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Active Surveillance, Bleomycin, Etoposide, Carboplatin or Cisplatin in Treating Pediatric and Adult Patients With Germ Cell Tumors
- cancer@cchmc.org
ALL
PHASE3
NCT03067181
Inclusion Criteria:
* There is no age limit for the low risk stratum (stage I ovarian immature teratoma and stage I non-seminoma or seminoma malignant GCT \[all sites\])
* Standard risk 1: Patients must be \< 11 years of age at enrollment
* Standard risk 2: Patients must be \>= 11 and \< 25 years of age at enrollment
* Patients enrolling on one of the low risk arms must be newly diagnosed with a stage I germ cell tumor; for the standard risk arms, patients must be newly diagnosed with malignant germ cell tumor (stage II or higher).
* Histologic confirmation of a primary extracranial germ cell tumor in any of the categories outlined below is required of all patients at enrollment , with the following exceptions:
* Among patients were initially diagnosed with completely resected non-seminoma malignant GCT and later recur during observation post surgery, a diagnostic biopsy is not required for enrollment if elevated tumor markers rise to \> 5 x upper limit of normal (ULN) on at least 2 measurements taken at least 1 week apart. The pathology report of initial surgery should be provided
* Patients may be enrolled without histologic or cytologic confirmation in the rare case where there are exceptionally raised tumor markers (alpha fetoprotein \[AFP- ≥ 500 ng/mL or HCG ≥ 500 IU/L) and radiologic features consistent with GCT. In addition, the treating clinician must deem that the patient's tumor is not suitable for upfront resection and that a biopsy is not in the patient's best interest; or that there is a need to start therapy urgently
* Low risk immature teratoma (IT); site: ovarian; stage: any; grade: any; histology: pure immature teratoma, mixed immature and mature teratoma, (may contain microscopic foci of yolk sac tumor \[\< 3 mm\], but no other pathological evidence of MGCT); tumor markers: alpha-FP =\< 1,000 ng/mL, beta-HCG institutional normal; all ages
* Low risk stage I non-seminoma MGCT; site: ovarian, testicular, or extragonadal; stage: COG stage I, FIGO stage IA and IB, American Joint Committee on Cancer (AJCC) testicular stage IA, IB and IS; histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma (pure or mixed); all ages
* Low risk stage I seminoma-MGCT; site: testicular; stage: COG stage I; AJCC testicular stage IA IB, and IS; histology: must contain only seminoma; may contain immature/mature teratoma; may NOT contain yolk sac tumor, embryonal carcinoma, or choriocarcinoma; all ages
* Standard risk 1 (SR1); site: ovarian, testicular, or extragonadal; stage: COG stage II-IV, FIGO stage IC-IV, (International Germ Cell Consensus Classification \[IGCCC\] criteria DO NOT apply); histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma; age (years) \< 11
* Standard risk 2 (SR2)
* Site: ovarian; stage: COG stage II, III, and III-X, FIGO stage IC, II and III; histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma; age (years) \>= 11 and \< 25
* Site: testicular; stage: COG stage II-IV, AJCC stage II, III, IGCCC good risk; histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma: must be IGCCC good risk; post op: alpha-FP \< 1,000 ng/mL, beta-HCG \< 5,000 IU/mL and lactate dehydrogenase (LDH) \< 3.0 x normal; age (years) \>= 11 and \< 25
* Notes:
* IGCCC criteria only apply to SR2 patients with a testicular primary tumor
* Use post-op tumor marker levels to determine IGCCC risk group
* Pure seminoma patients are not eligible for the standard risk arms of the study
* For the low risk stage I non-seminoma MGCT and the standard risk arms, components of yolk sac tumor, embryonal carcinoma, or choriocarcinoma can be mixed with other forms of GCT, such as seminoma or mature or immature teratoma; if yolk sac tumor is the only malignant component present, then it must be deemed by the pathologist to be greater than a "microscopic component" of yolk sac tumor
* Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1, 2 or 3; use Karnofsky for patients \> 16 years of age and Lansky for patients =\< 16 years of age
* Organ function requirements apply ONLY to patients who will receive chemotherapy (SR1 and SR2 patients)
* Adequate renal function defined as:
* Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 (within 7 days prior to enrollment) OR
* A serum creatinine based on age/sex as follows (within 7 days prior to enrollment): (mg/dL)
* 1 month to \< 6 months male: 0.4 female: 0.4
* 6 months to \< 1 year male: 0.5 female: 0.5
* 1 to \< 2 years male: 0.6 female: 0.6
* 2 to \< 6 years male: 0.8 female: 0.8
* 6 to \< 10 years male: 1 female: 1
* 10 to \< 13 years male: 1.2 female: 1.2
* 13 to \< 16 years: male: 1.5 female: 1.4
* \>= 16 years male: 1.7 female: 1.4
* Total bilirubin =\< 2 x upper limit of normal (ULN) for age (within 7 days prior to enrollment)
* Unless due to Gilbert's disease, malignant involvement of liver or vanishing bile duct syndrome
* Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \< 3 x upper limit of normal (ULN) (within 7 days prior to enrollment)
* Unless due to Gilbert's disease, malignant involvement of liver or vanishing bile duct syndrome
* Peripheral absolute neutrophil count (ANC) \>= 750/mm\^3 (within 7 days prior to enrollment) AND
* Platelet count \>= 75,000/mm\^3 (within 7 days prior to enrollment)
* Patients enrolling on the standard risk arms must be medically fit to receive protocol treatment and with no contraindications to protocol treatment
* Eligibility criteria to participate in the pilot study of the AYA-Hears instrument (patient reported outcomes \[PROs\] of ototoxicity) Note: participants in group 1 will not receive AGCT1531 protocol-directed therapy; all other AYA-HEARS patients must be enrolled on the AGCT1531 SR2 arm in order to participate
* \>= 11 and \< 25 years old at enrollment
* Able to fluently speak and read English
* Has received prior cisplatin- or carboplatin-based chemotherapy regimen for malignancy including diagnoses other than germ cell tumor
* Followed for cancer or survivorship care at one of the following institutions:
* Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
* Dana Farber/Harvard Cancer Center
* Hospital for Sick Children
* Children's Hospital of Eastern Ontario
* Oregon Health and Science University
* Seattle Children's Hospital
* Yale University
Exclusion Criteria:
* Patients with any diagnoses not listed including:
* Stage I testicular cancer patients who have undergone primary RPLND (retroperitoneal lymph node dissection)
* Pure ovarian or extragonadal dysgerminoma/seminoma
* Pure mature teratoma
* Pure immature teratoma with alpha-fetoprotein (AFP) \>= 1000 ng/mL
* "Poor risk" GCT (age \>= 11 years old and COG stage IV ovarian, COG stage II- IV extragonadal, or IGCCC intermediate or poor risk testicular), or
* Primary central nervous system (CNS) germ cell tumor
* Germ cell tumor with somatic malignant transformation
* Spermatocytic seminoma
* Patients must have had no prior systemic therapy for the current cancer diagnosis
* Patients must have had no prior radiation therapy with the exception of CNS irradiation of brain metastases; (this exception only applies to SR1 patients; any patients over age 11 with distant metastases to brain \[stage IV disease\] would be considered poor risk and therefore not eligible for this trial)
* Patients with significant, pre-existing co-morbid respiratory disease that contraindicate the use of bleomycin are ineligible for the standard risk arms of the trial
* 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; (this criteria applies ONLY to patients who will receive chemotherapy \[SR1 and SR2 patients\])
* Lactating females who plan to breastfeed their infants; (this criteria applies ONLY to patients who will receive chemotherapy \[SR1 and SR2 patients\])
* Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation; (this criteria applies ONLY to patients who will receive chemotherapy \[SR1 and SR2 patients\]) OTHER: Best Practice, PROCEDURE: Biopsy Procedure, PROCEDURE: Biospecimen Collection, BIOLOGICAL: Bleomycin Sulfate, DRUG: Carboplatin, DRUG: Cisplatin, PROCEDURE: Computed Tomography, DRUG: Etoposide, PROCEDURE: Magnetic Resonance Imaging, PROCEDURE: Pulmonary Function Test, OTHER: Questionnaire Administration
Childhood Extracranial Germ Cell Tumor, Extragonadal Embryonal Carcinoma, Germ Cell Tumor, Malignant Germ Cell Tumor, Malignant Ovarian Teratoma, Stage I Ovarian Choriocarcinoma, Stage I Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage I Ovarian Yolk Sac Tumor AJCC v6 and v7, Stage I Testicular Choriocarcinoma AJCC v6 and v7, Stage I Testicular Embryonal Carcinoma AJCC v6 and v7, Stage I Testicular Seminoma AJCC v6 and v7, Stage I Testicular Yolk Sac Tumor AJCC v6 and v7, Stage II Ovarian Choriocarcinoma, Stage II Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage II Ovarian Yolk Sac Tumor AJCC v6 and v7, Stage II Testicular Choriocarcinoma AJCC v6 and v7, Stage II Testicular Embryonal Carcinoma AJCC v6 and v7, Stage II Testicular Yolk Sac Tumor AJCC v6 and v7, Stage III Ovarian Choriocarcinoma, Stage III Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage III Ovarian Yolk Sac Tumor AJCC v6 and v7, Stage III Testicular Choriocarcinoma AJCC v6 and v7, Stage III Testicular Embryonal Carcinoma AJCC v6 and v7, Stage III Testicular Yolk Sac Tumor AJCC v6 and v7, Stage IV Ovarian Choriocarcinoma, Stage IV Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage IV Ovarian Yolk Sac Tumor AJCC v6 and v7, Testicular Mixed Choriocarcinoma and Embryonal Carcinoma, Testicular Mixed Choriocarcinoma and Teratoma, Testicular Mixed Choriocarcinoma and Yolk Sac Tumor
Proton and Photon Consortium Registry (PPCR): A Multi Center Registry of Pediatric Patients Treated With Radiation Therapy
- cancer@cchmc.org
ALL
Up to 21 years old
NCT01696721
Eligibility Criteria:
• Patients treated with radiation therapy at one of the participating centers
• Age \< 22 at time of treatment start.
• Patients may be enrolled regardless of previous local or systemic treatments received prior to enrollment in the PPCR.
• Patients may be enrolled regardless of other current local or systemic treatments or disease extent.
• Patients may be enrolled on the PPCR concurrently with another study or clinical trial.
• Patients treated with radiation therapy at one of the participating centers
• Age \< 22 at time of treatment start.
• Patients may be enrolled regardless of previous local or systemic treatments received prior to enrollment in the PPCR.
• Patients may be enrolled regardless of other current local or systemic treatments or disease extent.
• Patients may be enrolled on the PPCR concurrently with another study or clinical trial.
OTHER: No intervention
Pediatric Patients Treated With Radiation Therapy
Proton Therapy, Pediatrics, Radiation Oncology, Photon Therapy
Neonatal Platelet Transfusion Threshold Trial (NeoPlaTT)
Traci Beiersdorfer - traci.beiersdorfer@cchmc.org
ALL
1 hour to 48 hours old
NA
NCT06676904
Inclusion Criteria:
* Gestational age of 23 0/7 to 26 6/7 weeks
* Postnatal age of \< 48 hours
Exclusion Criteria:
* Comfort care or withdrawal of care planned
* Neonatal alloimmune thrombocytopenia or suspected/confirmed congenital platelet or bleeding disorder
* Receipt of platelet transfusion
* No receipt of Vitamin K
* Parents/guardian decline consent PROCEDURE: Higher Platelet Transfusion Threshold, PROCEDURE: Lower Platelet Transfusion Threshold
Thrombocytopenia, Neonatal, Platelet Transfusion, Infant, Newborn, Diseases, Infant, Extremely Low Birth Weight, Infant, Small for Gestational Age, Thrombosis
platelet transfusion, neonatal, thrombosis
Chicago Parent Program for Foster and Kinship Caregivers
Laura Fossett - laura.fossett@cchmc.org
ALL
2 years and over
NA
NCT06170047
Inclusion Criteria:
* Must be a licensed foster caregiver or kinship caregiver to a foster child between the ages of 2 and less than 9 years of age
* Must be a licensed foster caregiver or kinship caregiver to a foster child in Ohio and in the custody of Hamilton County Job and Family Services, Butler County Children Services, or Montgomery County Children Services
* Must be in good standing with the foster care agency
* Must be English-speaking
Exclusion Criteria:
* Not having a foster child between the ages of 2 and less than 9 years
* The foster child not being in the custody of Ohio counties: Hamilton County Job and Family Services, Butler County Children Services, or Montgomery County Children Services
* The foster child was placed in the home more than 45 days prior to enrollment
* The foster child being moved out of the placement prior to the start of the intervention
* The foster child having been previously enrolled with another caregiver
* The caregiver having been previously enrolled with another child
* The caregiver unable to commit to completing all study activities
* The foster child is not enrolled in the study BEHAVIORAL: Chicago Parent Program for Foster Care, OTHER: Usual Care
Behavior Problem, Parenting
Foster care, Child behavior, Parenting skills, Behavioral health, Prevention, Parenting confidence, Parenting stress
Shwachman Diamond Syndrome Registry and Study (SDSR)
Caitlin Cottrell - caitlin.cottrell@cchmc.org
ALL
NCT06056908
Inclusion Criteria:
Shwachman Diamond syndrome, Shwachman-Diamond Syndrome-Like conditions, or a genetically undefined condition that shares clinical features with Shwachman Diamond Syndrome.
* Biallelic mutations in SBDS, or pathogenic mutations in DNAJC21, EFL1, or SRP54 OR
* Shwachman-Diamond Syndrome defined clinically OR
* Clinically suspected Shwachman-Diamond Syndrome OR
* Phenotypic features suggestive of SDS OR
* Parents, siblings, and other blood relatives of any age, living and deceased, of patients with SDS or SDS-Like conditions are eligible for this study
Exclusion Criteria:
• Patients with other diagnosed causes of bone marrow failure, exocrine pancreatic insufficiency and cancer predisposition will be excluded. Shwachman-Diamond Syndrome, Shwachman-Diamond Syndrome-Like
Bone marrow failure, Inherited bone marrow failure syndromes, Bone marrow, Neutropenia, Leukemia, MDS, Pancreas, Cancer predisposition
Multi-Center Molecular Diagnosis and Host Response of Respiratory Viral Infections in Pediatric Transplant Recipients
Lara Danziger-Isakov, MD - Lara.Danziger-Isakov@cchmc.org
ALL
Up to 18 years old
NCT05550298
Recipient Inclusion Criteria
* Less than 18 years at the time of anticipated transplant
* Participant meets one of the following criteria:
• scheduled to receive allogeneic hematopoietic cell transplant within 14 days of enrollment or
• Scheduled to or received solid organ transplant within 7 days before or after enrollment * Participant is receiving care at the time of enrollment at one of the study participating institutions. * Parent/guardian willing and able to provide informed consent, and if appropriate, child willing and able to provide informed assent. Donor Inclusion Criteria * Donor for HCT recipient enrolled on the VIPER study. * Willing and able to provide informed consent.
• scheduled to receive allogeneic hematopoietic cell transplant within 14 days of enrollment or
• Scheduled to or received solid organ transplant within 7 days before or after enrollment * Participant is receiving care at the time of enrollment at one of the study participating institutions. * Parent/guardian willing and able to provide informed consent, and if appropriate, child willing and able to provide informed assent. Donor Inclusion Criteria * Donor for HCT recipient enrolled on the VIPER study. * Willing and able to provide informed consent.
Exclusion Criteria:
Recipient Exclusion Criteria
None
Donor Exclusion Criteria
* Is not an HCT donor for a participant enrolled on the VIPER study.
* Not available to provide pre-transplant research blood sample. Hematopoietic Cell Transplant, Solid Organ Transplant, Respiratory Viral Infection
Down Syndrome Obstructive Sleep Apnea (DOSA)
Suzanna Hicks - suzanna.hicks@cchmc.org
ALL
5 years to 18 years old
PHASE2
NCT06043440
Inclusion Criteria:
• Ages 5.0 to 17.9 years at the time of screening
• Children with OSA and obstructive apnea hypopnea index (OAHI) ≥5/hour.
• Absence of clinically significant hypoxia defined as oxygen saturation \<88% for 5 minutes or episodic desaturation to 60% as these levels would otherwise identify children eligible to routinely receive oxygen.
• Favorable response to oxygen therapy (allowing randomization) will be defined as follows:
• Oxygen saturation nadir \>92% and
• Decrease in obstructive index \< 5 / hour or by \> 50% from screening PSG
• Reaching an optimum oxygen flow which is defined as the flow that achieves the lowest level of AHI with maximum CO2 level less than 65 mmHg observed for 5 consecutive minutes and or an increase in CO2 by less than 15 points above baseline. The above criteria are observed while the patient spends a minimal of 30 minutes in the supine position and at least one cycle of rapid eye movement (REM) sleep.
• Oxygen flow required does not exceed 3.0 LPM and does not exceed a FiO2 \> 40 %.
• Willingness to comply with all study procedures and available for duration of study.
• At baseline the participant attempts to perform the neuropsychological tests
Exclusion Criteria:
• Current CPAP use with documented compliance(\> 4 hrs/ night; \> 70% of nights).
• Oxygen saturation \< 90% at rest during wakefulness.
• Chronic daytime or nighttime use of supplemental oxygen.
• Smoker in the child's bedroom.
• Unrepaired congenital heart disease.
• Moderate to severe pulmonary hypertension requiring treatment with oxygen and or pulmonary vasodilator.
• Unable to participate in a PSG.
• Individuals who develop alveolar hypoventilation with oxygen as previously defined.
• Other severe chronic diseases determined by their provider as making them poor study candidates.
• Enrolled or planning to enroll in another study that may conflict with protocol requirements or confound results in this trial.
• Documented clinically significant untreated hypothyroidism
• Children with adenotonsillar hypertrophy who are candidates for adenotonsillectomy and parents agree to the surgery.
DRUG: Oxygen
Down Syndrome, Obstructive Sleep Apnea
Testing Drug Efficacy in Cystic Fibrosis Through N-of-1 Trials (Nof1)
Sharon Kadon - sharon.kadon@cchmc.org
ALL
6 years and over
NA
NCT04580368
Inclusion Criteria:
* Signed informed consent (and assent when applicable)
* Willing and able to adhere to the study visit schedule and protocol requirements
* Male or Female ≥6 years old and within the FDA-approved range for the proposed modulator drug
* Ivacaftor: ≥4 months old
* Lumacaftor/Ivacaftor: 2 years old
* Tezacaftor/Ivacaftor: 12 years old
* Elexacaftor/Tezacaftor/Ivacaftor: ≥12 years old
* At least one rare CFTR variant (incidence of \<5% of the CF population)
* Documentation of a CF diagnosis as evidenced by one or more clinical features of CF plus at least one of the following:
* Sweat Chloride ≥60mmol/L by quantitative pilocarpine iontophoresis
* Two mutations in the CFTR gene
* Abnormal nasal potential difference (NPD) testing supportive of a CF diagnosis
* FEV1 \> 50% predicted for age
* Stable chronic CF therapies with no changes in \>28 days (except for chronic cycled inhaled antibiotics such as tobramycin)
* Prescribed CFTR modulator by a licensed physician
* No contraindication to treatment with the selected drug at the time of treatment initiation
Exclusion Criteria:
* Presence of any condition or abnormality that, in the opinion of the Investigator, would compromise the safety of the patient and/or quality of the data
* For women of child bearing potential:
* Positive pregnancy test or known pregnancy at Visit 1
* Lactating
* Unwilling to practice a medically acceptable form of contraception (acceptable forms include abstinence, hormonal birth control, intrauterine device, or barrier method plus a spermicidal agent), unless surgically sterilized or postmenopausal during the study
* BMI \< 10th percentile for age (if \<18 years old) or \< 20kg/m2 (if ≥18 years old)
* FEV1 ≤ 50% predicted for age
* Growth of CF pathogens from sputum cultures that are associated with unstable disease (e.g., nontuberculous mycobacteria, Burkholderia spp) within six months of enrollment
* Concomitant use of CYP3A inducers or inhibitors (e.g., voriconazole, fluconazole, rifampin) or prednisone (\>20mg daily)
* Concomitant conditions:
* Poorly controlled diabetes mellitus (HbA1c \>8.5 or glucosuria as noted below)
* Advanced CF liver disease (cirrhosis with portal hypertension, ascites, or abnormal liver laboratory testing as noted below)
* End stage renal disease
* History of organ transplantation
* Additional medical conditions that in the opinion of the Investigator place the patient at risk of participation or may impact the patient's ability to complete the trial (e.g., uncontrolled depression, anxiety disorder, poor adherence to CF therapies, active ABPA)
* Any of the following abnormal laboratory values at the Screening Visit:
* CBC
* WBC \>15,000 K/mcL or ANC \<1,500 K/mcL
* Hemoglobin \<10 gm/dL
* Platelets \<50,000 K/mcL
* Chemistries
* \>2+ Glucosuria
* Clinically significant abnormalities as assessed by the Investigator
* Glomerular filtration rate ≤50 mL/min/1.73 m2 (calculated by the Counahan-Barratt equation)
* Hepatic Function Testing / Coagulation Testing
* ≥3 × upper limit of normal (ULN) aspartate aminotransferase (AST)
* ≥3 × ULN alanine aminotransferase (ALT)
* ≥3 × ULN gamma-glutamyl transpeptidase
* Total or direct bilirubin \>2 × ULN
* INR \> 1.5 x ULN
* Positive pregnancy test DRUG: CFTR Modulators
Cystic Fibrosis
CFTR modulators, cystic fibrosis, HNE, human nasal epithelial cells, NPD, nasal potential difference, air-liquid interface, N-of-1
Dead Space and Inhaled Nitric Oxide in Pediatric Acute Respiratory Distress Syndrome (DiNO)
Abigayle Gibson - Abigayle.Gibson@cchmc.org
ALL
0 years to 21 years old
NCT06690801
Inclusion Criteria:
* Age \>37 weeks corrected gestational age to 21 years, including adults lacking the capacity to consent.
* Within 72 hours of the start of invasive mechanical ventilation and meet the criteria for pediatric ARDS (new infiltrate on chest imaging and a known ARDS risk factor within 7 days of the onset of hypoxemia) and either meet criteria for moderate or severe pediatric ARDS between 4-72 hours of IMV (OI ≥ 8 or OSI ≥ 7.5) OR have an OI ≥ 20 or an OSI ≥ 14 x 15 minutes between 0-4 hours of IMV.
* Subgroup of children eligible for longituduinal Blood Collection: Children with severe PARDS (OI ≥ 16 or an OSI ≥ 12 between 4-72 hours of IMV) or those with an OI ≥ 20 or an OSI ≥ 14 for 15 minutes between 0-4 hours of IMV will be eligible for collection of longitudinal plasma samples.
Exclusion Criteria:
* Non-conventional invasive mechanical ventilation (i.e. High Frequency Oscillatory Ventilation, Airway Pressure Release Ventilation) at the time of ICU admission
* ECMO or iNO (or other inhaled pulmonary vasodilator therapy) at the time of ICU admission
* Significant lower airways obstruction (examination of ventilator and capnography waveforms by site study or medical team)
* Air leak \>20% (endotracheal tube, tracheostomy tube, or thoracostomy tube)
* Home Invasive Mechanical Ventilation
* Cyanotic Congenital Heart Disease
* Previous enrollment in the DiNO study
* Do not resuscitate order at the time of pediatric ARDS diagnosis.
* Blood gas not obtained prior to initiation of ECMO, iNO, or non-conventional ventilation. Acute Respiratory Distress Syndrome
Pediatrics, Mechanical Ventilation, Inhaled Nitric Oxide
Primary Sclerosing Cholangitis in Children
Erin Chapman - erin.chapman@cchmc.org
ALL
2 years to 25 years old
NCT04181138
Inclusion Criteria:
Patients with the clinical diagnosis of large or small duct PSC made at any time prior to enrollment are screened for eligibility to participate in this prospective cohort study. The site PI will determine eligibility following review of MRCP or ERCP images with the site radiologist to confirm presence of an abnormal cholangiogram at the time of diagnosis of large duct PSC. Liver histopathology obtained at the time of diagnosis of small duct PSC will be reviewed with the site pathologist prior to enrollment.
Individuals must meet all of the Inclusion criteria in order to be eligible to participate in the study:
• Aged 2 through 25 years at time of screening.
• Diagnosis of large duct PSC based on review of cholangiogram by MRC, ERC, or intraoperative cholangiogram (IOC) by the site radiologist and interpreted to be consistent with PSC, based on one or more of the following: * Focal structuring of the bile duct(s) * Dominant stricture of the common bile duct * Saccular dilatation of bile duct(s) * Beaded appearance of bile duct(s) * Pruning appearance of the distal bile duct branches AND/OR
• Diagnosis of small duct PSC based on review of liver histopathology by the site pathologist and interpreted to be compatible with PSC: * Probable small duct PSC: biopsy with ≥3 of 5 criteria: periductal edema, concentric inflammation, bile duct injury, ductular reaction, and neutrophils in bile ducts (cholangitis) OR... * Definitive small duct PSC: Periductal fibrosis/ "onion skinning" around interlobular bile ducts or smaller profiles
• Stated willingness to comply with all study procedures and availability for the duration of the study.
• Able to provide informed consent/assent Participants for the imaging study are eligible if they are:
• Aged 8 through 25 years at the time of screening
• No absolute contraindication to MRI
• No skin condition that could be aggravated by MREL
• Meet all other eligibility criteria of the PSC Observational Study
• For whom none of the exclusion criteria apply
Exclusion Criteria:
An individual who meets any of the following criteria at baseline will be excluded from participation in this study.
• History of liver transplantation
• History bone marrow transplantation
• History of primary or acquired immunodeficiency predisposing to secondary sclerosing cholangitis, for instance: hyper-IgM syndrome, severe combined immunodeficiency (SCID) syndrome, common variable immunodeficiency (CVID) syndrome, cartilage hair hypoplasia syndrome, or HIV/AIDS
• History of histiocytosis, including Langerhans cell histiocytosis (LCH), or hemophagocytic lymphohistiocytosis (HLH)
• History of ischemic cholangitis
• History of portal vein thrombosis with biliopathy, veno-occlusive disease, or abdominal radiation vasculopathy
• History of recurrent pyogenic cholangitis
• History of biliary tract surgery for cholecystolithiasis prior to cholangiogram/liver biopsy evaluated to determine enrollment
• History of biliary tract surgery for choledochal cyst
• History of hepatocellular carcinoma, or hepatoblastoma
• History of surgical biliary trauma
• History of congenital cytomegalovirus (CMV) hepatitis
• History of Sickle Cell Disease
• History of cystic fibrosis, biliary atresia, Caroli disease/congenital hepatic fibrosis, or progressive familial intrahepatic cholestasis type 3/MDR3 disease
• History of cardiac hepatopathy.
• History of metabolic disorders, including Wilson's disease, glycogen storage disorder, Alpha-1 Antitrypsin deficiency
• Diagnosis of systemic lupus erythematosus (SLE)
• Concurrent pregnancy at the time of enrollment -
Primary Sclerosing Cholangitis, Liver Diseases, Cholangitis, Sclerosing
Pediatric Liver Disease, Primary Sclerosing Cholangitis
A Phase 3 Study of Revaccination in Subsequent Pregnancies With Bivalent RSV Vaccine and Duration of Protection of a Single Dose
Benjamin Kercsmar - Benjamin.Kercsmar@cchmc.org
ALL
Not specified
PHASE3
NCT06866405
Pregnant Participants-Cohort 1 and Cohort 2 Key Inclusion Criteria
* Women aged 18 to 49 who are pregnant, between 24 and 36 weeks along, and expecting one baby without known risks for complications can participate.
* Had the RSVpreF or Abrysvo vaccine during a previous pregnancy.
* Had an ultrasound scan at 18 weeks or later during their current pregnancy, with no major fetal problems detected.
* Based on their medical history, physical check-up, and the doctor's judgment, they are found suitable to join the study.
* Agrees to let their baby take part in the study and gives their permission.
* Able to sign a consent form, agreeing to follow the rules and conditions of the study.
Key Exclusion Criteria
* Received any approved or experimental RSV vaccine since their previous pregnancy.
* Has a pre-pregnancy body mass index (BMI) over 40 kg/m2.
* History of a severe bad reaction to a vaccine or a serious allergic reaction (like anaphylaxis) to any ingredient in the study vaccine or a similar vaccine.
* Current pregnancy problems or issues at the time of giving consent.
* Previous pregnancy issues or problems at the time of giving consent.
* Women who are breastfeeding at the time of enrollment
Infant Participants
* Proof that the parent(s) or legal guardian(s) has signed and dated a consent form.
* Parent(s) or legal guardian(s) must agree to attend scheduled visits and follow the study plan, including laboratory tests and other procedures.
Nonpregnant Participants-Cohort 3 Key Inclusion Criteria
* Have already received one dose of the RSVpreF vaccine during their previous pregnancy as part of the Pfizer clinical trial, and the results from that time can be used for this study.
* Able to sign a consent form, agreeing to follow the rules and requirements of the study.
Key Exclusion Criteria
* Received any approved or experimental RSV vaccine after participating in the Pfizer clinical trial.
* Taking part in other studies with new drugs within 28 days before giving consent or during the study period.
BIOLOGICAL: RSVpreF, BIOLOGICAL: Placebo
RSV Infection
Pregnancy, RSV vaccine, RSV, Maternal immunization
Impact of Soymilk on Liver Disease Severity of Children With Non-alcoholic Fatty Liver Disease (NAFLD)
Ann Popelar - Ann.Popelar@cchmc.org
ALL
5 years to 12 years old
PHASE2
NCT06133101
Inclusion Criteria:
* Children with overweight/obesity
* Non-alcoholic fatty liver disease (NAFLD) and an MRI PDFF \>10%
* Known NAFLD or elevated ALT for sex (\>22 for females and \>26 for males)
Exclusion Criteria:
* MRI-PDFF \<10%
* Baseline habitual (\>3 days per week) consumption of soy foods
* Allergy to soy or cow's milk protein
* Inability to undergo MRI
* Recent (past 8 weeks) antibiotic exposure
* Treatment for existing endocrine disorders DRUG: Standard Soy Milk, DRUG: 2% Fat Cow's Milk
Non-Alcoholic Fatty Liver Disease
NAFLD
MRI Biomarkers in as Predictor of Clinical Endpoints in Pediatric Autoimmune Liver Disease
Alexander Miethke, MD - alexander.miethke@cchmc.org
ALL
6 years to 23 years old
NCT03178630
Inclusion Criteria:
• Age 6-23 years old.
• Established clinical diagnosis of AIH or PSC.
Exclusion Criteria:
• History of liver transplantation.
• Chronic Hepatitis B or untreated hepatitis C virus infection.
• Pregnancy.
• Absolute contraindication for MRI (e.g. pacemaker, metallic implants, claustrophobia).
• Diagnosis of cystic fibrosis or biliary atresia
• Diagnosis of cardiac hepatopathy.
• Diagnosis of Wilson's disease, Alpha-1 Antitrypsin deficiency, or Glycogen storage disease.
• Skin conditions which could be aggravated by MREL (i.e. Epidermolysis bullosa).
Autoimmune Liver Disease, Autoimmune Hepatitis, Primary Sclerosing Cholangitis
Impact of Short and Mistimed Sleep on Adolescents With ADHD: The Adolescent Attention and Circadian Timing Study (AACT)
- teensleep@cchmc.org
ALL
13 years to 17 years old
NA
NCT07684417
Inclusion Criteria:
* Ages 13-17 years at time of informed consent/assent
* Based on semi-structured clinical interview, participants must meet full DSM-5 criteria for ADHD inattentive or ADHD combined presentation.
Exclusion Criteria:
* Non-traditional school setting (morning-afternoon Monday-Friday).
* Exclusionary diagnoses. We will exclude adolescents with known intellectual disability, autism spectrum disorder, psychosis, bipolar disorder, or neurologic conditions (e.g., epilepsy), per caregiver-report.
* Exclusionary sleep disorders. We will exclude adolescents with symptoms of obstructive sleep apnea or periodic limb movement disorder based on published cutoffs on a validated questionnaire.
* High caffeine intake. To promote adherence to directives not to consume caffeine the day of office visits without withdrawal effects, adolescents with daily intake of \>1 coffee or "energy drink" or \>2 caffeinated sodas per day based on caregiver- and adolescent-report will be excluded.
* Unwillingness or inability to take part in study procedures (e.g., visits, prescribed sleep conditions).
* Medication use or unwillingness to discontinue melatonin and/or stimulant medications during the 3-week study protocol.
* Refusal to refrain from automobile driving during the sleep restriction condition.
* "Intermediate" Chronotypes (neither morning larks nor night owls) based on habitual sleep timing on nights when adolescent has no morning obligations such as school or work. BEHAVIORAL: Aligned sleep extension, BEHAVIORAL: Misaligned sleep extension
ADHD
Safety and Efficacy of PMT Therapy of hPAP
Brenna Carey - brenna.carey@cchmc.org
ALL
18 years and over
PHASE1
NCT05761899
Inclusion Criteria:
Patients must meet all of the following conditions to be eligible for participation in this study:
• Male or female with a confirmed diagnosis of hPAP defined as: * Homozygous or compound heterozygous CSF2RA mutations - AND - * A normal GM-CSF autoantibody test result - AND - * An abnormal STAT5-PI test result - OR - * An abnormal GM-CSF 50% effective concentration (EC50) test result
• Diffuse ground glass opacification of the lungs visualized on a chest computed tomogram (CT)
• History of prior receipt of WLL therapy or moderate hPAP lung disease severity requiring therapy in the opinion of the Clinical Site Investigator and/or Sponsor
• Able to undergo bone marrow collection by routine clinical aspiration
• 18 years of age or older on the date the Informed consent form (ICF) is signed
• Females who have been post-menopausal for \>2 years or females of child-bearing potential after a confirmed menstrual period using a highly efficient method of contraception (as described in Section 11.4.2) for the period from 3 months prior to the first administration of gene-corrected macrophages until 12 months after the last administration of gene-corrected macrophages. Females of child-bearing potential must have a negative serum pregnancy test at Screening (Visit 1), at bone marrow collection (Visit 2), and immediately before each administration of gene-corrected macrophages (Visits 3, 5, 7), and must not be lactating.
• Males of reproductive potential must agree to use condoms for the period from the 1st administration of gene-corrected macrophages until 12 months after the last dose of gene-corrected macrophages, have a partner who is not of child-bearing potential (i.e. men or females who have been post-menopausal for \>2 years), or have a female partner who is using adequate contraception as described in Section 11.4.2.
• Signed written informed consent form (ICF)
Exclusion Criteria:
Patients who meet any of the following conditions will not be eligible for participation in this study:
• History of a confirmed diagnosis of any other PAP-causing disease defined as:
• PAP caused by function-altering mutations in CSF2RB, adenosine triphosphate (ATP)-binding cassette subfamily A member 3 (ABCA3), SFTPB, SFTPC, Thyroid Transcription Factor 1 (TTF-1), GATA-binding factor 2 (GATA2), SLC7A7, and methionyl-transfer RNA (tRNA) synthetase (MARS), or other genes demonstrated to cause PAP other than CSF2RA
• PAP associated with an abnormal GM-CSF autoantibody test
• PAP associated with hematologic disorders including but not limited to myelodysplasia, aplastic anemia, leukemia, multiple myeloma, lymphoma
• PAP associated with non-hematologic malignancies
• PAP associated with immune deficiency syndromes
• PAP associated with chronic inflammatory syndromes
• PAP associated with chronic infections including but not limited to human immunodeficiency virus, Mycobacteria tuberculosis or other Mycobacterial species, or other organisms
• PAP associated with inhaled materials including but not limited to inorganic dusts (e.g., silica, titanium, indium, aluminum), organic dusts (e.g., sawdust, fertilizer); or gases/vapors (e.g., cleaning products, paints, and welding-related fumes)
• Pulmonary fibrosis that is clinically significant in the opinion of Clinical Site Investigator and/or Sponsor
• A confirmed (i.e., repeated) positive serum anti-GM-CSF receptor antibody test and/or a confirmed positive anti-lentiviral antibody test at the time of screening and prior to each administration of gene-corrected macrophages
• History of receipt of any investigational agent within 3 months of Study Visit 3
• History of active chronic infection (e.g., HIV, Hepatitis, others) at the time of Screening
• History of significant alcohol consumption for a period of more than 3 consecutive months within 1 year prior to Study Visit 3, defined as more than 14 drinks/week for females or 21 drinks/week for males (1 drink - 5 ounces (150 ml) of wine or 12 ounces (360 ml) of beer, or 1.5 ounces (45 ml) of hard liquor)
• History of medication or illicit drug abuse within 1 year prior to Study Visit 3, including but not limited to cocaine, heroin, or other opioids
• Currently or planning to become pregnant between the Screening visit and Visit 14 and/or currently breast-feeding
• Any other medical, behavioral, or psychiatric condition that would interfere with the completion of Study Visits or assessments in the opinion of the Clinical Site Investigator and/or Sponsor
COMBINATION_PRODUCT: Gene-Corrected Macrophages administered by bronchoscopic instillation
Hereditary Pulmonary Alveolar Proteinosis
Pulmonary Alveolar Proteinosis, Pulmonary Macrophage Transplantation
Effects of Exercise on Lipids and Endothelial Function in Youth
Erin Cain - erin.cain@cchmc.org
ALL
10 years to 20 years old
NA
NCT07409064
Inclusion Criteria:
• Age: 10-20 years
• Obesity as defined by BMI ≥ 30 kg/m2 OR BMI ≥ 95%ile for age and sex
• Planned to start the Healthworks! structured weight loss program.
• English or Spanish speaking -
Exclusion Criteria:
1\. Physician judgement about inability to finish the protocol
\- OTHER: Exercise Program
Obesity & Overweight
Prospective Clinical Assessment Study in Children With Hypochondroplasia (HCH)
Laurie Laurie Bailey - Laurie.Bailey@cchmc.org
ALL
30 months to 16 years old
NCT06410976
Inclusion Criteria:
Signed informed consent.
Aged 2.5 to \<17 years at study entry.
Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test.
Participants are ambulatory and able to stand without assistance.
Study participants and parent(s), guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
Exclusion Criteria:
Have ACH or short stature condition other than HCH.
In females, having had their menarche. Annualized height growth velocity ≤1.5 cm/year over a period ≥6 months prior to screening.
Having a clinically significant disease or condition that in view of the investigator or Sponsor will interfere with the evaluation of growth, with study participation or not be in the best interest of the participant.
Clinically significant abnormality in any laboratory test result at screening
Current evidence of corneal or retinal disorders.
Have used any other investigational or approved product or medical device for the treatment of HCH or short stature for ≥ 30 days or with the last dose \<6 months before screening.
Have had regular long-term treatment (\>1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable).
Previous limb-lengthening surgery or guided growth surgery with plates still in place or removed within the 6 months prior to screening.
Having had a fracture of the long bones or spine within 12 months of screening.
History and/or current evidence of extensive ectopic tissue calcification.
History of malignancy.
Concurrent circumstance, disease, or condition that, in the view of the investigator and/or sponsor, would interfere with study participation, and/or would place the participant at high risk for poor compliance with study activities or for not completing the study.
Current participation in any other ongoing clinical study with another sponsor. Hypochondroplasia
skeletal dysplasia, endochondral ossification, hypochondroplasia, HCH, shortened proximal limbs, fibroblast growth factor receptor 3, FGFR3, endochondral bone formation, disproportionate short stature, quality of life, dwarfism, bone diseases, musculoskeletal diseases, osteochondrodysplasia, functional abilities, annualized growth velocity, annualized height velocity, growth, genetic diseases, congenital, AHV, AGV
A Study to See if Tolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)
Kelli Krallman - Kelli.Krallman@cchmc.org
ALL
28 days to 18 years old
PHASE3
NCT04782258
Inclusion Criteria:
• Male or female subjects between 28 days and less than 18 years of age, with clinical features that are consistent with a diagnosis of ARPKD.
• Ability for parent/legal guardian to provide written, informed consent prior to initiation of any trial-related procedures, and ability, in the opinion of the principal investigator, to comply with all the requirements of the trial. Ability to provide written informed assent from all subjects old enough per local laws to provide assent.
Exclusion Criteria:
• Premature birth (≤ 32 weeks gestational age) for infants 28 days to \< 12 weeks of age.
• Anuria or RRT defined as intermittent or continuous hemodialysis, peritoneal dialysis, hemofiltration, hemodiafiltration or history of kidney transplantation.
• Evidence of syndromic conditions associated with renal cysts (other than ARPKD).
• Abnormal liver function tests including ALT and AST, \> 1.2 × ULN (upper limit of normal).
• Has splenomegaly or portal hypertension (HTN).
• Parents with renal cystic disease.
• Receiving chronic diuretic that could not be adjusted after tolvaptan initiation.
• Cannot be monitored for fluid balance.
• Has or at risk of having sodium and potassium electrolyte imbalances, as determined by the investigator.
• Has or at risk of having significant hypovolemia as determined by investigator.
• Clinically significant anemia, as determined by investigator.
• Platelets \< 50000 µL.
• Severe systolic dysfunction defined as ejection fraction \< 14%.
• Serum sodium levels \< 130 mmol/L or \>145 mmol/L.
• Taking any other experimental medications.
• Require ventilator support.
• Taking medications known to induce CYP3A4 (CYP = Cytochrome P).
• Having an infection including viral that would require therapy disruptive to IMP (Investigational Medicinal Product) dosing.
• Females who are breast-feeding or who have a positive pregnancy test result prior to receiving IMP.
• Subjects with a history of substance abuse (within the last 6 months).
• Subjects who have bladder dysfunction and/or difficulty voiding.
• Subjects taking a vasopressin agonist (eg, desmopressin).
• Subjects with a history of persistent noncompliance with antihypertensive or other important medical therapy.
• Subjects taking medications or having concomitant illnesses likely to confound endpoint assessments, including taking approved (ie, marketed) therapies for the purpose of affecting PKD cysts such as tolvaptan, vasopressin antagonists, anti-sense ribonucleic acid (RNA) therapies, rapamycin, sirolimus, everolimus, or somatostatin analogs (ie, octreotide, sandostatin).
• Received or are scheduled to receive a liver transplant.
• History of cholangitis within the last 6 months.
• Has findings consistent with clinically significant portal hypertension (eg, varices, variceal bleeding, hypersplenism indicated by thrombocytopenia).
• Subjects who do not agree to remain abstinent or assent to use a combination of 2 of the following highly effective birth control methods for at least 28 days before the first dose of IMP, during the trial (including during IMP dose interruptions), and for at least 30 days after the last dose of IMP: * Barrier method of contraception: condoms (male or female) with or without a spermicidal agent, diaphragm or cervical cap with spermicide * Intrauterine device * Hormone-based contraceptives which are associated with inhibition of ovulation.
DRUG: Tolvaptan Suspension, DRUG: Tolvaptan Tablets
Autosomal Recessive Polycystic Kidney (ARPKD)
ARPKD, TOLVAPTAN, Polycystic Kidney Disease, Autosomal Recessive Polycystic Kidney Disease, Adolescent, Renal Cysts, Oligohydramnios, Anhydramnios
A Study of the Drug Selinexor With Radiation Therapy in Patients With Newly-Diagnosed Diffuse Intrinsic Pontine (DIPG) Glioma and High-Grade Glioma (HGG)
- cancer@cchmc.org
ALL
12 months to 21 years old
PHASE1
NCT05099003
Inclusion Criteria:
* PRE ENROLLMENT: Patients must be =\< 25 years of age at the time of enrollment on APEC14B1 part A central nervous system (CNS)/high grade glioma (HGG) pre-enrollment eligibility screening
* Please note:
* This required age range applies to pre-enrollment eligibility for all HGG patients. Individual treatment protocols may have different age criteria.
* Non-DIPG patients with tumors that do not harbor an H3K27M-mutation and are \>= 18 years of age will not be eligible to enroll on ACNS1821 (Step 1).
* PRE ENROLLMENT: Patient is suspected of having localized, newly diagnosed HGG, excluding metastatic disease, OR patient has an institutional diagnosis of DIPG
* Please note: there are specific radiographic criteria for DIPG patient enrollment on ACNS1821 (Step 1)
* As of February 14, 2025, stratum DIPG and stratum DMG have closed to accrual, and no patients will be enrolled on these strata after Amendment #4.
* PRE ENROLLMENT:
* For patients with non-pontine tumors: Patients and/or their parents or legal guardians must have signed informed consent for eligibility screening on APEC14B1 Part A.
* For patients with DIPG: Patients and/or their parents or legal guardians must have signed informed consent for ACNS1821.
* Note: As of February 14, 2025, stratum DIPG and stratum DMG have closed to accrual, and no patients will be enrolled on these strata after Amendment #4.
* PRE ENROLLMENT:
* For patients with non-pontine tumors only, the specimens obtained at the time of diagnostic biopsy or surgery must be submitted through APEC14B1 ASAP, preferably within 5 calendar days of definitive surgery
* STEP 1: Patients must be \>= 12 months and =\< 21 years of age at the time of enrollment
* STEP 1: Patients must have newly-diagnosed DIPG or HGG (including DMG).
* STEP 1: Stratum DIPG (Closed with Amendment #4)
* As of February 14, 2025, stratum DIPG and stratum DMG have closed to accrual, and no patients will be enrolled on these strata after Amendment #4.
* Patients with newly-diagnosed typical DIPG, defined as tumors with a pontine epicenter and diffuse involvement of at least 2/3 of the pons on at least 1 axial T2 weighted image, are eligible. No histologic confirmation is required.
* Patients with pontine tumors that do not meet radiographic criteria for typical DIPG (e.g., focal tumors or those involving less than 2/3 of the pontine cross-sectional area with or without extrapontine extension) are eligible if the tumors are biopsied and proven to be high-grade gliomas (such as anaplastic astrocytoma, glioblastoma, high-grade glioma not otherwise specified \[NOS\], and/or H3 K27M-mutant) by institutional diagnosis.
* STEP 1: Stratum DMG (with H3 K27M mutation) (Closed with Amendment #4)
* As of February 14, 2025, stratum DIPG and stratum DMG have closed to accrual, and no patients will be enrolled on these strata after Amendment #4.
* Patients must have newly-diagnosed non-pontine H3 K27M-mutant HGG without BRAF V600 or IDH1 mutations as confirmed by Rapid Central Pathology and Molecular Screening Reviews performed on APEC14B1
* Note: Patients need not have either measurable or evaluable disease, i.e., DMG patients may have complete resection of their tumor prior to enrollment. Primary spinal tumors are eligible for enrollment. For rare H3 K27M-mutant HGG in non-midline structures (e.g., cerebral hemispheres), these patients will be considered part of Stratum DMG.
* STEP 1: Stratum HGG (without H3 K27M mutation)
* Patients must have newly-diagnosed non-pontine H3 K27M-wild type HGG without BRAF V600 or IDH1 mutations as confirmed by Rapid Central Pathology and Molecular Screening Reviews performed on APEC14B1
* Please note:
* Patients who fall in this category and who are \>= 18 years of age are not eligible due to another standard-of-care regimen (radiation/temozolomide) that is available
* Patients need not have either measurable or evaluable disease, i.e., HGG patients may have complete resection of their tumor prior to enrollment. Primary spinal tumors are eligible for enrollment
* STEP 1: Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2. Use Karnofsky for patients \> 16 years of age and Lansky for patients =\<16 years of age. 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.
* STEP 1: Peripheral absolute neutrophil count (ANC) \>= 1000/uL (within 7 days prior to step 1 enrollment)
* STEP 1: Platelet count \>= 100,000/uL (transfusion independent) (within 7 days prior to step 1 enrollment)
* STEP 1: Hemoglobin \>= 8.0 g/dL (may receive red blood cell \[RBC\] transfusions) (within 7 days prior to step 1 enrollment)
* STEP 1: Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 (within 7 days prior to step 1 enrollment) or
A serum creatinine based on age/sex as follows (within 7 days prior to step 1 enrollment):
* Age / Maximum Serum Creatinine (mg/dL)
* 1 to \< 2 years / male: 0.6; female: 0.6
* 2 to \< 6 years / male: 0.8; female: 0.8
* 6 to \< 10 years / male: 1; female: 1
* 10 to \< 13 years / male: 1.2; female: 1.2
* 13 to \< 16 years / male: 1.5; female: 1.4
* \>= 16 years / male: 1.7; female: 1.4
* STEP 1: Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age
* STEP 1: Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) =\< 135 U/L. For the purpose of this study, the ULN for SGPT is 45 U/L.
* STEP 1: Serum amylase =\< 1.5 x ULN
* STEP 1: Serum lipase =\< 1.5 x ULN
* STEP 1: No evidence of dyspnea at rest, no exercise intolerance, and a pulse oximetry \> 94% if there is clinical indication for determination.
* STEP 1: Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled.
* STEP 1: Patients must be enrolled and protocol therapy must begin no later than 31 days after the date of radiographic diagnosis (in the case of non-biopsied DIPG patients only) or definitive surgery, whichever is the later date (Day 0).
For patients who have a biopsy followed by resection, the date of resection will be considered the date of definitive diagnostic surgery. If a biopsy only was performed, the biopsy date will be considered the date of definitive diagnostic surgery.
Exclusion Criteria:
* STEP 1: Patients must not have received any prior therapy for their central nervous system (CNS) malignancy except for surgery and steroid medications.
* STEP 1: Patients who are currently receiving another investigational drug are not eligible.
* STEP 1: Patients who are currently receiving other anti-cancer agents are not eligible.
* STEP 1: Patients \>=18 years of age who have H3 K27M-wild type HGG.
* STEP 1: Patients who have an uncontrolled infection.
* STEP 1: Patients who have received a prior solid organ transplantation.
* STEP 1: Patients with grade \> 1 extrapyramidal movement disorder.
* STEP 1: Patients with known macular degeneration, uncontrolled glaucoma, or cataracts.
* STEP 1: Patients with metastatic disease are not eligible; MRI of spine with and without contrast must be performed if metastatic disease is suspected by the treating physician.
* STEP 1: Patients with gliomatosis cerebri type 1 or 2 are not eligible, with the exception of H3 K27M-mutant bithalamic tumors.
* STEP 1: Patients who are not able to receive protocol specified radiation therapy.
* STEP 1:
* Female patients who are pregnant are ineligible since there is yet no available information regarding human fetal or teratogenic toxicities.
* Lactating females are not eligible unless they have agreed not to breastfeed their infants. It is not known whether selinexor is excreted in human milk.
* Female patients of childbearing potential are not eligible unless a negative pregnancy test result has been obtained.
* Sexually active patients of reproductive potential are not eligible unless they have agreed to use two effective methods of birth control (including a medically accepted barrier method of contraception, e.g., male or female condom) for the duration of their study participation and for 90 days after the last dose of selinexor. Abstinence is an acceptable method of birth control. PROCEDURE: Biopsy Procedure, PROCEDURE: Magnetic Resonance Imaging, RADIATION: Radiation Therapy, DRUG: Selinexor
Malignant Glioma
Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies
Charles Alexander - Charles.Alexander@cchmc.org
ALL
1 year to 21 years old
PHASE1
NCT05476770
Inclusion Criteria:
Age
* Patients must be ≥ 1 and ≤21 years of age at the time of study enrollment.
Diagnosis
* Relapsed and/or refractory hematologic malignancy (including, but not limited to, acute lymphoblastic leukemia, acute myeloid leukemia, myelodysplastic syndrome, mixed phenotype acute leukemia, acute undifferentiated leukemia, blastic plasmacytoid dendritic cell neoplasm, Hodgkin lymphoma, and non-Hodgkin lymphoma).
* Tumor cells must demonstrate surface expression of CD123 at the time of enrollment by flow cytometry or immunohistochemistry, as defined by the local institution.
Disease Status:
Monotherapy, Part 1
* Second or greater relapse; or
* Refractory after 2 or more chemotherapy cycles; or
* First relapse after primary chemotherapy-refractory disease; or
* BPDCN in first relapse or refractory after 1 or more chemotherapy cycles
Combination therapy, Part 2
* First or greater relapse; or
* Refractory after 2 or more chemotherapy cycles; or
* BPDCN in first relapse or refractory after 1 or more chemotherapy cycles
For relapsed/refractory leukemia, patients must have:
* \>5% blasts in the bone marrow aspirate or biopsy by morphology or flow cytometry
* Patients with 1% - 5% blasts are eligible for Part 2, Cohort C (only), if A single bone marrow sample with flow cytometry and at least one other test (e.g. karyotype, FISH, PCR, or NGS) shows ≥ 1% leukemic blasts and/or flow cytometry demonstrates a stable or rising level of disease on two serial bone marrows.
For relapsed/refractory non-Hodgkin or Hodgkin lymphoma, patients must have:
* Histologic verification of relapse
* Measurable disease documented by radiographic criteria or bone marrow
* Patients in Part 1 may have sites of non-CNS extramedullary disease, but no CNS disease. Patients in Part 2 may have CNS disease and/or other non-CNS extramedullary disease. No cranial irradiation is allowed during the protocol therapy.
* Patients with Down syndrome are eligible to participate in Part 1 only.
Performance Level
* Karnofsky \> 50% for patients \> 16 years of age and Lansky \> 50% for patients ≤ 16 years of age (See Appendix I for Performance Scales). 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 fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy, defined as resolution of all such toxicities to ≤ Grade 2 or lower per the inclusion/exclusion criteria.
Myelosuppressive chemotherapy: Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study. At least 14 day must have elapsed since the completion of myelosuppressive therapy. However, individuals may receive any of the following medications within 14 days without a "wash-out period":
* Hydroxyurea: Hydroxyurea can be initiated and/or continued for up to 24 hours prior to the start of protocol therapy.
* "Maintenance-style" therapy: therapy including vincristine (dosed a maximum of one-time weekly), oral 6-mercaptopurine, oral methotrexate (dosed a maximum of one-time weekly), intrathecal therapy (dosed a maximum of one-time weekly) and/or dexamethasone (dosed at ≤3 mg/m2/dose twice daily) or prednisone (dosed at ≤20 mg/m2/dose twice daily) can be continued for up to 24 hours prior to entering the study.
* Hematopoietic stem cell transplant: Patients who have experienced their relapse after a HSCT are eligible, provided they have no evidence of acute or chronic Graft-versus-Host Disease (GVHD) and are at least 100 days post-transplant at the time of enrollment.
* Hematopoietic growth factors: It must have been at least 7 days since the completion of therapy with granulocyte colony stimulating factor (GCSF) or other growth factors at the time of enrollment. It must have been at least 14 days since the completion of therapy with pegfilgrastim (Neulasta®).
* Biologic (anti-neoplastic agent): At least 7 days after the last 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.
* Monoclonal antibodies: Maximum of 3 half-lives of the antibody or 21 days (whichever is shorter) must have elapsed after the last dose of monoclonal antibody.
* Immunotherapy: At least 30 days from last infusion of chimeric antigen receptor T cell (CART) therapy or tumor vaccine.
* Radiation Therapy (XRT):
• ≥ 84 days must have passed, from the end of therapy, if patient received prior total body irradiation (TBI).
• ≥ 42 days must have passed, from the end of therapy, if patient received craniospinal irradiation (CSI).
• ≥ 14 days must have passed after whole brain radiotherapy or stereotactic radiation therapy.
• No washout period is required for: i. Extramedullary site other than CNS that is a maximum 10 x 10 cm total radiation non-CNS field. If the field is \> 10 x 10 cm, a 14-day washout period is required. ii. Local ocular radiotherapy as long as subject has measurable/evaluable disease outside the radiation port. * Patients that have received other non-tagraxofusp CD123 targeting agents are eligible. Patients that have previously received tagraxofusp are not eligible. Organ Function Requirements Adequate Bone Marrow Function Defined as: * Patients should not be known to be refractory to red blood cell or platelet transfusions. * Blood counts are not required to be normal prior to enrollment on trial. However, platelet count must be ≥20,000/mm3 to initiate therapy (may receive platelet transfusions). 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 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: * Total bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x institutional upper limit of normal for age * SGPT (ALT) and SGOT (AST) must be less than 3x institutional upper limit of normal. * Serum albumin ≥3.2 g/dL (albumin infusion independent). Adequate Cardiac Function Defined as: * Shortening fraction of ≥27% by echocardiogram, or * Ejection fraction of ≥ 50% by gated radionuclide study/echocardiogram. Adequate Pulmonary Function Defined as: * Pulse oximetry \> 94% on room air (\> 90% if at high altitude) * No evidence of dyspnea at rest and no exercise intolerance. 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 12 weeks after the last dose of tagraxofusp. Exclusion Criteria Disease Status: * Patients with CNS disease are not eligible for Part 1. * Patients with isolated CNS disease are not eligible for Part 1 or Part 2. * Patients with isolated non-CNS disease are eligible for Part 1 and Part 2. Concomitant Medications * Corticosteroids - Patients receiving corticosteroids for disease control 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. The definition of "investigational" for use in this protocol means any drug that is not licensed by the FDA, Health Canada or the Therapeutic Goods Administration to be sold in the countries they govern. (United States, Canada and Australia) * Anti-cancer Agents - Patients who are currently receiving or may receive while on therapy, other anti-cancer agents, radiation therapy or immunotherapy are not eligible \[with the exceptions being laid out in the inclusion criteria under 'Prior Therapy'\]. Intrathecal chemotherapy (at the discretion of the primary oncologist) may be given up to one week prior to the initiation of study treatment (day 1 therapy). * Anti-GVHD or agents to prevent organ rejection post-transplant - Patients who are receiving cyclosporine, tacrolimus or other agents to prevent either graft-versus-host disease post bone marrow transplant or organ rejection post-transplant are not eligible for this trial. At least 4 weeks must have elapsed after the last dose of GVHD meds. Infection Criteria - Patients are excluded if they have: * Positive blood culture within 48 hours of study enrollment; * Fever above 38.2 within 48 hours of study enrollment with clinical signs of infection. Fever that is determined to be due to tumor burden is allowed if patients have documented negative blood cultures for at least 48 hours prior to enrollment and no concurrent signs or symptoms of active infection or hemodynamic instability. * A positive fungal culture within 30 days of study enrollment. * Active fungal, viral, bacterial, or protozoal infection requiring IV treatment. Chronic prophylaxis therapy to prevent infections is allowed. * Patients will be excluded if they have a known allergy to any of the drugs used in the study. * 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. * Patients with DNA fragility syndromes (such as Fanconi anemia, Bloom syndrome) are excluded.
DRUG: Tagraxofusp, DRUG: Fludarabine, DRUG: Cytarabine, DRUG: Dexamethasone, DRUG: Vincristine, DRUG: Azacitidine, DRUG: Methotrexate, DRUG: Cytarabine IT, DRUG: Hydrocortisone
Hematologic Malignancy, AML, ALL, BPDCN, MDS, Lymphoblastic Lymphoma, Lymphoma, B-Cell, Lymphoma, T-Cell, Hodgkin Lymphoma, Mixed Phenotype Acute Leukemia, Acute Undifferentiated Leukemia
Follow-up Automatically vs. As-Needed Comparison (FAAN-C) Trial (FAAN-C)
Holly Flake - Holly.Flake@cchmc.org
ALL
Up to 18 years old
NA
NCT05471908
Inclusion Criteria:
* Age \<18 years at the time of randomization
* Hospitalization due to a primary diagnosis of pneumonia, skin and soft tissue infection, acute gastroenteritis, or urinary tract infection.
* Parent speaks English or Spanish.
Exclusion Criteria:
* Presence of a comorbid disease that is both chronic and complex
* Principal disease required surgical intervention (beyond superficial incision and drainage)
* Immunodeficiency
* A well-child check-up or post-hospitalization follow-up visit is already scheduled within 7 days of hospital discharge
* Parent or participant strongly prefers PRN or automatic follow-up
* A medical provider feels strongly that a post-hospitalization follow-up visit is needed within 7 days of hospital discharge
* Sibling concurrently hospitalized
* Unable to identify a clinic where the participant would receive any needed post-hospitalization follow-up
* Diagnosis of pneumonia complicated by:
o Receiving a chest tube
* Diagnosis of urinary tract infection complicated by:
* History of neurogenic bladder or urologic surgery
* Renal imaging anticipated within 7 days of hospital discharge
* Renal abscess
* Diagnosis of skin and soft tissue infection complicated by:
* Chronic wound
* Postoperative infection
* Predisposition to poor wound healing
* Discharging with a drain in place
* Complicated by necrotizing fasciitis or toxic shock syndrome
* Diagnosis of gastroenteritis complicated by:
* Hemolytic uremic syndrome BEHAVIORAL: As-needed follow up, BEHAVIORAL: Automatic follow-up
Pneumonia, Urinary Tract Infections, Soft Tissue Infections, Gastroenteritis
post-hospitalization, follow-up care, patient centered care, randomized control trial
A Global Prospective Observational Registry of Patients With Pompe Disease
Jenel Facey - jenel.facey@cchmc.org
ALL
NCT06121011
Inclusion Criteria:
* Diagnosis of LOPD or IOPD based on documented deficiency of GAA enzyme activity and/or GAA genotyping
Exclusion Criteria:
* Patients who are currently receiving investigational therapy for Pompe disease in a clinical trial, a compassionate use program, or an expanded access program (EAP) BIOLOGICAL: Cipaglucosidase alfa, DRUG: Miglustat, BIOLOGICAL: Alglucosidase alfa or Avalglucosidase alfa, OTHER: Untreated
Pompe Disease
A Gaucher Disease Gene Therapy Trial With FLT201 (GALILEO-3)
Laurie Bailey - laurie.bailey@cchmc.org
ALL
18 years and over
PHASE3
NCT07223944
Key
Inclusion Criteria:
* Aged ≥18 years at time of screening.
* Clinical diagnosis of Gaucher disease type 1
* Stable hemoglobin concentration at baseline
* Stable platelet count at baseline
* Receiving ERT or SRT without interruption for at least 2 years
Key Exclusion Criteria:
* Diagnosed or suspected Gaucher disease type 2 or type 3
* Positive for AAVS3 neutralizing antibodies.
* Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
* Positive pregnancy test or lactating
* History of hematopoietic stem cell transplant (HSCT)/bone marrow transplant or any solid organ transplant.
* History of receiving any gene therapy or cell therapy.
* History of total splenectomy. Note: Additional protocol defined Inclusion and Exclusion criteria apply GENETIC: FLT201
Gaucher Disease Type 1
Project: Every Child for Younger Patients With Cancer
- cancer@cchmc.org
ALL
Up to 25 years old
NCT02402244
Inclusion Criteria:
* Enrollment must occur within 6 months of initial disease presentation OR within 6 months of refractory disease, disease progression, disease recurrence, second or secondary malignancy, or post-mortem
* Patients previously enrolled on ACCRN07 are eligible to enroll on Tracking Outcome, Registry and Future Contact components of APEC14B1 any time after they reach age of majority
* Patients with a known or suspected neoplasm that occurs in the pediatric, adolescent or young adult populations are eligible for enrollment as follows:
* All cancer cases with an International Classification of Diseases for Oncology (ICD-O) histologic behavior code of one "1" (borderline), two "2" (carcinoma in situ) or three "3" (malignant)
* All neoplastic lesions of the central nervous system regardless of behavior, i.e., benign, borderline or malignant
* All neoplastic lesions of the kidney regardless of behavior, i.e., benign, borderline or malignant
* The following other benign/borderline conditions:
* Mesoblastic nephroma
* Teratomas (mature and immature types)
* Myeloproliferative diseases including transient myeloproliferative disease
* Langerhans cell histiocytosis
* Lymphoproliferative diseases
* Desmoid tumors
* Gonadal stromal cell tumors
* Neuroendocrine tumors including pheochromocytoma
* Melanocytic tumors, except clearly benign nevi
* Ganglioneuromas
* Subjects must be =\< 25 years of age at time of original diagnosis, except for patients who are being screened specifically for eligibility onto a COG (or COG participating National Clinical Trials Network \[NCTN\]) therapeutic study, for which there is a higher upper age limit
* All patients or their parents or legally authorized representatives must sign a written informed consent and agree to participate in at least one component of the study; parents will be asked to sign a separate consent for their own biospecimen submission
* If patients or their parents or legally authorized representatives have not signed the Part A subject consent form at the time of a diagnostic bone marrow procedure, it is recommended that they initially provide consent for drawing extra bone marrow using the Consent for Collection of Additional Bone Marrow; consent using the Part A subject consent form must be provided prior to any other procedures for eligibility screening or banking under APEC14B1 OTHER: Cytology Specimen Collection Procedure, OTHER: Medical Chart Review
Adrenal Gland Pheochromocytoma, Carcinoma In Situ, Central Nervous System Neoplasm, Childhood Immature Teratoma, Childhood Kidney Neoplasm, Childhood Langerhans Cell Histiocytosis, Childhood Mature Teratoma, Congenital Mesoblastic Nephroma, Desmoid Fibromatosis, Ganglioneuroma, Lymphoproliferative Disorder, Malignant Neoplasm, Malignant Solid Neoplasm, Melanocytic Neoplasm, Myeloproliferative Neoplasm, Neoplasm of Uncertain Malignant Potential, Neuroendocrine Neoplasm, Stromal Neoplasm
Registry for Stage 2 Type 1 Diabetes
Cierra Farrell - cierra.farrell@cchmc.org
ALL
NCT06481904
Inclusion Criteria:
TZIELD-Exposed Cohort
* Patients in the US diagnosed with Stage 2 T1D who are planned to initiate TZIELD treatment according to the currently approved label or who have initiated TZIELD treatment within 6 months prior to enrollment:
* Day 1: 65 mcg/m2
* Day 2: 125 mcg/m2
* Day 3: 250 mcg/m2
* Day 4: 500 mcg/m2
* Days 5 through 14: 1,030 mcg/m2 per day
* Cumulative dose is approximately 11,240 mcg/m2
* Appropriate written informed consent/assent as applicable for the age of the patient
TZIELD-Unexposed Cohort
* Patients in the US diagnosed with Stage 2 T1D but who are not treated with TZIELD
* Appropriate written informed consent/assent as applicable for the age of the patient
Exclusion Criteria:
* Patients who initiated TZIELD treatment more than 6 months prior to enrollment
* Patients who had participated in a previous clinical trial for TZIELD
* Patients in an ongoing clinical trial of an investigational product or who had ended participation within 6 months prior to study enrollment; patients participating in other observational studies may be enrolled
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial. DRUG: TZIELD (teplizumab-mzwv)
Type 1 Diabetes
BSGM to Evaluate Patients With GI Symptoms
Khalil El-Chammas, MD - Khalil.El-Chammas@cchmc.org
ALL
8 years to 25 years old
NCT05880199
Inclusion Criteria for Cases
• Males or females age 8 to 25 years.
• Females ≥11 years of age or who have reached menarche must have a negative urine pregnancy test.
• Confirmed diagnosis of a Functional Gastrointestinal and/or Motility Disorder OR undergoing one of the following procedures as part of their clinical care at one of the participating centers:
• HRVB
• PENFS
• ADM
• Colonic Manometry
• Pyloric Botox
• Pyloric Dilation
• Gastric Scintigraphy
• GES
• gammaCore
• Those with a body mass index of \< 35.
• Parental/guardian permission (informed consent) and if appropriate, child assent. Exclusion Criteria for Cases
• History of skin allergies or a history of extreme sensitivity to cosmetics or lotions. Currently open wounds, abrasions, infected or inflamed abdominal skin. (Please note, majority of feeding tubes can be accommodated by the array placement.)
• Pregnant women.
• Those with any condition, where fasting is not recommended by a physician.
• Any allergies to foods that may be present in the standardized meal that cannot be accommodated with an acceptable substitute meal.
• Those with physical limitations, who are not able to maintain a relaxed reclined position for the study visit duration.
• Those with major developmental delay or cognitive impairment, who are not able to report their symptoms/feelings in the questionnaires.
• Those with GI motility disorders that are limited in the esophagus, and the gastric mapping is restricted to capture relevant data based on the investigator's discretion.
• Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures. Inclusion Criteria for Controls
• Males or females age 8 to 25 years.
• Females ≥11 years of age or who have reached menarche must have a negative urine pregnancy test.
• Do not have an active Functional Gastrointestinal disorder (FGID) diagnosis and will not be undergoing any procedures outlined in the recruitment plan in the near future.
• Those with a body mass index of \< 35.
• Individuals may include siblings of those with FGIDs.
• Parental/guardian permission (informed consent) and if appropriate, child assent. Exclusion Criteria for Controls
• History of skin allergies or a history of extreme sensitivity to cosmetics or lotions. Currently open wounds, abrasions, infected or inflamed abdominal skin.
• Pregnant women.
• Those with any condition, where fasting is not recommended by a physician.
• Allergies to foods that may be included in the standardized meal that cannot be accommodated with an acceptable substitute meal.
• Those with physical limitations, who are not able to maintain a relaxed reclined position for the study duration.
• Those with major developmental delay or cognitive impairment, who are not able to report their symptoms/feelings in the questionnaires.
• Those with GI motility disorders that are limited in the esophagus, and the gastric mapping is restricted to capture relevant data based on the investigator's discretion.
• Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures.
• Males or females age 8 to 25 years.
• Females ≥11 years of age or who have reached menarche must have a negative urine pregnancy test.
• Confirmed diagnosis of a Functional Gastrointestinal and/or Motility Disorder OR undergoing one of the following procedures as part of their clinical care at one of the participating centers:
• HRVB
• PENFS
• ADM
• Colonic Manometry
• Pyloric Botox
• Pyloric Dilation
• Gastric Scintigraphy
• GES
• gammaCore
• Those with a body mass index of \< 35.
• Parental/guardian permission (informed consent) and if appropriate, child assent. Exclusion Criteria for Cases
• History of skin allergies or a history of extreme sensitivity to cosmetics or lotions. Currently open wounds, abrasions, infected or inflamed abdominal skin. (Please note, majority of feeding tubes can be accommodated by the array placement.)
• Pregnant women.
• Those with any condition, where fasting is not recommended by a physician.
• Any allergies to foods that may be present in the standardized meal that cannot be accommodated with an acceptable substitute meal.
• Those with physical limitations, who are not able to maintain a relaxed reclined position for the study visit duration.
• Those with major developmental delay or cognitive impairment, who are not able to report their symptoms/feelings in the questionnaires.
• Those with GI motility disorders that are limited in the esophagus, and the gastric mapping is restricted to capture relevant data based on the investigator's discretion.
• Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures. Inclusion Criteria for Controls
• Males or females age 8 to 25 years.
• Females ≥11 years of age or who have reached menarche must have a negative urine pregnancy test.
• Do not have an active Functional Gastrointestinal disorder (FGID) diagnosis and will not be undergoing any procedures outlined in the recruitment plan in the near future.
• Those with a body mass index of \< 35.
• Individuals may include siblings of those with FGIDs.
• Parental/guardian permission (informed consent) and if appropriate, child assent. Exclusion Criteria for Controls
• History of skin allergies or a history of extreme sensitivity to cosmetics or lotions. Currently open wounds, abrasions, infected or inflamed abdominal skin.
• Pregnant women.
• Those with any condition, where fasting is not recommended by a physician.
• Allergies to foods that may be included in the standardized meal that cannot be accommodated with an acceptable substitute meal.
• Those with physical limitations, who are not able to maintain a relaxed reclined position for the study duration.
• Those with major developmental delay or cognitive impairment, who are not able to report their symptoms/feelings in the questionnaires.
• Those with GI motility disorders that are limited in the esophagus, and the gastric mapping is restricted to capture relevant data based on the investigator's discretion.
• Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures.
DEVICE: Body surface gastric mapping device
Gastrointestinal Motility Disorders in Children, Functional Gastrointestinal Disorders, Gastroparesis, Dyspepsia and Other Specified Disorders of Function of Stomach
GI motility, gastroparesis, functional dyspepsia
Thebaine Urine Testing
Holly Flake - holly.flake@cchmc.org
ALL
18 years to 65 years old
NCT07192887
Inclusion Criteria:
* Male or female
* Ages 18-65
* Healthy
Exclusion Criteria:
* Allergy to poppy seeds, or any of the related food stuffs used in the study
* Celiac disease or gluten sensitivity
* Ongoing or opioid use in the past 6 months
* History of renal or hepatic disease or dysfunction
* Inability or unwillingness to give repeated urine specimens
* Unwillingness to refrain from eating poppy seed-containing products during the two-day course of the study
* Non-English speakers (due to study materials being in English)
* BMI greater than 30
* Antibiotic use in the previous 2 months
* Recent ingestion of poppy seeds from the previous 7 days Urine Specimen Collection
ACTEMRA® for the Treatment of Pediatric Adamantinomatous Craniopharyngioma
- cancer@cchmc.org
ALL
1 year to 39 years old
PHASE2
NCT05233397
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 (CLOSED): 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 or cyst fenestration, organ resection, 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 and Lumbar Punctures are considered minor procedures..
• 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: 1 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 within normal institutional limits * AST (SGOT) ≤ 2.5 × institutional upper limit of normal * ALT (SGPT) ≤ 2.5 × institutional upper limit of normal 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
• 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 major or intermediate 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.
DRUG: Tocilizumab
Adamantinomatous Craniopharyngioma, Recurrent Adamantinomatous Craniopharyngioma
Continued Pressure for Alveolar Protection (CPAP Trial) (CPAP)
Traci Beiersdorfer - traci.beiersdorfer@cchmc.org
ALL
Up to 7 month(s) old
NA
NCT07417111
Inclusion Criteria:
* Gestational age \<29 weeks at birth
* PMA \<32 weeks at study entry
* On treatment with CPAP without a rate in FiO2 \<0.25 and PEEP of 4-5 cmH2O
* Meet stability criteria:
* If previously intubated must be extubated ≥ 72 hours
* \<3 self-resolving apneas (≤ 20 s) and/or bradycardia (\<100 bpm) in any hour over previous 6 hours
* No episodes of apnea or bradycardia requiring intervention (oxygen/stimulation/bag and mask) for 24 hours
* Parents/legal guardians consent for enrollment
Exclusion Criteria:
* Major malformation
* Neuromuscular condition that affects respiration
* Terminal illness
* Decision to withhold or limit support
* Too sick to participate in opinion of Attending physician
* Clinical shock, sepsis
* Planned surgery during study period DEVICE: CPAP, DEVICE: Nasal Cannula
Bronchopulmonary Dysplasia (BPD)
Continuous Positive Airway Pressure, CPAP
Evaluating Additive Effects of Including Canines in Regulating Together
Carrie Fassler - carrie.fassler@cchmc.org
ALL
8 years to 15 years old
NA
NCT05803343
Inclusion Criteria:
* Concern of emotion dysregulation (ED) as measured by a score of 6 or greater on the Emotion Dysregulation Inventory-Reactivity (EDI-R)
* Diagnosis of autism spectrum disorder (ASD)
* Diagnosis confirmed by an experienced ASD clinician and further supported by scoring in the range for ASD on the Autism Diagnostic Observation Schedule (ADOS-2)
* A Full Scale Intelligence Quotient score of 65 or greater on the Weschler Abbreviated Scale Intelligence (WASI-II)
* English is the primary language
* Family willing to keep prescribed medication stable over the course of the study period
Exclusion Criteria:
* Participant has a phobia toward or is allergic to canines
* Participant has a history of aggression toward animals
* Participant has had any physical aggression toward other children outside the home in the past 2 weeks that resulted in injury
* Presence of comorbid major neuropsychiatric illness warranting other treatment approaches as determined by the study clinician(s) including substance use disorders, psychotic disorders/schizophrenia, and bipolar disorder, among others
* Presence of any major sensory impairment that would limit participating in the material including blindness or uncorrected hearing loss
* A legal guardian is not available to provide informed consent BEHAVIORAL: Regulating Together-Canine, BEHAVIORAL: Regulating Together-Standard
Autism Spectrum Disorder, Emotion Regulation