined as: * Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled * Patients must not be in status epilepticus, a coma or assisted ventilation at the time of study enrollment * Auditory function defined as: * Patients must have normal hearing (defined as International Society of Pediatric Oncology \[SIOP\] grade 0) in at least one ear confirmed by rapid central audiology review performed on APEC14B1 prior to enrollment * All patients and/or their parents or legal guardians must sign a written informed consent * All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met

Exclusion Criteria:

* Patients with metastatic disease by either MRI evaluation or lumbar CSF cytology are not eligible. Patients who are unable to undergo a lumbar puncture for assessment of CSF cytology are ineligible * Patients must not have received any prior radiation therapy or chemotherapy (tumor-directed therapy) other than surgical intervention and/or corticosteroids * Patients must not have any known hypersensitivity to STS, sulfates/sulfites, or other thiol agents (e.g., amifostine, n-acetylcysteine, MESNA, and captopril) * Pregnancy and Breastfeeding: * Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential * Lactating females who plan to breastfeed their infants * Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation

Interventions: PROCEDURE: Audiometric Test, PROCEDURE: Auditory Brainstem Response, PROCEDURE: Biospecimen Collection, DRUG: Cisplatin, DRUG: Cyclophosphamide, DRUG: Lomustine, PROCEDURE: Magnetic Resonance Imaging, OTHER: Quality-of-Life Assessment, RADIATION: Radiation Therapy, DRUG: Sodium Thiosulfate, OTHER: Survey Administration, DRUG: Vincristine

Conditions: Childhood Medulloblastoma

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BE WELL With Migraine: Brain Education and WELLness With Migraine

Description: The goal of this randomized clinical trial is to evaluate two different non-drug, virtual treatment options designed to improve the lives of patients with migraine. Both interventions involve 8 weekly sessions and an online platform with additional content and learning. Participants can stay on all their medications during this study. Information from this study may help determine how to better treat migraine.

Contact(s):

LeighAnn Chamberlin - LeighAnn.Chamberlin@cchmc.org

Sex: ALL

Age: 18 years and over

Phase: NA

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05987592

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Interventions: BEHAVIORAL: Brain Education and WELLness with Migraine Group A, BEHAVIORAL: Brain Education and WELLness with Migraine Group B

Conditions: Migraine

Keywords: Migraine, Migraine without aura, Migraine with aura, behavioral, mind-body, integrative, virtual, online

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A Study to Compare Standard Therapy to Treat Hodgkin Lymphoma to the Use of Two Drugs, Brentuximab Vedotin and Nivolumab

Description: This phase III trial compares the effect of adding immunotherapy (brentuximab vedotin and nivolumab) to standard treatment (chemotherapy with or without radiation) to the standard treatment alone in improving survival in patients with stage I and II classical Hodgkin lymphoma. Brentuximab vedotin is in a class of medications called antibody-drug conjugates. It is made of a monoclonal antibody called brentuximab that is linked to a cytotoxic agent called vedotin. Brentuximab attaches to CD30 positive lymphoma cells in a targeted way and delivers vedotin to kill them. A monoclonal antibody is a type of protein that can bind to certain targets in the body, such as molecules that cause the body to make an immune response (antigens). Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Chemotherapy drugs such as doxorubicin hydrochloride, bleomycin sulfate, vinblastine sulfate, dacarbazine, and procarbazine hydrochloride work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell's deoxyribonucleic acid (DNA) and may kill cancer cells. It may also lower the body's immune response. Etoposide is in a class of medications known as podophyllotoxin derivatives. It blocks a certain enzyme needed for cell division and DNA repair and may kill cancer cells. Vincristine is in a class of medications called vinca alkaloids. It works by stopping cancer cells from growing and dividing and may kill them. Prednisone is in a class of medications called corticosteroids. It is used to reduce inflammation and lower the body's immune response to help lessen the side effects of chemotherapy drugs. Radiation therapy uses high energy x-rays to kill tumor cells and shrink tumors. Adding immunotherapy to the standard treatment of chemotherapy with or without radiation may increase survival and/or fewer short-term or long-term side effects in patients with classical Hodgkin lymphoma compared to the standard treatment alone.

Contact(s):

- cancer@cchmc.org

Sex: ALL

Age: 5 years to 60 years old

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05675410

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Inclusion Criteria:

* Patients must be 5 to 60 years of age at the time of enrollment * Patients with newly diagnosed untreated histologically confirmed classic Hodgkin lymphoma (cHL) (nodular sclerosis, mixed cellularity, lymphocyte-rich, or lymphocyte-depleted, or not otherwise specified \[NOS\]) with stage I or II disease * Patients must have bidimensionally measurable disease (at least one lesion with longest diameter \>= 1.5 cm) * Patients must have a whole body or limited whole body PET scan performed within 42 days prior to enrollment. PET-CT is strongly preferred. PET-MRI allowed if intravenous contrast enhanced CT is also obtained * Pediatric patients (age 5-17 years) with known or suspected mediastinal disease must have an upright posteroanterior (PA) chest X-ray (CXR) for assessment of bulky mediastinal disease. * Note: Pediatric patients who have received both a CT chest and upright PA CXR may meet the definition of bulk through either modality. * Patients \>= 18 years must have a performance status corresponding to Zubrod scores of 0, 1 or 2 * Patients =\< 17 years of age must have a Lansky performance score of \>= 50 * Pediatric patients (age 5-17 years): A serum creatinine based on age/sex as follows (within 28 days prior to enrollment): * 2 to \< 6 years (age): 0.8 mg/dL (male), 0.8 mg/dL (female) * 6 to \< 10 years (age): 1 mg/dL (male), 1 mg/dL (female) * 10 to \< 13 years (age): 1.2 mg/dL (male), 1.2 mg/dL (female) * 13 to \< 16 years (age): 1.5 mg/dL (male), 1.4 mg/dL (female) * \>= 16 years (age): 1.7 mg/dL (male), 1.4 mg/dL (female) OR a 24 hour urine creatinine clearance \>= 50 mL/min/1.73 m\^2 (within 28 days prior to enrollment) OR a glomerular filtration rate (GFR) \>= 50 mL/min/1.73 m\^2 (within 28 days prior to enrollment). GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard) * Note: Estimated GFR (eGFR) from serum or plasma creatinine, cystatin C or other estimates are not acceptable for determining eligibility * For adult patients (age 18 years or older) (within 28 days prior to enrollment): Creatinine clearance \>= 30 mL/min, as estimated by the Cockcroft and Gault formula or a 24-hour urine collection. The creatinine value used in the calculation must have been obtained within 28 days prior to registration. Estimated creatinine clearance is based on actual body weight * Total bilirubin =\< 2 x upper limit of normal (ULN) (within 28 days prior to enrollment) * Unless due to Gilbert's disease, lymphomatous involvement of liver or vanishing bile duct syndrome * Aspartate aminotransferase (AST) =\< 3 x ULN (within 28 days prior to enrollment) * Unless due to Gilbert's disease, lymphomatous involvement of liver or vanishing bile duct syndrome * Alanine aminotransferase (ALT) =\< 3 x ULN (within 28 days prior to enrollment) * Unless due to Gilbert's disease, lymphomatous involvement of liver or vanishing bile duct syndrome * Shortening fraction of \>= 27% by echocardiogram (ECHO), multigated acquisition scan (MUGA), or functional cardiac imaging scan (within 28 days prior to enrollment) or ejection fraction of \>= 50% by radionuclide angiogram, ECHO, MUGA, or cardiac imaging scan (within 28 days prior to enrollment) * Diffusion capacity of the lung for carbon monoxide (DLCO) \>= 50% of predicted value as corrected for hemoglobin by pulmonary function test (PFT) (within 28 days prior to enrollment). If unable to obtain PFTs, the criterion is: a pulse oximetry reading of \> 92% on room air * Known human immunodeficiency virus (HIV)-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial * For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated. Patients with a history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load

Exclusion Criteria:

* Patients with nodular lymphocyte predominant Hodgkin lymphoma * Patients with a history of active interstitial pneumonitis or interstitial lung disease * Patients with a diagnosis of inherited or acquired immunodeficiency that is poorly controlled or requiring active medications, such as primary immunodeficiency syndromes or organ transplant recipients * Patients with any known uncontrolled intercurrent illness that would jeopardize the patient's safety such as infection, autoimmune conditions, cardiac arrhythmias, angina pectoris, and gastrointestinal disorders affecting swallowing and/or absorption of pills * Patients with a condition requiring systemic treatment with either corticosteroids (defined as equivalent to \> 10 mg daily predniSONE for patients \>= 18 years or \> 0.5 mg/kg \[up to 10 mg/day\] for patients \< 18 years) or other immunosuppressive medications within 14 days prior to enrollment * Note: Replacement therapy such as thyroxine, insulin, or physiologic corticosteroid for adrenal or pituitary insufficiency is not considered a form of systemic treatment. Inhaled or topical steroids, and adrenal replacement doses (=\< 10 mg daily for patients \>= 18 years or =\< 0.5 mg/kg \[up to 10 mg/day\] predniSONE equivalents) are permitted in the absence of active autoimmune disease * Note: Steroid use for the control of Hodgkin lymphoma symptoms is allowable, but must be discontinued by cycle 1, day 1 * Short term use of corticosteroids for premedication or treatment of an allergy or hypersensitivity is considered an acceptable use of corticosteroids. * Patients with peripheral neuropathy \> grade 1 at the time of enrollment or patients with known Charcot-Marie-Tooth syndrome * Patients with a prior or concurrent malignancy whose natural history or treatment has the potential to interfere with the safety or efficacy assessment of the investigational regimen * Administration of prior chemotherapy, radiation, or antibody-based treatment for cHL * Prior solid organ transplant * Prior allogeneic stem cell transplantation * Live vaccine within 30 days prior to planned day 1 of protocol therapy (e.g., measles, mumps, rubella, varicella, yellow fever, rabies, bacillus Calmette Guerin \[BCG\], oral polio vaccine, and oral typhoid). Administration of messenger ribonucleic acid (mRNA) vaccines are permitted * Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test within 28 days prior to enrollment is required for female patients of childbearing potential * Lactating females who plan to breastfeed their infants starting with the first dose of study therapy and for at least 6 months after the last treatment * Sexually active patients of reproductive potential who have not agreed to use a highly effective contraceptive method for the duration of their study drug therapy. Following therapy, patients will be advised to use contraception as per institutional practice or as listed below for investigational agents, whichever is longer * Men and women of childbearing potential (WOCBP) must use effective contraception during the study and for 2 months for WOCBP and 4 months for men, after last dose of brentuximab vedotin * WOCBP must continue contraception for a period of at least 5 months after the last dose of nivolumab * All patients and/or their parents or legal guardians must sign a written informed consent * All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met

Interventions: PROCEDURE: Biospecimen Collection, BIOLOGICAL: Bleomycin Sulfate, DRUG: Brentuximab Vedotin, PROCEDURE: Computed Tomography, DRUG: Cyclophosphamide, DRUG: Dacarbazine, DRUG: Doxorubicin Hydrochloride, DRUG: Etoposide, DRUG: Etoposide Phosphate, OTHER: Fludeoxyglucose F-18, RADIATION: Involved-site Radiation Therapy, PROCEDURE: Magnetic Resonance Imaging, BIOLOGICAL: Nivolumab, PROCEDURE: Positron Emission Tomography, DRUG: Prednisolone, DRUG: Prednisone, DRUG: Procarbazine Hydrochloride, OTHER: Questionnaire Administration, DRUG: Vinblastine Sulfate, DRUG: Vincristine Sulfate

Conditions: Lugano Classification Limited Stage Hodgkin Lymphoma AJCC v8

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A Study Comparing KB803 and Matched Placebo in Patients With Dystrophic Epidermolysis Bullosa (IOLITE)

Description: KB803-EYE-01 is a Phase 3 double-blind, randomized, placebo-controlled, crossover study to evaluate the safety and efficacy of KB803 versus matched placebo in pediatric and adult subjects with recurrent corneal abrasions due to dystrophic epidermolysis bullosa (DEB).

Contact(s):

Bret Augsburger - Bret.Augsburger@cchmc.org

Sex: ALL

Age: 6 month(s) and over

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT07016750

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Interventions: BIOLOGICAL: KB803, DRUG: Placebo

Conditions: Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa

Keywords: Dystrophic Epidermolysis Bullosa, DEB, Corneal Abrasions

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Sinus Disease in Young Children With Cystic Fibrosis

Description: This is a prospective, observational study examining the impact of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators on chronic rhinosinusitis (CRS) and olfactory dysfunction (OD) in young children with cystic fibrosis (YCwCF). This study involves two groups: children 2-8 years old, inclusive at initial visit, receiving highly effective modulator therapy (HEMT), and a control group of children 2-8 years old, inclusive at initial visit, not receiving HEMT. Outcomes will include sinus magnetic resonance imaging (MRI) scans, olfactory tests, and quality of life surveys obtained over a two-year period.

Contact(s):

Megan Schmitt - megan.schmitt@cchmc.org

Sex: ALL

Age: 2 years to 8 years old

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06191640

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Interventions: DRUG: Ivacaftor or elexacaftor/tezacaftor/ivacaftor

Conditions: Cystic Fibrosis in Children, Cystic Fibrosis, Chronic Rhinosinusitis (Diagnosis), Olfactory Disorder, Olfactory Impairment

Keywords: Cystic Fibrosis, Chronic Rhinosinusitis, Olfactory Dysfunction

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Inotuzumab Ozogamicin and Post-Induction Chemotherapy in Treating Patients With High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and B-LLy

Description: This phase III trial studies whether inotuzumab ozogamicin added to post-induction chemotherapy and immunotherapy (chemo-immunotherapy) for patients with High-Risk B-cell Acute Lymphoblastic Leukemia (B-ALL) improves outcomes. Inotuzumab ozogamicin is a monoclonal antibody, which is a type of protein that can bind to certain targets on the surface of cells. Inotuzumab ozogamicin is a monoclonal antibody that is linked to a type of chemotherapy called calicheamicin. Inotuzumab attaches to cancer cells by binding to the CD22 protein on the surface of the cancer cell and delivering calicheamicin inside the cells to kill them. Other drugs used in the chemotherapy regimen, such as cyclophosphamide, cytarabine, dexamethasone, doxorubicin, daunorubicin, methotrexate, leucovorin, mercaptopurine, prednisone, thioguanine, vincristine, and pegaspargase or calaspargase pegol work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Blinatumomab is a specialized type of monoclonal antibody known as a bispecific T-cell engager (BiTE). It works by simultaneously binding to CD19 on cancer cells and CD3 on normal immune cells, bringing them together to destroy leukemia cells. Blinatumomab is a standard part of chemo-immunotherapy treatment for B-ALL. This trial also studies the outcomes of patients with mixed phenotype acute leukemia (MPAL), and B-lymphoblastic lymphoma (B-LLy) when treated with ALL therapy without inotuzumab ozogamicin or blinatumomab. The overall goal of this study is to understand if adding inotuzumab ozogamicin to standard of care chemo-immunotherapy maintains or improves outcomes in High Risk B-cell Acute Lymphoblastic Leukemia (HR B-ALL). The first part of the study includes the first phase of therapy: Induction. This part will collect information on the leukemia, as well as the effects of the initial treatment, to classify patients into post-induction treatment groups. On the second part of this study, patients with HR B-ALL will receive the remainder of the chemotherapy cycles (consolidation, blinatumomab block 1, interim maintenance 1, blinatumomab block 2, delayed intensification, interim maintenance 2, maintenance), with some patients randomized to receive inotuzumab. The patients that receive inotuzumab will not receive part of consolidation or part of delayed intensification. Other aims of this study include evaluating 1) side effects of treatment using patient-reported outcomes and health-related quality of life, 2) the best ways to help patients adhere to oral chemotherapy regimens, 3) the relationship between levels of inotuzumab ozogamicin in the blood and side effects, 4) the impact of chemo-immunotherapy on the immune system and risk of infection, and 5) the impact of social determinants of health on outcomes. Finally, this study will be the first to track the outcomes of subjects with disseminated B-cell Lymphoblastic Leukemia (B-LLy) or Mixed Phenotype Acute Leukemia (MPAL) when treated with B-ALL chemotherapy.

Contact(s):

- cancer@cchmc.org

Sex: ALL

Age: 365 days to 25 years old

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT03959085

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Inclusion Criteria:

* B-ALL and MPAL patients must be enrolled on APEC14B1 and consented to eligibility studies (Part A) prior to treatment and enrollment on AALL1732. Note that central confirmation of MPAL diagnosis must occur within 22 days of enrollment for suspected MPAL patients. If not performed within this time frame, patients will be taken off protocol. * APEC14B1 is not a requirement for B-LLy patients but for institutional compliance every patient should be offered participation in APEC14B1. B-LLy patients may directly enroll on AALL1732. * Patients must be \> 365 days and \< 25 years of age * Initial white blood cell count (WBC) criteria for patients with B-ALL (within 7 days prior to the start of protocol-directed systemic therapy): * Age 1-9.99 years: WBC \>= 50,000/uL * Age 10-24.99 years: Any WBC * Age 1-9.99 years: WBC \< 50,000/uL with one or more of the following: * Testicular leukemia * CNS leukemia (CNS3) * Steroid pretreatment. * Initial white blood cell count (WBC) criteria for patients with MPAL (within 7 days prior to the start of protocol-directed systemic therapy): * Age 1-24.99 years: any WBC NOTE: Patients enrolled as suspected MPAL but found on central confirmatory testing to have B-ALL must meet the B-ALL criteria above (age, WBC, extramedullary disease, steroid pretreatment) to switch to the B-ALL stratum before the end of induction. * Patient has newly diagnosed B-ALL or MPAL (by World Health Organization \[WHO\] 2016 criteria) with \>= 25% blasts on a bone marrow (BM) aspirate; * OR If a BM aspirate is not obtained or is not diagnostic of acute leukemia, the diagnosis can be established by a pathologic diagnosis of acute leukemia on a BM biopsy; * OR A complete blood count (CBC) documenting the presence of at least 1,000/uL circulating leukemic cells if a bone marrow aspirate or biopsy cannot be performed. * Patient has newly diagnosed B-LLy Murphy stages III or IV. * Patient has newly diagnosed B-LLy Murphy stages I or II with steroid pretreatment. * Note: For B-LLy patients with tissue available for flow cytometry, the criterion for diagnosis should be analogous to B-ALL. For tissue processed by other means (i.e., paraffin blocks), the methodology and criteria for immunophenotypic analysis to establish the diagnosis of B-LLy defined by the submitting institution will be accepted. * Central nervous system (CNS) status must be determined prior to enrollment based on a sample obtained prior to administration of any systemic or intrathecal chemotherapy, except for steroid pretreatment and cytoreduction. Note that once cerebrospinal fluid (CSF) has been collected, protocol therapy can be initiated while final determination of CNS status is pending. It is recommended that intrathecal cytarabine be administered at the time of the diagnostic lumbar puncture. This is usually done at the time of the diagnostic bone marrow or venous line placement to avoid a second lumbar puncture. This is allowed prior to enrollment. Systemic chemotherapy must begin within 72 hours of this intrathecal therapy. * Direct bilirubin \< 2.0 mg/dL (34 micromoles/L) * Alanine aminotransferase (ALT) ≤ 10x upper limit of normal (ULN). For the purposes of this study, the ULN for ALT is defined as 45 U/L * Exceptions to this include patients with known Gilbert's Syndrome, or those with hepatic involvement from leukemic or lymphomatous infiltration * All patients and/or their parents or legal guardians must sign a written informed consent. * All institutional, Food and Drug Administration (FDA), and NCI requirements for human studies must be met.

Exclusion Criteria:

* Patients with Down syndrome are not eligible * With the exception of steroid pretreatment and steroid cytoreduction or the administration of intrathecal cytarabine, patients must not have received any prior cytotoxic chemotherapy for the current diagnosis of B-ALL, MPAL, or B-LLy or for any cancer diagnosed prior to initiation of protocol therapy on AALL1732. * Patients who have received \> 72 hours of hydroxyurea within one week prior to start of systemic protocol therapy. * Patients with B-ALL or MPAL who do not have sufficient diagnostic bone marrow submitted for APEC14B1 testing and who do not have a peripheral blood sample submitted containing \> 1,000/uL circulating leukemia cells. * Patients with acute undifferentiated leukemia (AUL) are not eligible. * For Murphy stage III/IV B-LLy patients, or stage I/II patients with steroid pretreatment, the following additional exclusion criteria apply: * T-lymphoblastic lymphoma. * Morphologically unclassifiable lymphoma. * Absence of both B-cell and T-cell phenotype markers in a case submitted as lymphoblastic lymphoma. * Patients with known Charcot-Marie-Tooth disease. * Patients with known MYC translocation associated with mature (Burkitt) B-cell ALL, regardless of blast immunophenotype. * Patients requiring radiation at diagnosis. * Female patients who are pregnant, since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential. * Lactating women who plan to breastfeed their infants while on study and for 2 months after the last dose of inotuzumab ozogamicin. * Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of study participation. For those patients randomized to inotuzumab ozogamicin, there is a minimum of 8 months after the last dose of inotuzumab ozogamicin for females and 5 months after the last dose of inotuzumab ozogamicin for males.

Interventions: PROCEDURE: Biospecimen Collection, BIOLOGICAL: Blinatumomab, PROCEDURE: Bone Marrow Aspiration, PROCEDURE: Bone Marrow Biopsy, PROCEDURE: Bone Scan, DRUG: Calaspargase Pegol, PROCEDURE: Computed Tomography, DRUG: Cyclophosphamide, DRUG: Cytarabine, DRUG: Daunorubicin Hydrochloride, DRUG: Dexamethasone, DRUG: Doxorubicin Hydrochloride, BIOLOGICAL: Inotuzumab Ozogamicin, DRUG: Leucovorin Calcium, PROCEDURE: Magnetic Resonance Imaging, DRUG: Mercaptopurine, DRUG: Methotrexate, DRUG: Pegaspargase, PROCEDURE: Positron Emission Tomography, DRUG: Prednisolone, DRUG: Prednisone, OTHER: Questionnaire Administration, RADIATION: Radiation Therapy, RADIATION: Radiation Therapy, DRUG: Thioguanine, DRUG: Vincristine Sulfate

Conditions: B Acute Lymphoblastic Leukemia, B Lymphoblastic Lymphoma, Central Nervous System Leukemia, Mixed Phenotype Acute Leukemia, Testicular Leukemia

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A National Registry For Pulmonary Alveolar Proteinosis

Description: The major goal of Part A of this study is to establish a National PAP Registry to help make reliable new research tests available to doctors to improve the diagnosis of PAP, increase awareness and knowledge of PAP, and give patients a 'seat at the table' in planning and conducting PAP research including the clinical testing of several new potential therapies. The major goal of Part B of this study is to define the natural history of autoimmune PAP (aPAP), develop a disease severity score that reflects how aPAP patients feel and function, and to develop and test novel tools to measure the severity of aPAP lung disease. Funding Source - FDA OOPD

Contact(s):

Brenna Carey - Brenna.Carey@cchmc.org

Sex: ALL

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT02461615

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Conditions: Pulmonary Alveolar Proteinosis

Keywords: Pulmonary Surfactant, Rare Lung Disease, Registry

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Precision Alemtuzumab Dosing for Allogeneic Hematopoietic Cell Transplantation

Description: Alemtuzumab is an antibody that reduces the strength of the immune system that is given in preparation for allogeneic hematopoietic cell transplant (HCT). In this research study the investigators want to find out if they can adjust the dose of alemtuzumab used as part of allogeneic HCT to target the level of Day 0 (the planned day of graft infusion) to an optimal therapeutic window of 0.15-0.9 ug/mL.

Contact(s):

Caitlin Cottrell - caitlin.cottrell@cchmc.org

Sex: ALL

Phase: PHASE2

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05501756

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Interventions: DRUG: Alemtuzumab

Conditions: Allogeneic Hematopoietic Cell Transplantation

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Quercetin in Patients With XIAP (X-linked Inhibitor of Apoptosis) Deficiency

Description: The purpose of the study is to evaluate safety and tolerability of oral quercetin in reducing inflammation in male patients with XIAP deficiency. Quercetin is a naturally occurring antioxidant that has many properties including the ability to decrease inflammation in other diseases.

Contact(s):

Kelly McIntosh - Kelly.mcintosh@cchmc.org

Sex: MALE

Age: 2 years and over

Phase: PHASE1

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT07433621

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Interventions: DRUG: Quercetin

Conditions: XIAP Deficiency

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A Study to Evaluate the Safety and Tolerability, Pharmacokinetics, and Antiviral Activity of Maribavir for the Treatment of Cytomegalovirus (CMV) Infection in Children and Adolescents Who Have Received a Hematopoietic Stem Cell Transplant (HSCT) or a Solid Organ Transplant (SOT)

Description: The main aim of this study is to find out the safety, tolerability and pharmacokinetics (PK) of maribavir for the treatment of CMV infection in children and teenagers after HSCT or SOT and to identify the optimal dose of maribavir using a 200 milligrams (mg) tablet formulation or powder for oral suspension. The participants will be treated with maribavir for 8 weeks. Participants need to visit their doctor during 12-week follow-up period.

Contact(s):

Kerrigan Perkins - kerrigan.perkins@cchmc.org

Sex: ALL

Age: Up to 17 years old

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05319353

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Inclusion Criteria:

* Parent/both parents or legally authorized representative (LAR) must provide signature of informed consent and there must be documentation of assent by the participant, as age appropriate, before completing any study-related procedures. * Be a male or female child or adolescent \< 18 years of age at the time of consent. For participants in Cohort 3 only (0 to \<6 years) must have a gestational age of at least 39 weeks and a minimum weight of 5 kg. * Be a recipient of an SOT or an HSCT that is functioning at the time of screening. * Have a documented CMV infection which may be a first episode of post-transplant CMV viremia (primary or reactivation) or refractory to other anti-CMV treatments, with a CMV DNA screening value of \>= 1365 International Units per milliliter (IU/mL) in whole blood or \>= 455 IU/mL in plasma in 2 consecutive assessments separated by at least 1 day, as determined by local laboratory quantitative polymerase chain reaction (qPCR) or comparable quantitative nucleic acid amplification test (qNAAT) results. Quantitative assays must be standardized to the World Health Organization (WHO) CMV International Standard. Both samples must be taken within 14 days of first dose of study drug, with the second sample obtained within 5 days prior to first dose of study drug. The same laboratory and same sample type (whole blood or plasma) must be used for both assessments. If documented and verified values are available in medical history that fulfill this criterion entirely, they may be used instead. * Have all the following results as part of screening laboratory assessments: * Absolute neutrophil count \>= 500 per cubic millimeter (/mm\^3) (0.5 × 10\^9 per liter \[/L\]) * Platelet count \>= 15,000/mm\^3 (15 × 10\^9/L) * Hemoglobin \>= 8 grams per deciliter (g/dL) (\>=80 grams per liter \[g/L\]). * Have an estimated glomerular filtration rate (creatinine-based Bedside Schwartz equation) \>= 30 milliliters per minute (mL/min) /1.73 meter square (m\^2). * Be a female of nonchildbearing potential. If a female of childbearing potential, have a negative serum human chorionic gonadotropin (hCG) or beta-human chorionic gonadotropin (β-hCG) pregnancy test at screening. Males, or nonpregnant, nonlactating females who are sexually active must agree to comply with the applicable contraceptive requirements of this protocol during the study treatment administration period and for 90 days after the last dose of study treatment. * Have life expectancy of \>= 8 weeks. * Be willing and have an understanding and ability to fully comply with the study procedures and restrictions defined in the protocol. For younger children, the parent/both parents or LAR must meet this criterion. * Participants must have a confirmed negative human immunodeficiency virus (HIV) test result within 3 months of first dose of study drug or, if unavailable, be tested by a local laboratory during the screening period.

Exclusion Criteria:

* Have CMV tissue invasive disease involving the central nervous system (CNS) or retina as assessed by the investigator at the time of screening. * Have uncontrolled other type of infection as assessed by the investigator on the date of enrollment. * Have a history of clinically relevant alcohol or drug abuse that may interfere with treatment compliance or assessments with the protocol as determined by the investigator. * Be receiving valganciclovir, ganciclovir, cidofovir, foscarnet, leflunomide, letermovir, or artesunate when study treatment is initiated, or anticipated to require one of these agents during the 8-week treatment period. * Have a known hypersensitivity to maribavir or to any excipients. * Have severe vomiting, diarrhea, or other severe gastrointestinal (GI) illness within 24 hours prior to the first dose of study treatment or a GI absorption abnormality that would preclude administration of oral medication. * Require mechanical ventilation or vasopressors for hemodynamic support at baseline (Visit 2/Day 1/Week 0). * Be pregnant (or expecting to conceive) or nursing. * Have previously completed, discontinued, or have been withdrawn from this study. * Have received any investigational agent or device within 30 days before initiation of study treatment (includes CMV specific T-cells) or plan to receive an investigational agent or device during the study. Previously approved agents under investigation for additional indications are not exclusionary. * Have previously received maribavir or CMV vaccine at any time. * Have any clinically significant medical or surgical condition that, in the investigator's opinion, could interfere with interpretation of study results, contraindicate the administration of the study treatment, or compromise the safety or well-being of the participant. * Have severe liver disease (Child-Pugh score of \>= 10). * Have serum aspartate aminotransferase greater than (\>) 5 times upper limit of normal (ULN) at screening, or serum alanine aminotransferase \> 5 times ULN at screening, or total bilirubin \>= 3.0 times ULN at screening (except for documented Gilbert's syndrome), as analyzed by local laboratory. * Have positive results for HIV. * Have active malignancy with the exception of nonmelanoma skin cancer, as determined by the investigator. Participants who experience relapse or progression of their underlying malignancy (for which HSCT or SOT was performed), as determined by the investigator, are not to be enrolled. * Be undergoing treatment for acute or chronic hepatitis B or hepatitis C. * Requiring ongoing treatment with or an anticipated need for treatment with a strong cytochrome P450 3A (CYP3A) inducer. * Have a low body weight where total blood volume (TBV) required during study participation will exceed 1 percent (%) TBV per study visit or 3% TBV over a 4-week period.

Interventions: DRUG: Maribavir

Conditions: Cytomegalovirus (CMV)

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Comparing Stainless Steel Crowns With Prefabricated Resin Crowns in Primary Molar Teeth

Description: The main reason for this research study is to learn more about a new flexible white dental crown (BioFLX) by comparing it to an existing flexible metal crown (Stainless Steel Crown). It is of interest to see if this new white crown is clinically equivalent to the existing silver crown that is mainly used in pediatric dentistry. A potential participant for this study would have cavities that require a crown, a type of filling that covers the entire tooth, and recommended dental work be done under general anesthesia.

Contact(s):

Patrick T Ruck - patrick.ruck@cchmc.org

Sex: ALL

Age: 2 years to 5 years old

Phase: NA

Healthy Volunteers:

This study is also accepting healthy volunteers

System ID: NCT06713330

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Inclusion Criteria:

* CCHMC pediatric dental patients between the ages of 2 years to 5 years and 11 months, at the time of recruitment, who present to any CCHMC dental clinic location and then are found to need full mouth dental rehabilitation. * Patients who speak the most common languages at CCHMC will be able to be recruited for the study. o English, Spanish, Arabic, Uzbek, Nepali, Chinese Mandarin, Russian, French. * These patients must qualify for treatment at the CCHMC dental in-office general anesthesia (IOGA) area or the Procedure Center (PC). IOGA and the PC will be selected as a venue of treatment to control behavioral factors. This is not specific to the study and would occur due to their treatment needs. * Participants will have at least one pair of contralateral primary molars with the need for a full coverage restoration in the same arch. * For example, tooth A \& J, B \& I, S \& L, or T \& K * For each participant, a minimum of one SSC or one PRC will be randomly assigned via a split mouth design to be placed as part of the study. * Need for Full coverage and high caries risk will be defined by AAPD Best Practice Guidelines 2,16 o Teeth With
• Extensive caries
• Cervical decalcification
• Developmental defects (e.g., hypoplasia, hypocalcification)
• When failure of other available restorative materials is likely (e.g., interproximal caries extending beyond line angles, patients with bruxism)
• Following pulpotomy or pulpectomy
• For definitive restorative treatment for high caries-risk children as defined by the AAPD
• For patients who exhibit high caries risk and whose treatment is performed under sedation or general anesthesia. This would be normal and not specific to the study. * Participants who consent to the study, and who can be available for follow-up recall appointments. * All participants will be ASA I or ASA II as defined by the American Society of Anesthesiologists.15

Exclusion Criteria:

* Participants who do not meet inclusion criteria will be excluded. * Participants whose teeth do not meet the inclusion criteria. * Participants who do not wish to participate in the study. * Patients who do not wish to or cannot reliably return for follow-up visits. * Red dye allergy as patient will not be able to be plaque disclosed during follow-up visits. * Participants who do not speak English, Spanish, Arabic, Uzbek, Nepali, Chinese Mandarin, Russian, French.

Interventions: DEVICE: BioFLX crown, DEVICE: 3M Stainless Steel Crown

Conditions: Dental Caries

Keywords: Pediatric dentistry

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The NODE-202 Study (Study of Etripamil Nasal Spray in Pediatric Patients)

Description: NODE-202 is a Phase 2, multicenter, multinational, single dose, open-label, 2-part, sequential design study in pediatric patients with an established diagnosis of paroxysmal supraventricular tachycardia (PSVT) presenting with a symptomatic episode of PSVT. In Part 1, at least 30 patients aged 12 to \<18 years will be enrolled and treated with etripamil nasal spray (NS). Efficacy, safety, tolerability and PK (for at least 12 patients) will be assessed after administration of 70 mg etripamil NS (Part 1A). At least 18 subsequent patients will be enrolled and treated with the etripamil NS with the dose determined by the Pharmacokinetic (PK) analysis and will undergo efficacy and safety/tolerability assessments (Part 1B). In Part 2, at least 30 patients aged 6 to \<12 years will be enrolled and treated with etripamil NS at a dose selected based on appropriate body size-based modeling, as well as efficacy, safety/tolerability, and PK data collected in Part 1. Efficacy, safety, tolerability and PK (for at least 12 patients) will be assessed after administration of etripamil NS (Part 2A). At least 18 subsequent patients will be enrolled and treated with the etripamil NS with the dose determined by the PK analysis and will undergo efficacy and safety/tolerability assessments (Part 2B). The study will include the following visits: A Screening Visit, A Treatment Visit, , and A Follow-Up/End of Study Visit.

Contact(s):

Allison Ackermann - allison.ackermann@cchmc.org

Sex: ALL

Age: 6 years to 17 years old

Phase: PHASE2

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05763953

Show full eligibility criteria

Inclusion Criteria:

Patients will be eligible for study participation if they meet all of the following criteria at screening:
• Male or female patients
• Part 1: patients 12 to \<18 years of age
• Part 2: patients 6 to \<12 years of age
• Body mass index (BMI) between the 5th and the 85th percentiles interpreted relative sex and age
• History of PSVT documented by ECG or other monitoring modality (e.g., Holter monitor, event recorder) showing SVT involving the Atrioventricular (AV) node (i.e., Atrioventricular nodal reentry tachycardia (AVNRT) or Atrioventricular reentrant tachycardia (AVRT)). If patient had a prior ablation for PSVT, patient must have documented evidence of PSVT post-ablation
• Signed written informed consent/assent obtained
• Per Investigator's decision, females of childbearing potential (defined as any woman or adolescent who has begun menstruation) must additionally satisfy the following criteria:
• Negative pregnancy test at screening
• Adequate contraception, unless total abstinence is used
• Willing and able to comply with study procedures.

Exclusion Criteria:

Patients will be excluded from the study if they meet any of the following criteria:
• History or presence of any of the following at the screening visit:
• Patients with a history of atrial arrhythmia that does not involve the AV node as part of the tachycardia circuit (e.g., atrial fibrillation, atrial flutter, atrial tachycardia) are not eligible
• Permanent junctional reciprocating tachycardia
• Ventricular pre-excitation (e.g., delta wave on ECG, Wolff Parkinson White syndrome)
• Second- or third-degree AV block
• Sick sinus syndrome or clinically significant bradycardia (\<50 bpm or equivalent in this patient population) on the resting ECG
• Ventricular tachycardia
• Long QT syndrome
• Major structural heart disease (e.g., Ebstein's anomaly, corrected congenital heart disease) or symptoms of congestive heart failure (New York Heart Association class II to IV).
• Evidence of impaired liver function (alanine aminotransferase \[ALT\] and/or aspartate aminotransferase \[AST\] \>3 x upper limit of normal for age and gender) at the Screening Visit
• Evidence of End-Stage Renal Disease as determined by an estimated glomerular filtration rate assessed at the Screening Visit of \<15 mL/min/1.73m2, or requiring hemodialysis;
• Treatment with any of the following that cannot or will not be discontinued during study participation:
• Any investigational drug within 60 days prior to study drug administration
• IV beta-adrenergic blocking drugs (e.g., propranolol, esmolol), calcium channel blocking drugs (e.g., verapamil, diltiazem) or amiodarone within 24 hours prior to study drug administration
• Oral amiodarone within 30 days prior to study drug administration
• Class I or III antiarrhythmic agents (e.g., flecainide, propafenone, sotalol) within five half-lives prior to study drug administration
• Any other drug that has a contraindication with verapamil
• Known hypersensitivity to verapamil or to any of the excipients of the study drug
• Any other significant co-morbid condition that may have a negative impact on the patient's participation in the study or likely to result in non-compliance
• History of hyperthyroidism
• Current pregnancy or breastfeeding

Interventions: DRUG: Etripamil NS

Conditions: Paroxysmal Supraventricular Tachycardia

Keywords: PSVT

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A Research Study of Abdominal Ultrasound (FAST) in Children With Blunt Torso Trauma (FAST)

Description: Bleeding from intra-abdominal injuries is a leading cause of traumatic deaths in children. Abdominal CT is the reference standard test for diagnosing intra-abdominal injuries. Compelling reasons exist, however, to both aggressively evaluate injured children for intra-abdominal injuries with CT and to limit abdominal CT evaluation to solely those at non-negligible risk. The focused assessment sonography for trauma (FAST) examination can help focus patient evaluation in just this manner by potentially safely decreasing abdominal CT use in low risk children. This research study is a multicenter, randomized, controlled trial to determine whether use of the FAST examination, a bedside abdominal ultrasound, impacts care in 3,194 hemodynamically stable children with blunt abdominal trauma. The overall objectives of this proposal are 1) to determine the efficacy of using the FAST examination during the initial evaluation of children with blunt abdominal trauma, and 2) to identify factors associated with abdominal CT use in children considered very low risk for IAI after a negative FAST examination. The long-term objective of the research is to determine appropriate evaluation strategies to optimize the care of injured children, leading to improved quality of care and a reduction in morbidity and mortality.

Contact(s):

Allison Ackermann - allison.ackermann@cchmc.org

Sex: ALL

Age: Up to 17 years old

Phase: NA

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05910567

Show full eligibility criteria

Children younger than 18 years of age (0 to 17.9999 years) with blunt abdominal trauma presenting to the participating EDs within 24 hours of the traumatic event will be eligible if the do not meet any exclusion criteria and meet any one of the following inclusion criteria.

Inclusion Criteria:

• Blunt torso trauma resulting from a significant mechanism of injury: * Motor vehicle collision: greater than 60 mph, ejection, or rollover * Automobile versus pedestrian/bicycle: automobile speed \> 25 mph * Falls greater than 20 feet in height * Crush injury to the torso * Physical assault involving the abdomen
• Decreased level of consciousness (Glasgow Coma Scale (GCS) score 9-14 or below age-appropriate behavior) in association with blunt torso trauma
• Blunt traumatic event with any of the following (regardless of the mechanism): * Extremity paralysis * Multiple long bone fractures (e.g., tibia and humerus fracture)
• History and physical examination suggestive of blunt torso trauma of any mechanism (including mechanisms of injury of less severity than mentioned above)

Exclusion Criteria:

The following patients will be excluded from the study:
• Age-adjusted low blood pressure (Hemodynamic instability) * Patients will be excluded for prehospital or initial age-adjusted ED low blood pressure. This is because the standard evaluation of these patients involves immediate FAST based on prior work by our group. Low blood pressure is determined based upon the patient's age, and will be defined as a systolic blood pressure less than 70 mm Hg for patients younger than 1 month, less than 80 mm Hg for ages 1 month to 5 years, and less than 90 mm Hg for ages over 5 years.
• Penetrating trauma: Patients who are victims of stab or gunshot wounds
• Traumatic injury occurring \> 24 hours prior to the time of presentation to the ED
• Transfer of the patient to the ED from an outside facility with abdominal CT scan, diagnostic peritoneal lavage, or laparotomy previously performed
• Transferred with FAST exam already performed at outside hospital
• Patients with known disease processes resulting in intraperitoneal fluid including liver failure and the presence of ventriculoperitoneal shunts
• Initial GCS score ≤ 8 as it is standard for children with GCS scores ≤ 8 to undergo abdominal CT if blunt abdominal trauma is suspected
• Known pregnancy
• Known prisoner
• Known intra-abdominal injury diagnosed within 30 days prior of this ED visit

Interventions: DIAGNOSTIC_TEST: Focused Assessment with Sonography for Trauma (FAST) Examination, OTHER: No Intervention: Standard of Care - Without the FAST Examination

Conditions: Blunt Trauma to Abdomen, Wounds and Injuries, Abdomen Injury, Abdominal Injury, Abdomen, Acute

Keywords: Child, Wounds and Injuries, Blunt Trauma to Abdomen, Abdomen Injury, Blunt Abdominal Trauma

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Safety and Efficacy Study of Intracystic TARA-002 for the Treatment of Lymphatic Malformations in Participants 6 Months to Less Than 18 Years of Age (STARBORN-1)

Description: This is a Phase 2a/b single arm open label study to evaluate the safety, reactogenicity, and efficacy of intracystic injection of TARA-002 in participants 6 months to less than 18 years of age for the treatment of macrocystic and mixed cystic lymphatic malformations. The Phase 2a safety lead-in, age de-escalation study is designed to establish the safety of TARA-002 in older participants 6 years to less than 18 years before proceeding to younger participants 2 years to less than 6 years, then 6 months to less than 2 years. The Phase 2b is an expansion study in which enrollment of participants will be initiated after safety has been established in each cohort during the Phase 2a safety lead-in study. Each participant will receive up to 4 injections of TARA-002 spaced approximately 6 weeks apart.

Contact(s):

Kimberly Whittaker - kimberly.whittaker@cchmc.org

Sex: ALL

Age: 6 months to 18 years old

Phase: PHASE2

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05871970

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Interventions: BIOLOGICAL: TARA-002

Conditions: Lymphatic Malformation

Keywords: Macrocystic lymphatic malformations, Mixed-cystic lymphatic malformations

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A Study to Learn More About How Well Finerenone Works, How Safe it is, and How it Moves Into, Through, and Out of the Body Compared to Placebo When Taken With Standard Treatment in Children With Heart Failure and Left Ventricular Systolic Dysfunction (FIORE)

Description: Researchers are looking for a better way to treat children who have heart failure with left ventricular systolic dysfunction (LVSD). Heart failure is a serious condition where the heart is unable to pump enough blood to meet the body's needs. This can lead to symptoms like shortness of breath, fatigue, and poor growth in children. The study treatment, finerenone (also called BAY94-8862), works by blocking a protein involved in inflammation, scarring, and thickening of the heart and blood vessels. This may help the heart to pump blood more effectively. This is the first study to explore its use specifically for children with heart failure and LVSD. The main purpose of this study is to learn if finerenone works to help the heart compared to placebo in children with heart failure and LVSD. For this, the researchers will collect and analyze data on the levels of a protein called NT-proBNP in the blood, which indicates heart stress, and monitor the safety of the treatment. The study will include children with heart failure and LVSD aged from 6 months to less than 18 years. The study participants will be randomly assigned to one of two treatment groups. Based on their group, they will receive either finerenone or a placebo for a duration of 3 months. A placebo looks like a treatment but does not have any medicine in it. Throughout the study, all participants will continue to receive their standard heart failure treatments. At the start of this study, the doctors will check each participant's medical history and current medications. If participants qualify for the treatment phase, they will undergo treatment for about 90 days. During this time, they will visit the study site at least 3 times. During these visits, the participants will: * have their blood pressure, heart rate, temperature, respiratory rate, height and weight measured * have their heart examined by electrocardiogram (ECG) and echocardiogram * have blood samples taken * have physical examinations * answer questions about their medication and whether they have any adverse events, or have their parents or guardians' answers An adverse event is any medical problem that a participant has during a study. Doctors keep track of all adverse events that happen in studies, even if they do not think the adverse events might be related to the study treatments. After the initial three-month study, eligible participants will have the option to join a nine-month open-label extension study where all will receive finerenone. Participants who choose not to enroll in the extension will have a follow-up visit 30 days after their last treatment.

Contact(s):

Elise Pickering - Elise.Pickering@cchmc.org

Sex: ALL

Age: 6 months to 17 years old

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT07188805

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Inclusion Criteria:

* Participants must be 6 months to \<18 years old at the time when the informed consent/assent is signed. * Left ventricular systolic dysfunction (LVSD) with left ventricular ejection fraction (LVEF) ≤ 50% at screening assessed by echocardiography. * Elevated NT-pro BNP levels * \>500 ng/l for children ≥ 6 months to \< 2 years of age * \>300 ng/l, for children ≥ 2 years to \<18 years * Heart failure etiologies including congenital heart defects (CHD) with biventricular physiology and systemic LV; idiopathic cardiomyopathy (CM); familial/inherited and/or genetic CM; history of myocarditis (diagnosis of an acute episode was at least 3 months prior to randomization); neuromuscular disorder (eg, duchenne muscular dystrophy); inborn error of metabolism; mitochondrial disorder; acquired (chemotherapy, iatrogenic, infection, rheumatic, or nutritional); ischemic (eg, Kawasaki disease and postoperative heart failure \[HF\]); LV noncompaction. * Receiving standard of care (SoC) treatment for heart failure according to local guidelines or investigator´s discretion and being on a stable regimen for 30 days prior to randomization. * Study participants must have a body weight ≥ 4.0 kg at Visit 1.

Exclusion Criteria:

* Serum/plasma potassium (based on local laboratory results): * \> 5.0 mmol/L for children ≥ 2 years of age at either screening or randomization visit * \> 5.3 mmol/L for children ≥ 6 months to \< 2 years of age at either screening or randomization visit (if estimated glomerular filtration rate \[eGFR\] \< 60 mL/min/1.73m², threshold of \> 5.0 mmol/L will be used) * Severe renal dysfunction with eGFR \< 30 mL/min/1.73m² at randomization visit based on local laboratory results. * Systolic blood pressure (SBP) \< 5th percentile for age, sex and height at screening or randomization. * Sustained or symptomatic arrhythmias not controlled by drug or device therapy within 30 days prior to randomization. * Treatment with a mineralocorticoid receptor antagonist (e.g., spironolactone, eplerenone) within 30 days of randomization. * Requirement of any intravenous (IV) vasoactive agents, mechanical ventilation, mechanical circulatory support within 30 days prior to randomization. * Recent surgical procedure or other intervention to correct or palliate CHD within 3 months prior to randomization or anticipated to undergo cardiac surgery during the 3 months after randomization.

Interventions: DRUG: Finerenone (Kerendia, BAY94-8862), DRUG: Placebo

Conditions: Left Ventricular Systolic Dysfunction, Heart Failure (Pediatric)

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Sleep and Adolescent Vaccine Immunogenicity Pilot/Observational Study (SAVI)

Description: This study tests whether the sleep habits of 11-12 years-olds impact their response to a vaccine that protects against meningococcal illness. That vaccine is typically given at that age by primary care doctors, but nobody has studied how sleep affects children's response to it. This could be important because research on adults suggests that sleep affects the immune system. Families will have their child keep their regular sleep schedule during the 5-week study. They will wear a special wristwatch at night to track their sleep. Each day they will fill out a short online form. They and a parent/guardian will come to Cincinnati Children's twice for 1 - 1 ½ hours. During the first visit, the child get the vaccine. During the second, they have a blood test.

Contact(s):

Dean Beebe - BEEBD0@cchmc.org

Sex: ALL

Age: 11 years to 12 years old

Healthy Volunteers:

This study is also accepting healthy volunteers

System ID: NCT07636525

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Conditions: Vaccination

Keywords: Sleep

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A Study to Learn More About How Safe Finerenone is, When it is Taken for a Longer Time With Standard Treatment, in Children and Young Adults With Heart Failure and Left Ventricular Systolic Dysfunction (FIORELLO)

Description: Researchers are looking for a better way to treat children and young adults who have heart failure with left ventricular systolic dysfunction (LVSD). Heart failure with left ventricular systolic dysfunction (LVSD) is a condition where the left side of the heart is weak and struggles to pump blood effectively, leading to symptoms like shortness of breath, fatigue, and poor growth. The study treatment, finerenone (also called BAY94-8862), is under development to treat newborns, children, and young adults with heart failure and LVSD. It works by blocking a protein that contributes to inflammation, scarring, and thickening in the heart and blood vessels, which may help the heart pump more blood effectively. The main purpose of this study is to learn about how safe finerenone is and how well it works in the long-term treatment of heart failure and LVSD. To understand how safe the treatment is, the study team will gather information on the number of patients who experience medical problems after taking finerenone, also known as "treatment emergent adverse events" (TEAEs). Additionally, they will collect blood samples to measure levels of an electrolyte called potassium and monitor blood pressure. They will also assess kidneys function using the estimated glomerular filtration rate (eGFR). In this study, which is an extension of the earlier done FIORE study, finerenone will also be studied in newly enrolled newborns under 6 months with heart failure and LVSD and children and young adults from the FIORE study. The participants will be aged from newborns up to 18 years. All the participants will continue to receive their standard treatment as routine care for heart failure, along with finerenone during the study. The participants will be in the study for around 10 to 11 months, depending on whether they rolled-over from the FIORE study or are newly enrolled newborns and infants \<6 months of age. They will take study treatment for up to 9 months. During this period, at least 6 visits are planned for participants. During these visits, the study team will: * have their blood pressure, heart rate, temperature, respiratory rate, height and weight measured * have blood samples taken * have physical examinations * have their heart examined by an electrocardiogram and echocardiography * answer questions about their medication and whether they have any adverse events, or have their parents or guardians' answer * for newborns and infants, evaluate the acceptability of the study drug formulation through parents or guardians' feedback. An adverse event is any medical problem that a participant has during a study. Doctors keep track of all adverse events that happen in studies, even if they do not think the adverse events might be related to the study treatments. The doctors will check the participants' health a month after the participants take their last treatment.

Contact(s):

Elise Pickering - elise.pickering@cchmc.org

Sex: ALL

Age: Up to 18 years old

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT07192952

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Inclusion Criteria:

* For participants rolling over from randomized controlled trial (RCT): Prior participation in the finerenone Phase 3 study FIORE (21466) and not permanently discontinued from the study intervention prior to the end of treatment (EoT) visit in FIORE. * For newly enrolled infants \<6 months of age: Left ventricular systolic dysfunction (LVSD) with left ventricular ejection fraction (LVEF) ≤ 50% at screening assessed by echocardiography. * For newly enrolled infants \<6 months of age: Elevated NT-pro BNP levels (\> 500 ng/L) at screening. * For newly enrolled infants \<6 months of age: Heart failure (HF) etiologies include congenital heart defects (CHD) with biventricular physiology and systemic LV; idiopathic cardiomyopathy (CM); familial/inherited and/or genetic CM; history of myocarditis (diagnosis of an acute episode at least 3 months prior to treatment assignment); neuromuscular disorder; inborn error of metabolism; mitochondrial disorder; acquired (chemotherapy, iatrogenic, infection, rheumatic, or nutritional); ischemic (e.g., Kawasaki disease and postoperative HF); LV noncompaction. * For newly enrolled infants \<6 months of age: Receiving standard of care (SoC) treatment for heart failure according to local guidelines or investigator´s discretion (on a stable regimen for 30 days before baseline). * Newly enrolled newborns and infants \< 6 months of age must have a body weight of ≥3 kg at Visit 1.

Exclusion Criteria:

* For participants rolling over from randomized controlled trial (RCT): To roll-over to FIORELLO, all participants: Potassium (K+) \>5.5 mmol/L based on local laboratory results. After unblinding: * For participants who received finerenone in FIORE: K+ \>5.5 mmol/ L * For participants who received placebo in FIORE: K+ \>5.0 mmol/L for children ≥2 years of age, and \>5.3 mmol/L for children \<2 years of age (if eGFR is \<60 mL/min/1.73m² for participants \<2 years of age, the serum potassium threshold of \>5.0 mmol/L will be used for exclusion) * For newly enrolled newborns and infants \< 6 months of age: Potassium ≥ 5.3 mmol/l (if eGFR is \<60 mL/min/1.73m², the serum potassium threshold of \>5.0 mmol/L will be used for exclusion). * For participants rolling over from RCT: Severe renal dysfunction with estimated glomerular filtration rate (eGFR) \< 30 ml/min/1.73m² based on local laboratory results at FIORELLO Visit 1. * For newly enrolled infants \< 6 months of age: Severe renal dysfunction with eGFR \< 30 ml/min/1.73m2 at screening or Visit 1. * Treatment with a mineralocorticoid receptor antagonist, other than the study intervention, (e.g., spironolactone, eplerenone) within 30 days of Visit 1. * Requirement of any intravenous (IV) vasoactive agents; mechanical ventilation; mechanical circulatory support; sustained or symptomatic arrhythmias not controlled by drug or device therapy within 30 days prior to study treatment.

Interventions: DRUG: Finerenone (Kerendia, BAY94-8862)

Conditions: Left Ventricular Systolic Dysfunction, Heart Failure (Pediatric)

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Study of CM4620 to Reduce the Severity of Pancreatitis Due to Asparaginase

Description: This is a phase I/II clinical trial assessing the tolerability and efficacy of CM4620 in children and young adults with acute pancreatitis caused by asparaginase. The tolerability of CM4620 when given to patients receiving frontline chemotherapy will be determined. The effectiveness in reducing the severity of pancreatitis will be estimated. Primary Objectives To assess the safety of CM4620 administration in children and young adults with asparaginase associated pancreatitis (AAP). To profile dose-limiting toxicities and responses of the patients treated in the dose-finding phase. To estimate the efficacy of CM4620 to prevent pseudocyst or necrotizing pancreatitis in children with AAP. Secondary Objectives To determine the effect of CM4620 on the incidence of severe pancreatitis To determine the effect of CM4620 on the incidence of Systemic Inflammatory Response Syndrome (SIRS).

Contact(s):

Lori Backus - lori.backus@cchmc.org

Sex: ALL

Age: Up to 21 years old

Phase: PHASE1

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT04195347

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Interventions: DRUG: CM4620

Conditions: Acute Pancreatitis

Keywords: Acute Pancreatitis, Asparaginase, Asparaginase Associated Pancreatitis, Acute Lymphoblastic Leukemia, Acute Lymphoblastic Lymphoma, CM4620, Children, SIRS, Systemic Inflammatory Response, Young Adults

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STRW-T Intervention for Autistic Adolescents in 11th and 12th Grade (STRW-T)

Description: The current study seeks to compare outcomes of a telehealth intervention targeting daily living skills (Surviving and Thriving in the Real World - Telehealth, or STRW-T) intervention to a control group telehealth intervention targeting social skills (Program for the Education and Enrichment of Relational Skills- Telehealth, or PEERS-T). The key endpoint will be change in daily living skills on primary and secondary outcome measures at the end of treatment.

Contact(s):

Carrie Fassler - carrie.fassler@cchmc.org

Sex: ALL

Age: 15 years to 21 years old

Phase: NA

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06552286

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Interventions: BEHAVIORAL: STRW-T, BEHAVIORAL: PEERS-T

Conditions: Autism Spectrum Disorder

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A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition (EXCEL)

Description: XLMTM (X-linked myotubular myopathy) is a serious genetic muscle condition. It is caused by changes in the MTM1 gene which stops or slows down normal muscle development, causing severe muscle weakness. There is currently no cure for XLMTM. Ongoing care is needed to manage symptoms and prevent further medical problems from this condition. Recent research shows that individuals with XLMTM often have reduced bile flow which can affect liver and gallbladder health. Bile is a liquid made in the liver that helps digest fat. Ongoing liver health checks may help with the routine care of people with XLMTM. There is a need to understand liver problems that develop in individuals with XLMTM over time. The main aim of the study is to learn how many boys with XLMTM have new cases of liver problems during the study. This study is about collecting information only. This is known as an observational study. The individual's doctor decides on treatment, not the study sponsor (Astellas). In this study, boys under 18 diagnosed with XLMTM will be followed for about 1 year. The health of their liver and gallbladder will be checked about every 6 weeks. This can be done at home, if preferred. A scan called a Fibroscan (also known as transient elastography) will check for signs of scarring in the liver (fibrosis) and the build-up of lipids. It is suggested that each boy will have a Fibroscan when they start the study and another scan when they complete the study. This study will help understand liver, gallbladder, and bile duct issues in individuals with XLMTM over time. The goal is to improve their care and provide information to use in future clinical studies.

Contact(s):

Morgan Morgan Thurza - morgan.thurza@cchmc.org

Sex: MALE

Age: Up to 17 years old

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06581146

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Interventions: OTHER: No Intervention

Conditions: X-Linked Myotubular Myopathy

Keywords: XLMTM, Hepatobiliary

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MEKTOVI® for the Treatment of Pediatric Adamantinomatous Craniopharyngioma

Description: MEKTOVI (binimetinib) is an oral, highly selective reversible inhibitor of mitogen-activated extracellular signal regulated kinase 1 (MEK1) and MEK2. The biological activity of binimetinib that has been evaluated bith in vitro and in vivo in a wide variety of tumor types In this Phase II, the drug will be used to treat pediatric patients diagnosed with recurrent Adamantinomatous Craniopharyngioma including patients who have undergone surgery and/or radiation therapy.

Contact(s):

- cancer@cchmc.org

Sex: ALL

Age: 1 year to 39 years old

Phase: PHASE2

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05286788

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Inclusion Criteria:

• Age: Patients must be ≥ 12 months and ≤ 39 years of age at the time of study enrollment.
• Diagnosis: Patients with histologically-confirmed adamantinomatous craniopharyngioma (ACP) Histologic confirmation of ACP may be made on solid tumor or, if no solid tumor can be safely obtained, cyst fluid with classic ACP characteristics of thick, cholesterol-rich, greenish-brown liquid in the context of imaging features consistent with craniopharyngioma, including lobulated, cystic/solid mass with calcifications that originates in the sellar/suprasellar region.
• Disease Status: Patients must have measurable disease. * Stratum 1: Patients with progressive or recurrent ACP who demonstrate cystic and/or solid recurrence or progression at least 6 months post completion of radiation therapy * Stratum 2 (NOT CURRENTLY ENROLLING): Patients with measurable ACP who have undergone surgery but have NOT previously undergone irradiation (but may have received prior systemic or intracystic therapy). Progressive disease is allowed but not required.
• Performance Level: Karnofsky ≥ 50% for patients \> 16 years of age and Lansky ≥ 50 for patients ≤ 16 years of age (See Appendix I). Note: Neurologic deficits in patients with CNS tumors must have been stable for at least 7 days prior to study enrollment. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
• Prior Therapy: Patients must have recovered or stabilized from the acute toxic effects of prior treatments * Biologic (anti-neoplastic agent): At least 7 days must have elapsed after the last (systemic or intracystic) dose of a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair * Immunotherapy: At least 42 days after the completion of any type of systemic immunotherapy, e.g. tumor vaccines. * Monoclonal antibodies: At least 21 days after the last dose of a monoclonal antibody. * Radiation therapy: Patients must have had their last (conventional or hypofractionated) fraction of: a) Focal irradiation \> 6 months prior to enrollment and b) No prior craniospinal irradiation is permitted. * Corticosteroids: Patients receiving dexamethasone must be on a stable or decreasing dose for at least 1 week prior to enrollment * Myelosuppressive systemic therapy: At least 21 days must have elapsed after the last systemic myelosuppressive therapy. * Surgery: At least 6 weeks must have elapsed since major or intermediate surgery. Major surgery includes major craniotomy for tumor resection of cyst fenestration, organ resection, and exploratory laparotomy. Intermediate procedures include ventriculoperitoneal shunt placement, stereotactic brain biopsy, and intraventricular catheter placement. Minor procedures that are not excluded include skin biopsy/incision and drainage, bone marrow aspirate, and central venous catheter placement, ommaya aspirations, lumbar punctures, and nasal endoscopy to remove packing.
• Organ Function Requirements Adequate Bone Marrow Function Defined as: * Peripheral absolute neutrophil count (ANC) ≥1000/mm3 * Platelet count ≥100,000/mm3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment) * Hemoglobin \>8 g/dL (may be transfused) Adequate Renal Function Defined as: * Creatinine clearance or radioisotope GFR \> 70ml/min/1.73 m2 or * A serum creatinine based on (Schwartz et al. J. Peds, 106:522, 1985) age/gender as follows:
• to \< 2 years: maximum serum creatinine 0.6 mg/dL for males and females. 2 to \< 6 years: maximum serum creatinine 0.8 mg/dL for males and females. 6 to \< 10 years: maximum serum creatinine 1.0 mg/dL for males and females. 10 to \< 13 years: maximum serum creatinine 1.2 mg/dL for males and females. 13 to \< 16 years: maximum serum creatinine 1.5 mg/dL for males and 1.4 mg/dL for females. ≥ 16 years: maximum serum creatinine 1.7 mg/dL for males and 1.4 mg/dL for females. Adequate Liver Function Defined as: * Total bilirubin ≤ 1.5 × institutional upper limit of normal * AST (SGOT) ≤ 2.5 × institutional upper limit of normal * ALT (SGPT) ≤ 2.5 × institutional upper limit of normal Adequate Cardiac Function Defined as: * Left Ventricular Ejection Fraction greater than the institutional lower limit of normal by echocardiogram * QTc ≤ 480 msec (by Bazett formula) Adequate Neurologic Function Defined as: * Patients with neurological deficits should have deficits that are stable for a minimum of 1 week prior to enrollment. * Patients with current seizure disorders may be enrolled if seizures are well-controlled on antiepileptic therapies.
• Informed Consent: All patients and/or their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines

Exclusion Criteria:

• Pregnancy or Breast-Feeding: Pregnant or breast-feeding women will not be entered on this study due to unknown risks of fetal and teratogenic adverse events as seen in animal/human studies. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method for at least 90 days after discontinuation of drug for females and at least 60 days for males. For females of childbearing potential, agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive methods (bilateral tubal ligation, male sterilization, hormonal contraceptives that inhibit ovulation, hormone-releasing intrauterine devices, and copper intrauterine devices; hormonal contraceptive methods must be supplemented by a barrier method) and agreement to refrain from donating eggs are required. For males of reproductive potential, agreement to remain abstinent (refrain from heterosexual intercourse) or use a condom, and agreement to refrain from donating sperm.
• Gastrointestinal Disease: * Patients with a history of serious gastrointestinal disease, including inflammatory bowel disease or gastrointestinal perforation * Patients who are unable to absorb enteral medications * Administration via NG/NJ/G-tube is allowed
• Concomitant Medications * Corticosteroids: Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible. * Investigational Drugs: Patients who are currently receiving another investigational drug are not eligible. * Anti-cancer Agents: Patients who are currently receiving other anti-cancer agents are not eligible.
• Study Specific: * Patients who have an uncontrolled infection are not eligible. * Patients who have received any live or attenuated vaccinations within three months prior to start of therapy are not eligible. * Any significant concurrent medical or surgical condition that would jeopardize the patient's safety or ability to complete the study, including, but not limited to, disease of the nervous, renal, hepatic, cardiac (such as symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia), pulmonary, or endocrine system * Patients who have a history of Human Immunodeficiency Virus, Hepatitis B Virus, Hepatitis C Virus or Tuberculosis infection are not eligible. * Patients who have received a prior solid organ transplantation are not eligible. * Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible. * Patients who have a history of alcohol, drug, or chemical abuse within 6 months of screening. * Patients who have had surgery within the last 6 weeks or who have concerns for poor postsurgical wound healing. * Patients who have a history of allergic reactions attributed to compounds of similar chemical or biologic composition to tocilizumab and its excipients are not eligible.

Interventions: DRUG: Binimetinib Oral Tablet [Mektovi]

Conditions: Adamantinous Craniopharyngioma, Recurrent Adamantinomatous Craniopharyngioma

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A Study to Find Out if BI 764198 Helps Adults and Adolescents With a Kidney Condition Called Focal Segmental Glomerulosclerosis (FSGS)

Description: PODOMOUNT-pFSGS This study is open to adults and adolescents with a kidney condition called focal segmental glomerulosclerosis (FSGS). The purpose of this study is to find out whether a medicine called BI 764198 helps people with FSGS. Participants are put into 2 groups randomly, which means by chance. Every participant has an equal chance of being in each group. One group takes BI 764198 tablets, and the other group takes placebo tablets. Placebo tablets look like BI 764198 tablets but do not contain any medicine. Participants take a tablet once a day for up to 2 years. All participants also continue their standard medication for FSGS. Participants are in the study for up to 2 years. During this time, they visit the study site about every 3 months. Participants regularly collect urine samples. This is done to check their kidneys. The results are compared between the two groups to see whether the treatment works. The doctors also regularly check participants' health and take note of any unwanted effects.

Contact(s):

Ilham Boutahri - Ilham.Boutahri@cchmc.org

Sex: ALL

Age: 12 years and over

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT07220083

Show full eligibility criteria

Inclusion criteria:
• Male or female participants ≥12 years old on the day of signing informed consent/assent (Visit 1)
• Weight of ≥40 kg at the screening visit (Visit 1)
• Body mass index (BMI) of ≤40 kg/m² at the screening visit (Visit 1)
• Participants with a diagnosis prior to the screening visit (Visit 1) of either: * Biopsy-confirmed primary focal segmental glomerulosclerosis (pFSGS) (based on Investigator's judgement) OR * Genetic focal segmental glomerulosclerosis (FSGS) resulting from a gain-of-function mutation in the transient receptor potential cation subfamily C member 6 (TRPC6) gene (based on historical genetic test)
• Urine protein-creatinine ratio (UPCR) ≥1500 mg/g based on the mean of the spot urine sample and first morning void (FMV) urine sample (both assessed by central laboratory) at the screening visit (Visit 1)
• Estimated glomerular filtration rate (eGFR) * For adult participants (≥18 years): ≥25 mL/min/1.73 m² (chronic kidney disease epidemiology collaboration (CKD-EPI) formula based on serum cystatin C) at the screening visit (Visit 1) * For adolescent participants (12 to \<18 years): ≥25 mL/min/1.73 m² based on chronic kidney disease under 25 years (CKiD U25) formula using serum cystatin C at the screening visit (Visit 1) Further inclusion criteria apply. Exclusion criteria:
• Known monogenic or syndromic causes of FSGS (with the exception of TRPC6 gain-of-function gene mutations)
• Clinical or histologic evidence of secondary adaptive or toxic forms of FSGS (based on Investigator's judgement)
• FSGS of undetermined cause (FSGS-UC) with a diagnosis prior to the screening visit (Visit 1) (based on Investigator's judgement)
• A history of organ transplantation or planned organ transplantation during the course of the trial
• Use of intravenous immunosuppressive agents (e.g. cyclophosphamide, rituximab, obinutuzumab) in the last 6 months prior to screening (Visit 1) Further exclusion criteria apply.

Interventions: DRUG: BI 764198, DRUG: Placebo

Conditions: Focal Segmental Glomerulosclerosis

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Study of Ribociclib and Everolimus in HGG and DIPG or Ribociclib and Temozolomide in DHG, H3G34-mutant

Description: The goal of this study is to determine the efficacy of the 1) ribociclib and everolimus to treat pediatric and young adult patients newly diagnosed with a high-grade glioma (HGG), including DIPG, that have genetic changes in pathways (cell cycle, PI3K/mTOR) that these drugs target or 2) ribociclib and temozolomide to treat pediatric and young adult patients newly diagnosed with diffuse hemispheric glioma (DHG), H3G34-mutant. The main question the study aims to answer is whether the combinations of ribociclib and everolimus or ribociclib and temozolomide can prolong the life of patients diagnosed with HGG/DIPG or DHG H3G34-mutant.

Contact(s):

- cancer@cchmc.org

Sex: ALL

Age: 12 months to 39 years old

Phase: PHASE2

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05843253

Show full eligibility criteria

TarGeT-A study strata definitions Part1: Initial Feasibility Study for the combination of ribociclib PfOS formulation with everolimus: Enrollment on this cohort will be limited to patients aged \<21 years with primary intracranial localized HGG and DIPG Part 2 * Stratum A: Patients with localized, intracranial, non-pontine, and non-thalamic HGG (who do not meet criteria for strata C-D) * Stratum B: Patients with DIPG * Stratum C: Patients with primary thalamic, spinal cord, and/or secondary/radiation-related HGG. * Stratum D: Patients with metastatic/disseminated HGG, multifocal HGG, and/or gliomatosis cerebri who received CSI. Stratum E * Stratum E: Patients with localized DHG, H3G34-mutant.

Inclusion Criteria:

• Inclusion criteria already met to enroll on TarGeT-SCR (central molecular and histopathologic screening) based on:
• 1) Age: patients must be ≥12 months and ≤39 years of age at the time of enrollment on TarGeT-SCR. For the Part 1 Initial Feasibility Cohort (receiving ribociclib and everolimus) only: patients must be \<21 years of age at the time of enrollment on this protocol.
• 2) Diagnosis: patients with newly-diagnosed HGG, including DIPG are eligible. All patients must have histologic confirmation tumor tissue from diagnostic biopsy or resection, without exceptions. The diagnosis of HGG, including DIPG, must have been confirmed through TarGeT-SCR: * For the diagnosis of DIPG, patients must have a tumor with pontine epicenter and diffuse involvement of at least 2/3 of the pons, with histopathology, consistent with diffuse WHO grade 2-4 glioma * All other HGGs must be WHO grade 3 or 4.
• 3) Disease status: There are no disease status requirements for enrollment * Patients without measurable disease are eligible. * Patients with metastatic or multifocal disease or gliomatosis cerebri who received upfront CSI are eligible * Patients with a primary spinal HGG are eligible * Patients with secondary, radiation-related HGG are eligible.
• Inclusion criteria for assignment to TarGeT-A, for all strata:
• 1) Presence of at least one relevant actionable somatic alteration, detailed here: * Pathogenic alterations presumed to cause activation of cell cycle: * Amplification of CDK4 or CDK6 * Deletion of CDKN2A, CDKN2B, or CDKN2C * Amplification of CCND1 or CCND2 * Pathogenic alterations presumed to cause activation of the PI3K/mTOR pathway: * Deletion or mutation of PTEN * Mutation or amplification of PIK3CA * Mutation of PIK3R1 * Deletion or mutation of TSC1 or TSC2 * Patients with evidence of homozygous (biallelic) RB1 loss by sequencing are excluded from TarGeT-A * Patients whose tumors harbor other alterations suspected to activate the cell cycle and/or PI3K/mTOR pathway could potentially also be eligible, but only following consensus recommendation by the international multidisciplinary molecular screening committee. * For Stratum E: H3G34 (R/V) mutation
• 2) Performance Level: Karnofsky ≥ 50% for patients \> 16 years of age and Lansky ≥ 50 for patients ≤ 16 years of ag. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
• 3) Prior Therapy for HGG: * Surgery, RT, dexamethasone are permissible. Temozolomide administered concurrently with RT is permissible. Avastin/bevacizumab use is permitted given the last dose was administered \> 21 days prior to enrollment. No other prior anticancer therapy for HGG will be allowed. * Patients must have received photon or proton RT. * Patients must have started RT \< 42 calendar days from initial diagnosis defined as the date of diagnostic biopsy or resection. If a patient underwent 2 upfront surgeries (e.g., biopsy then resection or debulking), this is the date of the second surgery. * RT delivered via photon or proton beam, must have been administered at a standard dose including (54 Gy in 30 fractions for DIPG, 54-59.4 Gy in 30-33 fractions), 45 Gy-54 Gy for primary spinal disease, and/or 36 Gy-39.6 Gy craniospinal for patients with spinal or leptomeningeal metastatic disease with supplemental boost to 45-54 Gy for metastasis within the thecal sac and 54 Gy-60 Gy for intracranial metastasis. Any variances in the radiotherapy dose within 10% of the standard doses outlined above will be discussed with the Sponsor-Investigator to confirm eligibility prior to study enrollment. * Patients must enroll and start treatment No later than 35 calendar days post-completion of RT. The earliest patients can begin protocol treatment is 28 calendar days post-completion of RT.
• 4) Organ Function Requirements
• 4.1) Adequate Bone Marrow Function Defined as: * Peripheral absolute neutrophil count (ANC) ≥ 1000/mm3 * Platelet count ≥ 100,000/mm3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment) * Hemoglobin \>8 g/dL (may be transfused)
• 4.2) Adequate Renal Function Defined as: * Creatinine clearance or radioisotope GFR ≥ 70ml/min/1.73 m2 OR * Maximum serum creatinine based on (Schwartz et al. J. Peds, 106:522, 1985) age/gender as follows: 1 to \< 2 years=0.6 mg/dL for males and females; 2 to \< 6 years=0.8 mg/dL for males and females; 6 to \< 10 years= 1.0 mg/dL for males and females; 10 to \< 13 years=1.2 mg/dL for males and females. 13 to \< 16 years=1.5 mg/dL for males and 1.4 mg/dL for females.
• 4.3) Adequate Liver Function Defined as: * Total bilirubin must be ≤ 1.5 times institutional upper limit of normal for age * AST(SGOT)/ALT(SGPT) ≤ 3 times institutional upper limit of normal * Serum albumin ≥ 2g/dL
• 4.4) Adequate Cardiac Function Defined as: * Ejection fraction of ≥ 50% by echocardiogram * QTc ≤ 450 msec (by Bazett formula)
• 4.5) Adequate Neurologic Function Defined as: Patients with seizure disorder may be enrolled if well-controlled on anticonvulsants that are not strong inducers or inhibitors of CYP3A4/5.
• 4.6) Adequate Pulmonary Function Defined as: No evidence of dyspnea at rest, and a pulse oximetry \>94% on room air if there is clinical indication for determination.
• 5) Ability to take medications by mouth: For ribociclib and everolimus strata, patients must be able to take study medications by mouth as administration via NG/NJ/G tube is not allowed.
• 6) Informed Consent: All patients and/or their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines
• 7) Contraception: Male and female patients of childbearing potential must be willing to use a highly effective contraception method. Exclusion Criteria
• Pregnant or Breast-Feeding Pregnant or breast-feeding women will not be entered on this study due to known potential risks of fetal and teratogenic adverse events as seen in animal/human studies. Pregnancy tests must be obtained in girls who are post-menarchal. Patients of childbearing or child fathering potential must agree to use at least one highly effective method of contraception while being treated on this study and for 3 months after completing therapy. A woman is considered of childbearing potential if she is fertile, following menarche and until becoming post-menopausal unless permanently sterile. A postmenopausal state is defined as no menses for 12 months without an alternative medical cause. A high follicle stimulating hormone (FSH) level in the postmenopausal range may be used to confirm a post-menopausal state in women not using hormonal contraception or hormonal replacement therapy. However, in the absence of 12 months of amenorrhea, a single FSH measurement is insufficient. A man is considered fertile after puberty unless permanently sterile by bilateral orchidectomy. Male participants should refrain from sperm donation throughout the duration of treatment and for 3 months after completion of therapy A highly effective contraception method is defined as one that results in a low failure rate (\<1% per year) when used consistently and correctly. The following are considered highly effective contraception methods: * Combined estrogen and progesterone containing hormonal contraception associated with inhibition of ovulation. * Progesterone-only hormonal contraception associated with inhibition of ovulation. * Intra Uterine Device (IUD) * Intra Uterine hormone releasing system * Bilateral tubal occlusion * Vasectomized partner * Sexual abstinence (avoiding having heterosexual intercourse) The following contraceptive measures are NOT considered effective * Progesterone-only hormonal contraception (birth control pill) that that does NOT stop ovulation * Male or female condom with or without spermicide * Cap, diaphragm or sponge with spermicide
• Concomitant Medications * Patients receiving corticosteroids are eligible. The use of corticosteroids must be reported. * Patients who are currently receiving another investigational drug are not eligible. * Patients who are currently receiving other anti-cancer agents are not eligible, with the exception of temozolomide given concurrently with RT only. * Patients who are receiving enzyme inducing anticonvulsants that are strong inducers or inhibitors of CYP3A4/5 are not eligible. * Patients who are receiving strong inducers or inhibitors of CYP3A4/5 are not eligible and should be avoided from 14 days prior to enrollment to the end of the study. * Patients who are receiving medications known to prolong QTc interval are not eligible. * Patients who are receiving therapeutic anticoagulation with warfarin or other coumadin-derived anticoagulants are not eligible. Therapy with heparin, low molecular weight heparin (LMWH), or fondaparinux is allowed as long as the patient has adequate coagulation defined as aPTT \< 1.5Xs ULN and INR \< 1.5.
• Patients who have an uncontrolled infection are not eligible.
• Patients who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study are not eligible.
• Patients with known clinically significant active malabsorption syndrome or other condition that could affect absorption are not eligible.
• Patients with prior or ongoing clinically significant medical or psychiatric condition that, in the investigator's opinion, could affect the safety of the subject, or could impair the assessment of study results are not eligible.

Interventions: DRUG: Ribociclib, DRUG: Everolimus, DRUG: Temozolomide (TMZ)

Conditions: High Grade Glioma, Diffuse Intrinsic Pontine Glioma, Anaplastic Astrocytoma, Glioblastoma, Glioblastoma Multiforme, Diffuse Midline Glioma, H3 K27M-Mutant, Metastatic Brain Tumor, WHO Grade III Glioma, WHO Grade IV Glioma, Diffuse Hemispheric Glioma, H3 G34-Mutant

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CAR-T Long Term Follow Up (LTFU) Study (PAVO)

Description: Per Health Authorities guidelines for gene therapy medicinal products that utilize integrating vectors (e.g. lentiviral vectors), long term safety and efficacy follow up of treated patients is required. The purpose of this study is to monitor all patients exposed to CAR-T therapied for 15 years following their last CAR-T (e.g. CTL019) infusion to assess the risk of delayed adverse events (AEs), monitor for replication competent lentivirus (RCL) and assess long-term efficacy, including vector persistence.

Contact(s):

Stephanie Edwards - StephanieL.Edwards@cchmc.org

Sex: ALL

Age: 0 years to 100 years old

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT02445222

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Interventions: GENETIC: Previously treated CAR-T patients

Conditions: Long Term Safety of Patients Receiving CAR-T in an Eligible Clinical Trial or Managed Access Program

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Pediatric Dose Optimization for Seizures in Emergency Medical Services (PediDOSE)

Description: The Pediatric Dose Optimization for Seizures in Emergency Medical Services (PediDOSE) study is designed to improve how paramedics treat seizures in children on ambulances. Seizures are one of the most common reasons why people call an ambulance for a child, and paramedics typically administer midazolam to stop the seizure. One-third of children with active seizures on ambulances arrive at emergency departments still seizing. Prior research suggests that seizures on ambulances continue due to under-dosing and delayed delivery of medication. Under-dosing happens when calculation errors occur, and delayed medication delivery occurs due to the time required for dose calculation and placement of an intravenous line to give the medication. Seizures stop quickly when standardized medication doses are given as a muscular injection or a nasal spray. This research has primarily been done in adults, and evidence is needed to determine if this is effective and safe in children. PediDOSE optimizes how paramedics choose the midazolam dose by eliminating calculations and making the dose age-based. This study involves changing the seizure treatment protocols for ambulance services in 20 different cities, in a staggered and randomly-assigned manner. One aim of PediDOSE is to determine if using age to select one of four standardized doses of midazolam and giving it as a muscular injection or nasal spray is more effective than the current calculation-based method, as measured by the number of children arriving at emergency departments still seizing. The investigators believe that a standardized seizure protocol with age-based doses is more effective than current practice. Another aim of PediDOSE is to determine if a standardized seizure protocol with age-based doses is just as safe as current practice, since either ongoing seizures or receiving too much midazolam can interfere with breathing. The investigators believe that a standardized seizure protocol with age-based doses is just as safe as current practice, since the seizures may stop faster and these doses are safely used in children in other healthcare settings. If this study demonstrates that standardized, age-based midazolam dosing is equally safe and more effective in comparison to current practice, the potential impact of this study is a shift in the treatment of pediatric seizures that can be easily implemented in ambulance services across the United States and in other parts of the world.

Contact(s):

Lauren Riney - lauren.riney@cchmc.org

Sex: ALL

Age: 6 months to 13 years old

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05121324

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Interventions: DRUG: Standardized seizure protocol, DRUG: Conventional seizure protocol

Conditions: Seizures

Keywords: Child, Emergency Medical Services

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A Prospective Analysis of Long-Term Clinical Outcomes and 3D Spine Growth in Anterior Vertebral Body Tethering

Description: Anterior vertebral body tethering (AVBT) is a novel, minimally invasive, growth modulation technique that was recently approved by the FDA under a Humanitarian Device Exemption (HDE). The goal of AVBT is to control curve progression by applying compression on the convex side of the spine deformity. While there has been great initial enthusiasm about the technique as an alternate treatment option to spinal fusion for skeletally immature children with scoliosis, there is a need to better understand the long-term outcomes. The purpose of this study is to report the long-term clinical outcomes of skeletally immature patients treated with AVBT, specifically: 1. The effect on three-dimensional spine growth as compared to normal controls 2. Maintenance of major Cobb angle less than or equal to 50 degrees at skeletal maturity 3. Complications associated with both the procedure and the device

Contact(s):

Nichole Leitsinger - nichole.leitsinger@cchmc.org

Sex: ALL

Age: Up to 18 years old

Phase: NA

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT04914507

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Interventions: DEVICE: Anterior Vertebral Body Tethering

Conditions: Scoliosis Idiopathic

Keywords: anterior vertebral body tethering

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