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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
A Study to Evaluate Glofitamab Monotherapy and Glofitamab + Chemoimmunotherapy in Pediatric and Young Adult Participants With Relapsed/Refractory Mature B-Cell Non-Hodgkin Lymphoma (iMATRIX GLO)
- cancer@cchmc.org
ALL
6 months to 30 years old
PHASE1
NCT05533775
Inclusion Criteria:
* Age 6 months to \< 18 years at the time of signing Informed Consent for Cohort A Part 1 and Cohort B of the study, and age 6 months to \< 30 years old at the time of signing Informed Consent for Cohort A Part 2 of the study
* Histologically re-confirmed diagnosis, via tissue biopsy, or bone marrow aspirate, pleural effusion, or ascites, prior to study entry of aggressive mature B-NHL that expresses CD20 (reconfirmed by IHC or flow cytometry if IHC is not possible), including BL, BAL (mature B-cell leukemia FAB L3), DLBCL, and PMBCL, at the time of first R/R disease for Cohort A and second or greater R/R disease for Cohort B
* Refractory or relapsed disease (i.e., prior treatment was ineffective or intolerable) following first-line standard-of-care chemoimmunotherapy for Cohort A and following at least two prior systemic chemoimmunotherapy regimens and who have exhausted all available established therapies for Cohort B
* Measurable disease, defined as: At least one bi-dimensionally measurable nodal lesion, defined as \> 1.5 cm in its longest dimension, or at least one bi dimensionally measurable extranodal lesion, defined as \> 1.0 cm in its longest dimension; or percentage of bone marrow involvement with lymphoma cells defined by cytomorphological analysis of bone marrow aspirates
* Adequate performance status, as assessed according to the Lansky or Karnofsky Performance Status scales: Participants \< 16 years old: Lansky Performance Status ≥ 50%; Participants ≥ 16 years old: Karnofsky Performance Status ≥ 50%
* Adequate bone marrow, liver, and renal function
* Negative test results for acute or chronic hepatitis B virus (HBV), hepatitis C virus (HCV)
* Negative HIV test at screening, with the following exception: Individuals with a positive HIV test at screening are eligible provided they are stable on anti-retroviral therapy for at least 4 weeks, have a CD4 count ≥200/uL, have an undetectable viral load, and have not had a history of opportunistic infection attributable to AIDS within the last 12 months
* Negative SARS-CoV-2 antigen or PCR test within 7 days prior to enrollment
* Participants and/or caregivers who are willing and able to complete clinical outcome assessments throughout the study using either paper or interviewer methods
Exclusion Criteria:
* Isolated CNS disease of mature B-NHL without systemic involvement, and primary CNS lymphoma
* Receipt of glofitamab prior to study enrollment
* Ongoing adverse events from prior anti-cancer therapy that were not resolved to Grade ≤ 1 (exceptions: alopecia, Grade 2 peripheral neuropathy)
* Grade ≥ 3 adverse events, with the exception of Grade 3 endocrinopathy managed with replacement therapy
* Participants with active infections which are not resolved prior to Day 1 of Cycle 1
* Prior solid organ transplantation
* Known or suspected history of hemophagocytic lymphohistiocytosis (HLH), or chronic active Epstein-Barr viral infection (CAEBV)
* Active autoimmune disease requiring treatment
* History of severe allergic or anaphylactic reactions to monoclonal antibody therapy (or recombinant antibody-related fusion proteins) or known sensitivity or allergy to murine products, except if the participant was able to safely receive it after initial administration (consider consultation with Medical Monitor)
* History of confirmed progressive multifocal leukoencephalopathy
* Current or past history of uncontrolled non-malignant CNS disease, such as stroke, epilepsy, CNS vasculitis, or neurodegenerative disease
* Evidence of significant and uncontrolled concomitant diseases that could affect compliance with the protocol or interpretation of results
* Major surgery or significant traumatic injury \< 28 days prior to the obinutuzumab pretreatment infusion (excluding biopsies) or anticipation of the need for major surgery during study treatment
* Administration of a live, attenuated vaccine within 4 weeks before the start of study treatment (obinutuzumab pretreatment) or at any time during the study treatment period and within 12 months after end of study treatment
* Participants with any other diseases, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that would contraindicate the use of an investigational drug DRUG: Obinutuzumab, DRUG: Glofitamab, DRUG: Rituximab, DRUG: Ifosfamide, DRUG: Carboplatin, DRUG: Etoposide, DRUG: Tocilizumab
Mature B-Cell Non-Hodgkin Lymphoma
Relapsed, Refractory, Pediatrics, B-NHL
INTO-HLH: A Disease Registry for Patients With Hemophagocytic Lymphohistiocytosis (HLH)
Brian Hils - brian.hils@cchmc.org
ALL
NCT05277272
Inclusion Criteria:
* Patients with clinically suspected or confirmed HLH, including those meeting the HLH-2004 diagnostic criteria (primary or secondary forms, including malignancy) and other forms of HLH (macrophage activation syndrome \[MAS\], cytokine release syndrome \[CRS\], etc.)
* Signed and dated informed consent and assent (adolescents)
Exclusion Criteria:
* None Hemophagocytic Lymphohistiocytoses
Outcomes in Pediatric and Young Adult B-Cell Malignancies After Commercially Available Immunotherapy
- cancer@cchmc.org
ALL
Up to 26 years old
NCT05865301
Inclusion Criteria (Arm A)
\* Disease Status - B cell precursor acute lymphoblastic leukemia (ALL) or B cell lymphoma
Who either:
* Experienced refractory or relapsed disease, treated with standard chemotherapy, without immunotherapy treatment.
OR
* Previously undergone standard of care immunotherapy with FDA approved therapies, such as Kymriah™ (CTL019, tisagenlecleucel), blinatumomab or
* Age: Greater than or equal to 0 year of age and less than or equal to 26 years of age.
Inclusion Criteria (Arm B)
* Disease Status - B cell precursor acute lymphoblastic leukemia (ALL) or B cell lymphoma
* Age: Greater than or equal to 0 year of age and less than or equal to 26 years of age
* Patients who are either:
* Undergoing evaluation for leukapheresis for planned standard of care tisagenlecleucel therapy, or planned for therapy with blinatumomab or inotuzumab. (Patients who received prior tisagenlecleucel, blinatumomab or inotuzumab on an established clinical trial and are now scheduled for commercial CAR, blinatumomab or inotuzumab therapy are also eligible) Or
* Experienced refractory or relapsed B cell precursor acute lymphoblastic leukemia (ALL) or B cell lymphoma
* Ability to give informed consent. All subjects ≥ 18 years of age must be able to give informed consent or have legal authorized representative (LAR) (i.e. parent or guardian) to consent, if not in capacity to give consent independently. For subjects \<18 years old their LAR must give informed consent. Pediatric subjects will be included in age appropriate discussion and written assent will be obtained for those \> 7 years of age, when appropriate, according to institutional procedures.
OTHER: Questionnaire for patients receiving therapy
Lymphoid Leukemia
PRWCC, Foresight
Diaphragmatic Hernia Research & Exploration, Advancing Molecular Science (DHREAMS)
Trish Burns - trish.burns@cchmc.org
ALL
NCT00950118
Inclusion Criteria:
* All individuals affected with a congenital diaphragmatic hernia (CDH), or with a family history of a CDH
Exclusion Criteria:
* Individuals with no personal history of a CDH or family history of a family member affected with congenital diaphragmatic hernia Congenital Diaphragmatic Hernia
Congenital Diaphragmatic Hernia (CDH), Genes, Genetic, Genetic testing, exome sequencing, genome sequencing, RNAseq
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Sydney Lakes - Sydney.Lakes@cchmc.org
ALL
2 years to 11 years old
PHASE3
NCT06904261
Inclusion Criteria
* Male or female subjects, diagnosed with Fabry disease who are between ages 2 and \< 12 years at randomization (subjects aged 11 years must have birthdays \> 30 days after randomization)
* Subject's parent or legally authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable.
* Subject has a GLA variant documented in his/her medical record that is amenable to migalastat prior to Visit 2.
* Subject has not received ERT (eg, Replagal® \[agalsidase alfa\] or Fabrazyme® \[agalsidase beta\]) for at least 14 days prior to Baseline visit.
* Subject has at least 1 documented complication (ie, historical or current laboratory abnormality or sign/symptom) of Fabry disease
* If of reproductive potential, both male and female subjects agree to use a medically accepted method of contraception throughout the duration of the study and for up to 30 days after their last dose of migalastat.
Exclusion Criteria
* Has moderate or severe renal impairment (eGFR \< 60 mL/min/1.73 m2 at Visit 1 \[screening\]).
* Has advanced kidney disease requiring dialysis or kidney transplantation.
* History of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol).
* Has received any investigational/experimental drug, biologic, or device within 30 days or 5 half-lives of the investigational product (whichever is longer) before Visit 1 (screening).
* Has received any gene therapy at any time or anticipates starting gene therapy during the study period.
* Requires treatment with Glyset (miglitol) or Zavesca (miglustat), within 6 months before Visit 1(screening) or throughout the study.
* Has any intercurrent illness or condition at Visit 1 (screening) or Visit 2 (baseline) that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study.
* Pregnant or breastfeeding
* Otherwise unsuitable for the study in the opinion of the investigator
DRUG: Migalastat HCl 20 mg
Fabry Disease
migalastat, AT1001, Galafold, lysosomal disease
A Study of Selinexor in People With Wilms Tumors and Other Solid Tumors
- cancer@cchmc.org
ALL
1 year and over
PHASE2
NCT05985161
Inclusion Criteria:
* Age:
• Age ≥ 6 at the time of informed consent
• Age ≥ 2 years to \< 6 years at time of informed consent (Refer to Section 4.3): If PK cohort 1 is open, patients in this age range may enroll onto this cohort. If PK cohort 1 has been completed and deemed sufficient to proceed, then such patients may enroll onto the phase 2.
• Age ≥ 12 months to \< 2 years at time of informed consent (Refer to Section 4.3): If PK cohort 2 is open, patients in this age range may enroll onto this cohort. If PK cohort 2 has been completed and deemed sufficient to proceed, then such patients may enroll onto the phase 2. * 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. * Performance: Karnofsky ≥ 60% for patients \> 16 years of age and Lansky ≥ 60 for patients ≤ 16 years of age. * Diagnosis: Patients must enroll into one of the following cohorts:
• Cohort A: Any type of Wilms tumor or nephroblastoma is eligible for this study provided they meet at least one of these criteria: (1) in their second or greater relapse, (2) refractory or in their first relapse with high risk histology (i.e., any anaplastic or blastemal-type after neoadjuvant chemotherapy), or (3) refractory or in first relapse without high risk histology but after having received chemotherapies other than the initial 4 agents used as current standard of care in the up-front setting for non-high risk cases - specifically vincristine, dactinomycin, doxorubicin, and irinotecan (i.e., any patient who relapses following an initial regimen more intense than EE4A, DD4A, VAD, AVD, or VIVA; for example, those including cyclophosphamide/etoposide - such as Regimen I, M, or MVI - or those additionally including carboplatin - such as Regimens UH-1, UH-2, or UH-3).
• Cohort B: Any Rhabdoid tumor is eligible for this cohort. This includes, but is not limited to, related subtypes of rhabdoid tumors such as atypical teratoid rhabdoid tumors (ATRT), malignant rhabdoid tumors of the kidney (MRTK), malignant rhabdoid tumors of the soft tissue and liver, small cell undifferentiated hepatoblastomas (SCUH), and small-cell carcinoma of the ovary of hypercalcemic type (SCCOHT). Patients must have failed to respond to at least 1 line of systemic therapy prior to enrollment.
• Cohort C: Patients with progressive, relapsed, unresectable or metastatic MPNST, are eligible for this cohort. Patients must have failed to respond to at least 1 line of systemic therapy prior to enrollment.
• Cohort D: Patients must not qualify for Cohorts A, B, or C but have a solid tumor (no hematologic malignancies including lymphoma) for which there is specific evidence that this particular patient's tumor may benefit from selinexor. Patients must have failed to respond to at least 1 line of systemic therapy prior to enrollment. Examples of evidence are listed below. All patients in this cohort require approval of study principal investigator and must provide documentation of specific supporting evidence. i. Tumor XPO1 Dependency: Defined as either Darwin OncoTarget demonstrating XPO1 as aberrantly activated or Darwin OncoTreat demonstrating context-specific tumor checkpoint inversion with Selinexor, both of which must be significant at a -log10 (Bonferroni corrected p-value) of 5 or greater. ii. Tumor XPO1 Activation: Defined as the detection of a gain of function mutation in XPO1, specifically E571K. Additionally, detection of elevated transcriptomic or proteomic expression of XPO1 in the tumor via RNAseq or IHC, respectively, would be considered sufficient for treatment. iii. Preclinical Tumor Testing: Defined as testing of Selinexor on patient derived cell line, organoid, or xenograft models of the patient's tumor (or other related tumors) performed in a laboratory context and for which, in the investigator's opinion, demonstrates promising activity. Testing may include commercial testing as well as academic laboratory testing. * Cohort E: Patients must have a solid tumor with an activating genomic alteration (e.g. fusion or internal tandem duplication) involving BCOR. Specific examples of qualifying alterations including BCOR-ITD, BCOR-CCNB3, BCOR-MAML3 and ZC3H7B-BCOR; Other potentially qualifying BCOR alterations require approval of study principal investigator; note that loss of function alterations of BCOR would not qualify. * Disease Status: Patients on the phase II portion of the study must have measurable disease whereas patients on the PK cohorts can have either evaluable or measurable disease as measured by the revised Response Evaluation Criteria in Solid Tumors (RECIST) guideline (Version 1.1). a. Primary Brain Tumors: Patients with primary brain tumors are eligible and must also have measurable disease for the phase II (as well as evaluable or measurable for the PK cohorts), but this can be defined as at least equal or greater than twice the slice thickness in two perpendicular diameters on MRI OR diffuse leptomeningeal disease OR clear MRI evidence of disease that may not be measurable in two perpendicular diameters OR positive CSF cytology alone. * Prior Therapy: Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and meet minimum washout durations (shown below) from prior therapy.
• Anti-cancer agents not known to be myelosuppressive: ≥ 7 days
• Anti-cancer and cytotoxic agents known to be myelosuppressive: ≥ 21 days
• Immunotherapies (including antibodies, interleukins, interferons, etc.): ≥ 21 days
• Adoptive cellular therapies (including modified T cells, vaccines, etc.): ≥ 42 days
• Autologous stem cell infusion (boost, no conditioning): ≥ 21 days
• Autologous stem cell transplantation (with conditioning): ≥ 42 days
• Allogeneic bone marrow transplantation: ≥ 84 days
• Focal external beam radiation (e.g., limited sites of disease): ≥ 14 days
• Substantial external beam radiation (e.g. whole lung or abdomen): ≥ 42 days
• Radiopharmaceutical therapy (e.g., radiolabeled antibody or MIBG): ≥ 42 days * Hepatic Function: Adequate function (within 14 days prior to C1D1), defined as:
• Total bilirubin \< 1.5 × upper limit of normal (ULN) (except patients with Gilbert's syndrome, who must have a total bilirubin of \<3 × ULN)
• Alanine aminotransferase (ALT) \< 3 × ULN
• Serum albumin ≥ 2 g/dL * Renal Function: Adequate function (within 14 days prior to C1D1) defined as a GFR ≥ 50 ml/min/1.73 m2 determined via any of these methods:
• Nuclear radioisotope
• 24 hr urine creatinine clearance
• Serum cystatin c
• Serum creatinine using the Schwartz formula for estimating creatinine clearance (Schwartz et al. J Peds, 106:522, 1985) * Hematologic Function: Adequate function (within 14 days prior to C1D1), defined as:
• Absolute neutrophil count (ANC) ≥ 1000/mm3
• Platelet count ≥ 100,000/mm3
• Note: patients may not receive platelet transfusions nor hematopoietic growth factor support, including granulocyte-colony stimulating factor (e.g. filgrastim) and platelet stimulators (e.g. romiplostim) for at least 7 days prior to demonstrating adequate hematologic function.
Exclusion Criteria:
* Prior Therapy: Has received selinexor or another XPO1 inhibitor previously.
* Infection: Patients who have an uncontrolled infection are not eligible. Patients on prophylactic antibiotics or with a controlled infection within 1 week prior to C1D1 are acceptable
* Transplants: Patients who have received allogeneic bone marrow transplant are potentially eligible unless they are being actively treated for GvHD. Patients who have had a prior solid organ transplantation are not eligible.
* Compliance: Patients who as a result of serious medical, psychiatric, and/or social situation(s), in the opinion of the investigator, may not be able to comply with supportive care, safety monitoring, or any other key requirements of the study protocols are not eligible.
* Pregnancy and Breast-feeding: Pregnant or breast-feeding women will not be entered on this study because there is yet no available information regarding human fetal or teratogenic toxicities. Pregnancy tests must be obtained in girls who are post-menarchal.
* Contraception: Males or females of reproductive potential may not participate unless they have agreed to use two effective methods of birth control, including a medically accepted barrier or contraceptive method (e.g., male or female condom) for the duration of the study. Abstinence is an acceptable method of birth control. DRUG: Selinexor
Wilms Tumor, Rhabdoid Tumor, Malignant Peripheral Nerve Sheath Tumors, MPNST, Nephroblastoma, XPO1 Gene Mutation, Solid Tumor
Wilms Tumor, Rhabdoid Tumor, Malignant Peripheral Nerve Sheath Tumors, MPNST, Nephroblastoma, XPO1, Selinexor, Memorial Sloan Kettering Cancer Center, 22-393, Solid Tumor
Responding With Evidence and Access for Childhood Headaches
Paula LaFollette - reachstudy@cchmc.org
ALL
10 years to 17 years old
NA
NCT05889624
Inclusion Criteria:
* Diagnosis: Migraine with or without aura that meets the International Classification of Headache Disorders (ICHD) criteria 5 or presentation of continuous headache that includes migranous episodes based upon headache history obtained by site PI or designee. (includes presentation with or without medication overuse headache as well)
* Headache Frequency: 4 or more headache days based upon prospective headache diary of 28 days prior to randomization
* Amitriptyline Eligible: Site PI or medical staff determined participant to be eligible for clinical prescription of amitriptyline as a preventive treatment for migraine
* English fluency: Able to complete the study visits and questionnaires in English
Exclusion Criteria:
* Current treatment includes amitriptyline and/or CBT specific to headache care
* Current prescribed preventive antimigraine medication within a period equivalent to \< 5 half-lives of that medication before entering the baseline phase
* Current treatment with onabotulinumtoxinA (Botox) or CGRP-based monoclonal antibody medications for migraine prevention
* Youth who are pregnant
* Report of current or ongoing suicidal thoughts. Suicide attempt within the past six months. History of bipolar disorder, prolonged QT, or pre-existing significant constipation or gastroparesis
* Any and all other diagnoses or conditions which, in the opinion of the site investigator, would prevent the patient from being a suitable candidate for the study or interfere with the medical care needs of the participant BEHAVIORAL: CBT, DRUG: Amitriptyline
Headache, Headache Disorders, Headache, Migraine, Migraine, Migraine Disorders, Migraine With Aura, Migraine Without Aura, Chronic Migraine
headache, migraine, cognitive behavioral therapy, pediatrics, diaphragmatic breathing, progressive muscle relaxation, imagery, relaxation, biofeedback
Long-Term Follow-Up of Patients Who Have Participated in Children's Oncology Group Studies
- cancer@cchmc.org
ALL
NCT00736749
Inclusion Criteria:
* The patient must reside in the U.S. on the date of enrollment to ALTE05N1
* 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 PROCEDURE: Assessment of Therapy Complications, OTHER: Questionnaire Administration
Hematopoietic Cell Transplantation Recipient, Leukemia, Solid Tumor
Stress Hydrocortisone In Pediatric Septic Shock (SHIPSS)
Abigayle Gibson - abigayle.gibson@cchmc.org
ALL
1 month to 17 years old
PHASE3
NCT03401398
Inclusion Criteria:
A child receiving treatment in a pediatric intensive care unit is eligible for recruitment into SHIPSS if she/he meets all of the following inclusion criteria:
• Age is at least 1 month (with corrected gestational age ≥42 weeks), but less than 17 years and 8 months of age
• A documented focus of infection or a strong suspicion of infection at PICU admission, or for patients who develop septic shock during PICU stay, at the onset of the septic shock event
• Surveillance cultures (e.g. blood, urine, cerebral spinal fluid, wound) and/or other microbial diagnostic tests have been obtained
• One or more antimicrobials have been prescribed
• Core temperature \>38.5 C or \<36.0 C or leukocytosis or leukopenia (as defined by the local laboratory) or a left-shifted leukocyte differential (\>10% immature granulocyte forms) or a neutrophil count of \<0.5 x 109 cells per litre documented at least once within the 24 hours preceding screening
• Treatment with a continuous infusion of vasoactive-inotropic agent(s) to maintain mean or systolic arterial blood pressure above the age-appropriate target set by the treating clinician
• Administration of two or more vasoactive-inotropic agents at any dose or epinephrine or norepinephrine infusion(s) alone at greater than or equal to 0.10 mcg/kg/min for \>1 hour.
Exclusion Criteria:
A child receiving treatment in a pediatric intensive care unit for sepsis is ineligible for enrollment into SHIPSS if she/he meets any of the following exclusion criteria:
• All inclusion criteria have been present for \> 12 hours
• Attending physician expects to prescribe systemic corticosteroids for an indication other than septic shock
• Patient has received any doses of systemic corticosteroids during treatment for sepsis
• Enrolled concurrently in a competing interventional clinical trial (formal assessment to be conducted by SHIPSS Core Committee for each potential competing trial)
• Etomidate or ketoconazole treatment within past 48 hours
• Patient in whom steroids are contraindicated at time of screening (e.g. treatment for systemic fungal infection, cerebral malaria, strongyloides)
• Known or suspected hypothalamic, pituitary or adrenal disease (including patient has received acute or chronic corticosteroid administration and the physician intends to provide corticosteroid for suspected adrenal suppression)
• Attending physician, PICU care team, or legally recognized guardians not committed to full treatment and resuscitation at the time of screening
• Patient documented to be pregnant
• Previous enrollment in the SHIPSS study
• Patient admitted directly to the PICU with a thermal burn who has been in the PICU for \<72 hours prior to meeting SHIPSS inclusion criteria.
• (U.S. sites only) Patient in the custody of US protective services
• Patient being evaluated for brain death
• Vasoactive-inotropic agents prescribed solely for an indication other than septic shock
• Confirmed dengue fever
DRUG: Hydrocortisone, sodium succinate, DRUG: Normal saline
Septic Shock
hydrocortisone, refractory septic shock, sepsis, new/progressive MODS, mortality, health-related quality of life, corticosteroid adverse events, sepsis biomarkers
A Study to Find the Highest Dose of Imetelstat in Combination With Fludarabine and Cytarabine for Patients With AML, MDS or JMML That Has Come Back or Does Not Respond to Therapy
- cancer@cchmc.org
ALL
1 year to 18 years old
PHASE1
NCT06247787
Inclusion Criteria:
* Patients must be ≥ 1 year and ≤ 18 years of age at the time of study enrollment
* Patients, with or without down syndrome (DS), and with acute myeloid leukemia, therapy-related AML, MDS or JMML and meet one of the following:
* Second or greater relapse or refractory AML, including isolated extramedullary disease (EMD), but excluding isolated central nervous system (CNS) or isolated testicular relapse
* First or greater relapse of MDS
* First or greater relapse of JMML
* For flow cytometry, it's strongly recommended to enroll onto APAL2020SC or to send samples to Hematologics, Inc. Otherwise, assessments must be performed at a College of American Pathologists (CAP)/Clinical Laboratory Improvement Act (CLIA) certified lab that has expertise in AML.
* For fluorescence in situ hybridization (FISH)/Karyotype, samples must be sent to a Children's Oncology Group (COG)-approved Cytogenetics Lab
* Bone marrow relapse AML: (patients must meet one of the following criteria to be defined as having relapsed disease)
* A single bone marrow sample showing ≥ 5% leukemic blasts by flow cytometry, fluorescence in situ hybridization (FISH) testing, or other molecular method
* A single bone marrow with at least two tests showing ≥ 1% leukemic blasts; examples of tests include:
* Flow cytometry showing ≥ 1% leukemic blasts by multidimensional flow cytometry (MDF)
* Karyotypic abnormality with at least one metaphase similar or identical to diagnosis
* FISH abnormality identical to one present at diagnosis
* Polymerase chain reaction (PCR) or next generation sequencing (NGS)-based demonstration of leukemogenic lesion identical to diagnosis and ≥ 1%
* In cases where a bone marrow aspirate cannot be obtained because of extensive fibrosis, blast count can be obtained from touch imprints or estimated from an adequate bone marrow core biopsy. A complete blood count documenting the presence of at least 1,000/uL (i.e., a white blood cell \[WBC\] count ≥ 10,000/uL with ≥ 10% blasts or a WBC count of ≥ 5,000/uL with ≥ 20% blasts) circulating leukemic cells (blasts) can also be used if a bone marrow aspirate or biopsy cannot be performed
* Extramedullary relapse: Biopsy proven extramedullary disease after documented complete remission
* Refractory disease AML: Following a re-induction cycle after a second relapse, or refractory to two reinduction attempts after either primary induction failure or first relapse with:
* A single bone marrow sample showing ≥ 5% leukemic blasts by flow cytometry, fluorescence in situ hybridization (FISH) testing, or other molecular method
* A single bone marrow with at least two tests showing ≥ 1% leukemic blasts: examples of tests include:
* Flow cytometry showing ≥ 1% leukemic blasts by multidimensional flow cytometry (MDF)
* Karyotypic abnormality with at least one metaphase similar or identical to diagnosis.
* FISH abnormality identical to one present at diagnosis
* Polymerase chain reaction (PCR) or next generation sequencing (NGS)-based demonstration of leukemogenic lesion identical to diagnosis and ≥ 1%
* In cases where a bone marrow aspirate cannot be obtained because of extensive fibrosis, blast count can be obtained from touch imprints or estimated from an adequate bone marrow core biopsy. A complete blood count documenting the presence of at least 1,000/uL (i.e., a WBC count ≥ 10,000/uL with ≥ 10% blasts or a WBC count of ≥ 5,000/uL with ≥ 20% blasts) circulating leukemic cells (blasts) can also be used if a bone marrow aspirate or biopsy cannot be performed
* Extramedullary refractory disease:
* Biopsy proven persistent extramedullary disease
* In cases where a bone marrow aspirate cannot be obtained because of extensive fibrosis, blast count can be obtained from touch imprints or estimated from an adequate bone marrow core biopsy. A complete blood count documenting the presence of at least 1,000/ µL (i.e., a WBC count ≥ 10,000/μL with ≥ 10% blasts or a WBC count of ≥ 5,000/μL with ≥ 20% blasts) circulating leukemic cells (blasts) can also be used if a bone marrow aspirate or biopsy cannot be performed
* Central nervous system disease: Patients with relapsed or refractory disease with central nervous system (CNS) 1 and CNS 2 status are eligible
* MDS: Bone marrow relapse: (patients must meet one of the following criteria to be defined as having relapsed disease)
* A single bone marrow sample showing ≥ 5% leukemic blasts by flow cytometry, FISH, or other molecular method
* A single bone marrow with at least two tests showing ≥ 1% leukemic blasts; examples of tests include:
* Flow cytometry showing ≥ 1% leukemic blasts by multidimensional flow cytometry (MDF)
* Karyotypic abnormality with at least one metaphase similar or identical to diagnosis
* FISH abnormality identical to one present at diagnosis
* Polymerase chain reaction (PCR) or next generation sequencing (NGS)-based demonstration of MDS associated lesion identical to diagnosis and ≥ 1%
* In cases where a bone marrow aspirate cannot be obtained because of extensive fibrosis, blast count can be obtained from touch imprints or estimated from an adequate bone marrow core biopsy
* JMML: Diagnosis: Patients must have had histologic verification of juvenile myelomonocytic leukemia (JMML) at original diagnosis and currently have relapsed or refractory disease. The diagnosis is made based on the following criteria
* JMML category 1 (all of the following):
* Splenomegaly
* \> 1000 (1 x 10\^9 /uL) circulating monocytes
* \< 20% Blasts in the bone marrow or peripheral blood
* Absence of the t(9;22) or BCR/ABL fusion gene
* The diagnostic criteria must include all features in category 1 and either (i) one of the features in category 2 or (ii) two features from category 3 to make the diagnosis
* JMML category 2 (at least one of the following if at least two category 3 criteria are not present):
* Somatic mutation in RAS or PTPN11
* Clinical diagnosis of NF1 or NF1 gene mutation
* Homozygous mutation in CBL
* Monosomy 7
* JMML category 3 (at least two of the following if no category 2 criteria are met):
* Circulating myeloid precursors
* White blood cell count, \> 10,000 (10 x 10\^9 / uL)
* Increased hemoglobin F for age
* Clonal cytogenetic abnormality
* Granulocyte-macrophage-colony-stimulating factor (GM-CSF) hypersensitivity
* Patients with relapsed JMML must have had at least one cycle of intensive frontline therapy or at least 2 cycles of a deoxyribonucleic acid (DNA) hypomethylating agent with persistence of disease, defined by clinical symptoms or the presence of a clonal abnormality. Frontline therapy is defined as one cycle of intravenous chemotherapy that includes of any of the following agents: fludarabine, cytarabine, or any anthracycline but specifically excludes oral 6-mercaptopurine. Frontline therapy will also include any conditioning regimen as part of a stem cell transplant. Patients who transform to AML at any point with more than 20% blasts are eligible for this trial per the AML specific criteria
* Patient's current disease state must be one for which there is no known curative therapy or therapy proven to prolong survival with an acceptable quality of life
* Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2. Use Karnofsky (≥ 50) for patients \> 16 years of age and Lansky for patients ≤ 16 years of age (≥ 50)
* Patients must have fully recovered (grade \< 2) from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. If after the required time frame, the numerical eligibility criteria are met, e.g. blood count criteria, the patient is considered to have recovered adequately
* NOTE: IT therapy does not require a washout period
* Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive: See DVL homepage on the COG Members site for commercial and investigational agent classifications (https://cogmembers.org/uploadedFiles/Site/Disc/DVL/Documents/TableOfMyelosuppressiveAnti-CancerAgents.pdf). For agents not listed, the duration of this interval must be discussed with the study chair and the study-assigned research coordinator prior to enrollment
* ≥ 14 days must have elapsed after the completion of other cytotoxic therapy, with the exception of hydroxyurea, for patients not receiving standard maintenance therapy. Additionally, patients must have fully recovered from all acute toxic effects of prior therapy
* Note: Cytoreduction with hydroxyurea must be discontinued ≥ 24 hours prior to the start of protocol therapy
* Anti-cancer agents not known to be myelosuppressive (e.g. not associated with reduced platelet or absolute neutrophil \[ANC\] counts):
* ≥ 7 days after the last dose of agent. See the DVL homepage on the COG Members site for commercial and investigational agent classifications. For agents not listed, the duration of this interval must be discussed with the study chair and the study-assigned research coordinator prior to enrollment
* Antibodies: ≥ 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade ≤ 1
* Hematopoietic growth factors: ≥ 14 days after the last dose of a long-acting growth factor (e.g. pegfilgrastim) or 7 days for short-acting growth factor. 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
* Interleukins, interferons and cytokines (other than hematopoietic growth factors): ≥ 21 days after the completion of interleukins, interferon, or cytokines (other than hematopoetic growth factors)
* Stem cell Infusions (with or without total body irradiation \[TBI\]):
* Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion (DLI) or boost infusion: ≥ 84 days after infusion and no evidence of graft-versus-host disease (GVHD)
* Patients must be off calcineurin inhibitors for at least 28 days prior to the date of enrollment. Patients may be on physiological doses of steroids (equivalent to ≤ 10 mg prednisone daily for patients ≥ 18 years or ≤ 10mg/m\^2/day \[up to a maximum of 10 mg/day\] for patients \< 18 years)
* Autologous stem cell infusion including boost infusion: ≥ 30 days
* Cellular Therapy: ≥ 30 days after the completion of any type of cellular therapy (eg, modified T cells, natural killer \[NK\] cells, dendritic cells, etc.)
* External Beam Radiation (XRT)/external beam irradiation including protons: ≥ 14 days after local XRT; ≥ 150 days after TBI, craniospinal XRT or if radiation to ≥ 50% of the pelvis; ≥ 42 days if other substantial bone marrow (BM) radiation
* Radiopharmaceutical therapy (eg, radiolabeled antibody, 131I MIBG): ≥ 42 days after systemically administered radiopharmaceutical therapy
* Patients must not have received prior exposure to imetelstat
* For patients with leukemia:
* Platelet count ≥ 25,000/uL (may receive platelet transfusions). These patients must not be known to be refractory to red cell or platelet transfusion
* Hemoglobin \>= 8.0 g/dL at baseline (may receive red blood cell \[RBC\] transfusions)
* Adequate renal function defined as:
* Estimated glomerular filtration rate (GFR) (eGFR) ≥ 70 mL/min/1.73 m\^2 OR
* a 24 hour urine creatinine clearance ≥ 70 mL/min/1.73 m\^2 OR
* a GFR ≥ 70 mL/min/1.73 m\^2. 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)
* Adequate liver function defined as:
* Bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x upper limit of normal (ULN) for age
* Serum glutamic-pyruvic transaminase (SGPT) (alanine aminotransferase \[ALT\]) ≤ 3 x ULN, unless attributed to leukemia involvement
* AST ≤ 3 x ULN, unless attributed to leukemia involvement
* Albumin ≥ 2 g/dL
* Shortening fraction of ≥ 27% by echocardiogram, or ejection fraction of ≥ 50% by gated radionuclide study
Exclusion Criteria:
* Pregnant or breast-feeding women will not be entered on this study due to risks of fetal and teratogenic adverse events as seen in animal/human studies, or because there is yet no available information regarding human fetal or teratogenic toxicities. 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 two effective methods of birth control, including a medically accepted barrier or contraceptive method (eg, male or female condom) for the duration of the study. Abstinence is an acceptable method of birth control. Women of childbearing potential must use highly effective contraception in addition to a barrier method during treatment and for at least 1 month after the last dose of imetelstat or longer if required by the institutional guidelines for conventional chemotherapy (fludarabine/cytarabine). Male patients who can father a child should use contraception during treatment and for 3 months after the last dose of imetelstat or longer if required by the institutional guidelines for conventional chemotherapy (fludarabine/cytarabine). Imetelstat should not be administered to nursing mothers
* 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. Patients may be on physiological doses of steroids (equivalent to ≤ 10 mg prednisone daily for patients ≥ 18 years or ≤ 10mg/m\^2/day \[up to a maximum of 10 mg/day\] for patients \< 18 years). If used to modify immune adverse events related to prior therapy, ≥ 14 days must have elapsed since last dose of corticosteroid
* 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 except patients receiving hydroxyurea, which may be continued until 24 hours prior to start of protocol therapy
* Anti-GVHD agents post-transplant: Patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial
* Specific to MDS patients: Low-grade MDS/refractory cytopenia of childhood (RCC) - MDS with less than 2% blasts in peripheral blood (PB) or less than 5% blasts in the bone marrow (BM) by morphology
* Uncontrolled seizure disorder that is not stabilized with anti-convulsants
* Patient has undergone surgery that requires general anesthesia within 3 weeks before enrollment (line placement/removal/revision or tissue collection is allowed)
* Known hypersensitivity to the study drug or excipients of the preparation
* Patients with acute promyelocytic leukemia (APL) with PML-RARA genetic abnormality according to World Health Organization (WHO) classification or t(15;17) are not eligible
* Patients known to have a congenital bone marrow failure syndrome where increased risk of toxicity may be expected as judged by the Investigator, for example dyskeratosis congenita, are not eligible
* Patients with isolated or refractory CNS or isolated or refractory testicular relapse are not eligible
* Patients who have an uncontrolled 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 PROCEDURE: Biospecimen Collection, PROCEDURE: Bone Marrow Aspiration, PROCEDURE: Bone Marrow Biopsy, DRUG: Cytarabine, PROCEDURE: Echocardiography Test, DRUG: Fludarabine, DRUG: Hydrocortisone Sodium Succinate, BIOLOGICAL: Imetelstat, DRUG: Leucovorin Calcium, PROCEDURE: Lumbar Puncture, DRUG: Methotrexate
Recurrent Childhood Acute Myeloid Leukemia, Recurrent Childhood Myelodysplastic Syndrome, Recurrent Juvenile Myelomonocytic Leukemia, Refractory Childhood Acute Myeloid Leukemia, Refractory Childhood Myelodysplastic Syndrome, Refractory Juvenile Myelomonocytic Leukemia
A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma
- cancer@cchmc.org
ALL
2 years to 21 years old
PHASE3
NCT04166409
Inclusion Criteria:
* Patients must be \>= 2 years and =\< 21 years at the time of enrollment
* Patients must have a body surface area (BSA) of \>= 0.5 m\^2 at enrollment
* Patients must have non-neurofibromatosis type 1 (non-NF1) low-grade glioma (LGG) without a BRAFV600E mutation as confirmed by Rapid Central Pathology and Molecular Screening Reviews performed on APEC14B1 (NCT02402244) Childhood Cancer Data Initiative (CCDI)-MCI, or accepted Clinical Laboratory Improvement Act (CLIA)-certified test and that has not been treated with any modality besides surgery. Note: Patients may be newly-diagnosed OR previously diagnosed, and there is no required time frame between biopsy/surgery and treatment initiation.
* Patients with residual tumor after resection or progressive tumor after initial diagnosis (with or without surgery) who have not received treatment (chemotherapy and/or radiation) are eligible
* Patients must have two-dimensional measurable tumor \>= 1 cm\^2 to be eligible
* Patients with ependymoma are not eligible
* Eligible histologies will include all tumors considered low-grade glioma or low-grade astrocytoma (World Health Organization \[WHO\] grade I and II) by 5th edition WHO classification of central nervous system (CNS) tumors with the exception of subependymal giant cell astrocytoma
* Patients with metastatic disease or multiple independent primary LGG are eligible
* Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 OR a serum creatinine based on age/sex as follows (performed within 7 days prior to enrollment):
* Age: Maximum Serum Creatinine (mg/dL)
* 2 to \< 6 years: 0.8 mg/dL (male); 0.8 mg/dL (female)
* 6 to \< 10 years: 1 mg/dL (male); 1 mg/dL (female)
* 10 to \< 13 years: 1.2 mg/dL (male); 1.2 mg/dL (female)
* 13 to \< 16 years: 1.5 mg/dL (male); 1.4 mg/dL (female)
* \>= 16 years: 1.7 mg/dL (male); 1.4 mg/dL (female)
* Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age (performed within 7 days prior to enrollment) (children with a diagnosis of Gilbert's syndrome will be allowed on study regardless of their total and indirect \[unconjugated\] bilirubin levels as long as their direct \[conjugated\] bilirubin is \< 3.1 mg/dL)
* Serum glutamic pyruvic transaminase (SGPT) (alanine aminotransferase \[ALT\]) =\< 135 U/L (performed within 7 days prior to enrollment). For the purpose of this study, the ULN for SGPT is 45 U/L
* Albumin \>= 2 g/dL (performed within 7 days prior to enrollment)
* Left ventricular ejection fraction (LVEF) \>= 53% (or institutional normal; if the LVEF result is given as a range of values, then the upper value of the range will be used) by echocardiogram (performed within 4 weeks prior to enrollment)
* Corrected QT (QTc) interval =\< 450 msec by electrocardiography (EKG) (performed within 4 weeks prior to enrollment)
* Absolute neutrophil count \>= 1,000/uL (unsupported) (performed within 7 days prior to enrollment)
* Platelets \>= 100,000/uL (unsupported) (performed within 7 days prior to enrollment)
* Hemoglobin \>= 8 g/dL (may be supported) (performed within 7 days prior to enrollment)
* Patients with a known seizure disorder must be stable and must not have experienced a significant increase in seizure frequency within 2 weeks prior to enrollment
* Patients 2-17 years of age must have a blood pressure that is =\< 95th percentile for age, height, and sex at the time of enrollment (with or without the use of anti-hypertensive medications)
* Patients \>= 18 years of age must have a blood pressure =\< 130/80 mmHg at the time of enrollment (with or without the use of anti-hypertensive medications)
* Note for patients of all ages: Adequate blood pressure can be achieved using medication for the treatment of hypertension
* All patients must have ophthalmology toxicity assessments performed within 8 weeks prior to enrollment
* For all patients, an MRI of the brain (with orbital cuts for optic pathway tumors) and/or spine (depending on the site(s) of primary disease) with and without contrast must be performed within 8 weeks prior to enrollment
* 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 must have the ability to swallow whole capsules
* 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
* All patients have signed an appropriate consent form and Health Insurance Portability and Accountability Act (HIPAA) authorization form (if applicable)
* All patients have been consented and enrolled on APEC14B1 (NCT02402244) Part A for Pre-Enrollment Eligibility Screening for ACNS1833
Exclusion Criteria:
* Patients must not have received any prior tumor-directed therapy including chemotherapy, radiation therapy, immunotherapy, or bone marrow transplant. Prior surgical intervention (with the exclusion of laser interstitial thermal therapy \[LITT\]) is permitted
* Patients with a concurrent malignancy or history of treatment (other than surgery) for another tumor within the last year are ineligible
* Patients with diffuse intrinsic pontine tumors as seen on MRI (\> 2/3 of pons involvement on imaging) are not eligible even if biopsy reveals grade I/II histology
* Patients may not be receiving any other investigational agents
* Patients with any serious medical or psychiatric illness/condition, including substance use disorders or ophthalmological conditions, likely in the judgment of the investigator to interfere or limit compliance with study requirements/treatment
* Patients who, in the opinion of the investigator, are not able to comply with the study procedures are not eligible
* Female patients who are pregnant are not eligible 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 are not eligible
* Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation and for 1 week after stopping study therapy are not eligible.
* Note: Women study participants of child-bearing potential must use acceptable contraception during the study and for 1 week (7 days) after the last dose of selumetinib. Men study participants with sexual partners who are pregnant or who are of child-bearing potential must use acceptable contraception during the study and for 1 week (7 days) after the last dose of study agent. Acceptable contraception includes implants, injectables, or oral contraceptives (all combined with barrier methods), some intrauterine devices (IUDs), vasectomy or abstinence
* Known genetic disorder that increases risk for coronary artery disease. Note: The presence of dyslipidemia in a family with a history of myocardial infarction is not in itself an exclusion unless there is a known genetic disorder documented
* Symptomatic heart failure
* New York Health Association (NYHA) class II-IV prior or current cardiomyopathy
* Severe valvular heart disease
* History of atrial fibrillation
* Current or past history of central serous retinopathy
* Current or past history of retinal vein occlusion or retinal detachment
* Patients with uncontrolled glaucoma
* If checking pressure is clinically indicated, patients with intraocular pressure (IOP) \> 22 mmHg or ULN adjusted by age are not eligible
* Supplementation with vitamin E greater than 100% of the daily recommended dose. Any multivitamin containing vitamin E must be stopped prior to study enrollment even if less than 100% of the daily recommended dosing for vitamin E
* Surgery within 2 weeks prior to enrollment, with the exception of surgical biopsy, placement of a vascular access device or cerebral spinal fluid (CSF) diverting procedure such as endoscopic third ventriculostomy (ETV) and ventriculoperitoneal (VP) shunt.
* Note: Patients must have healed from any prior surgery
* Patients who have an uncontrolled infection are not eligible PROCEDURE: Biospecimen Collection, DRUG: Carboplatin, PROCEDURE: Echocardiography Test, PROCEDURE: Magnetic Resonance Imaging, OTHER: Questionnaire Administration, DRUG: Selumetinib Sulfate, DRUG: Vincristine Sulfate
Low Grade Astrocytoma, Low Grade Glioma, Metastatic Low Grade Astrocytoma, Metastatic Low Grade Glioma, WHO Grade 1 Glioma, WHO Grade 2 Glioma
A Study to Compare Blinatumomab Alone to Blinatumomab With Nivolumab in Patients Diagnosed With First Relapse B-Cell Acute Lymphoblastic Leukemia (B-ALL)
- cancer@cchmc.org
ALL
1 year to 30 years old
PHASE2
NCT04546399
Inclusion Criteria:
* Patients must be \>= 1 and \< 31 years at time of enrollment
* Patients must have first relapse of CD19+ B-ALL (relapse blasts must express CD19) in one of the following categories:
* Isolated bone marrow relapse
* Isolated central nervous system (CNS) (excluding known optic nerve/retinal and CNS chloromas) and/or testicular relapse
* Combined bone marrow with extramedullary relapse in the CNS (excluding known optic nerve/retinal and CNS chloromas) and/or testes
* Patients with Down syndrome (DS) are eligible in the following categories:
* Isolated bone marrow relapse
* Combined bone marrow with CNS (excluding known optic nerve/retinal and CNS chloromas) and/or testicular relapse
* 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
* Of note, for patients with developmental delay (e.g., Down syndrome) regardless of age, Lansky scale may be substituted for Karnofsky scale. However, the requirement for ECOG 0-2 remains, regardless of known history of developmental delay
* Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study
* Patients with prior blinatumomab or CD19+ chimeric antigen receptor therapy in the upfront setting will be eligible, provided relapsed lymphoblasts retain CD19 expression
* Patients must not have had a prior hematopoietic stem cell transplant
* A single intrathecal chemotherapy at the time of relapse will be allowed. If \< 7 days have elapsed between this intrathecal therapy (IT) and the start of protocol therapy, then the day 1 intrathecal chemotherapy (i.e. methotrexate, cytarabine, or triple intrathecal) may be omitted
* In the 28 days prior to enrollment, up to five days of post-relapse, pre-enrollment therapy (steroids and/or hydroxyurea only) is permissible
* Patients with Down syndrome who received pre-enrollment therapy and have a white blood count (WBC) \>= 30,000/ul at the time of enrollment still must receive protocol specified cytoreductive therapy with vincristine and dexamethasone, and no "washout" is required
* Patients with Down syndrome who received pre-enrollment therapy and have a WBC \< 30,000/ul at the time of enrollment must be given a 24 hour "washout" before starting immunotherapy
* Note: There is no waiting period or "washout" for patients who relapse while receiving upfront therapy
* Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 OR a serum creatinine based on age/sex as follows (within 7 calendar days prior to enrollment):
* Age: Maximum serum creatinine (mg/dL)
* 1 to \< 2 years: 0.6 (male), 0.6 (female)
* 2 to \< 6 years: 0.8 (male), 0.8 (female)
* 6 to \< 10 years: 1 (male), 1 (female)
* 10 to \< 13 years: 1.2 (male), 1.2 (female)
* 13 to \< 16 years: 1.5 (male), 1.4 (female)
* \>= 16 years: 1.7 (male), 1.4 (female)
* The threshold creatinine values in this Table were derived from the Schwartz formula for estimating GFR utilizing child length and stature data published by the Center for Disease Control (CDC)
* Shortening fraction of \>= 27% by echocardiogram, or ejection fraction of \>= 50% by echocardiogram, cardiac magnetic resonance imaging (MRI) or radionuclide angiogram
* No evidence of dyspnea at rest, no exercise intolerance, and a pulse oximetry \> 94% if there is clinical indication for determination
* 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 B-lymphoblastic lymphoma (B-LLy)
* Patients with Burkitt leukemia/lymphoma or mature B-cell leukemia
* Patients with Philadelphia chromosome positive (Ph+) B-ALL or ABL class Ph-like B-ALL (i.e. rearrangements involving ABL1, ABL2, CSF1R or PDGFRB and predicted to be sensitive to imatinib or dasatinib)
* Patients with mixed phenotype acute leukemia (MPAL)
* Patients with known Charcot-Marie-Tooth disease
* Patients with known MYC translocation associated with mature (Burkitt) B-cell ALL, regardless of blast immunophenotype
* Patients with active, uncontrolled infection defined as:
* Positive bacterial blood culture within 48 hours of study enrollment
* Receiving IV or PO antibiotics for an infection with continued signs or symptoms. Note: Patients may be receiving IV or oral antibiotics to complete a course of therapy for a prior documented infection if cultures have been negative for at least 48 hours and signs or symptoms of active infection have resolved. For patients with clostridium (C.) difficile diarrhea, at least 72 hours of antibacterial therapy must have elapsed and stools must have normalized to baseline.
* Fever above 38.2 degrees Celsius (C) within 48 hours of study enrollment with clinical signs of infection. Fever without clinical signs of infection that is attributed to tumor burden is allowed if blood cultures are negative for \> 48 hours
* A positive fungal culture within 30 days of study enrollment or active therapy for presumed invasive fungal infection
* Active viral or protozoal infection requiring IV treatment
* Patients known to have one of the following concomitant genetic syndromes: Bloom syndrome, ataxia-telangiectasia, Fanconi anemia, Kostmann syndrome, Shwachman syndrome or any other known bone marrow failure syndrome are not eligible.
* Patients with uncontrolled HIV, hepatitis B, or hepatitis C infection. Of note, patients with known human immunodeficiency virus (HIV) infection on effective anti-retroviral therapy with undetectable viral load for at least the last 6 months prior to enrollment are eligible. Similarly, hepatitis B and hepatitis C positive patients who have been treated and have no viral detectable burden are also eligible
* Patients with significant central nervous system pathology that would preclude treatment with blinatumomab, including history of severe neurologic disorder or autoimmune disease with CNS involvement
* Note: Patients with a history of seizures that are well controlled on stable doses of anti-epileptic drugs are eligible Patients with a history of cerebrovascular ischemia/hemorrhage with residual deficits are not eligible. Patients with a history of cerebrovascular ischemia/hemorrhage remain eligible provided all neurologic deficits have resolved
* Patients with an active known/suspected autoimmune disease are not eligible. However, patients with type I diabetes mellitus, hypothyroidism only requiring hormone replacement, skin disorders (such as vitiligo, psoriasis, or alopecia) not requiring systemic treatment, or conditions not expected to recur in the absence of an external trigger are permitted to enroll
* Patients with DS patients with known non-hematopoietic, non-CNS/testicular extramedullary disease (i.e., chloromatous disease) are not eligible
* Note: Group 3 and 4 patients with known non-hematopoietic, non-CNS/testicular extramedullary disease (i.e., chloromatous disease) are eligible if this is NOT the only site of relapsed disease
* Female patients of childbearing potential are not eligible unless a negative pregnancy test result has been obtained within 7 days prior to enrollment. Patients who are sexually active and of reproductive potential are not eligible unless they agree to use an effective contraceptive method for the duration of this study. Men with female partners of childbearing potential should use effective contraception during the duration of their treatment. The effect of blinatumomab on fertility has not been evaluated. Blinatumomab is not recommended for pregnant women or women of childbearing potential (WOCBP) not using contraception. Females of reproductive potential must use effective contraception during treatment and for at least 48 hours after the last dose of blinatumomab. Studies in animal models have shown that nivolumab can adversely impair pregnancy. Thus, nivolumab is expected to cause fetal harm during pregnancy. WOCBP receiving nivolumab must continue contraception for a period of at least 5 months after the last dose of nivolumab. It is unknown whether nivolumab is present in breast milk, thus breastfeeding should be discontinued while a patient is receiving nivolumab
* Lactating females are not eligible unless they agree to not breastfeed their infants. It is unknown whether blinatumomab or its metabolites are excreted in human breast milk. Women are not permitted to breastfeed while receiving blinatumomab and for the last 48 hours after the last blinatumomab dose. Due to the potential for serious adverse reactions in the breastfed infant, women are not permitted to breastfeed during treatment and for 5 months after the last nivolumab dose RADIATION: 3-Dimensional Conformal Radiation Therapy, DRUG: Asparaginase Erwinia chrysanthemi, PROCEDURE: Biospecimen Collection, BIOLOGICAL: Blinatumomab, PROCEDURE: Bone Marrow Aspiration, PROCEDURE: Bone Marrow Biopsy, DRUG: Calaspargase Pegol, DRUG: Cytarabine, DRUG: Dexamethasone, DRUG: Hydrocortisone Sodium Succinate, PROCEDURE: Lumbar Puncture, DRUG: Mercaptopurine, DRUG: Methotrexate, BIOLOGICAL: Nivolumab, DRUG: Pegaspargase, DRUG: Vincristine Sulfate
Down Syndrome, Recurrent B Acute Lymphoblastic Leukemia
Data and Sample Collection Study to Elucidate the Mechanisms of Eosinophilic Disorders
Bliss Magella - bliss.magella@cchmc.org
ALL
NCT00267501
Inclusion Criteria:
* Signed informed consent obtained from the patient or parent/guardian. Assent will be obtained from all minors 11 years of age and older.
* Carrying a diagnosis of eosinophilic gastrointestinal disease, eosinophilic inflammatory disease, or food allergy OR family member, or normal control Eosinophilic Gastrointestinal Disease Eosinophilic Inflammatory Disease, Food Allergy
Eosinophilic Disorders
The Congenital Dyserythropoietic Anemia Registry (CDAR)
Theodosia Kalfa - theodosia.kalfa@cchmc.org
ALL
NCT02964494
Inclusion Criteria:
* Diagnosis of Congenital Dyserythropoietic Anemia (CDA), whether a genetic mutation is identified or not
* Evidence of congenital anemia/jaundice or a positive family history
* Evidence of ineffective erythropoiesis
* Typical morphological appearance of bone marrow erythroblasts
* All ages (ages 0-99)
Exclusion Criteria:
* Diagnosis of cancer
* Myelodysplasia
* Secondary dyserythropoiesis: e.g.; vitamin B12 deficiency or drug-related.
Note1: Patients with rare band 3 (SLC4A1) mutations recently described to be associated with dyserythropoiesis will be eligible since the mechanisms appear to involve direct participation of band 3 in the erythroblast mitosis and cytokinesis.
Note2: Siblings, parents, and family members of patients with confirmed CDA diagnosis are encouraged to participate in the study. Congenital Dyserythropoietic Anemia (CDA)
Testing the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL)
- cancer@cchmc.org
ALL
365 days to 30 years old
PHASE3
NCT03126916
Inclusion Criteria:
* FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Patients must be enrolled on ANBL00B1 (NCT00904241) or APEC14B1 (NCT02402244) prior to enrollment on ANBL1531 (NCT03126916)
* FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Patient must be \>= 365 days and =\< 30 years of age at diagnosis
* FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Patients must have a diagnosis of neuroblastoma or ganglioneuroblastoma (nodular) verified by tumor pathology analysis or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites; the following disease groups are eligible:
* Patients with International Neuroblastoma Risk Group (INRG) stage M disease are eligible if found to have either of the following features:
* MYCN amplification (\> 4-fold increase in MYCN signals as compared to reference signals), regardless of additional biologic features; OR
* Age \> 547 days regardless of biologic features
* Patients with INRG stage MS disease with MYCN amplification
* Patients with INRG stage L2 disease with MYCN amplification
* Patients \> 547 days of age initially diagnosed with INRG stage L1, L2 or MS disease who progressed to stage M without prior chemotherapy may enroll within 4 weeks of progression to stage M
* Patients \>= 365 days of age initially diagnosed with MYCN amplified INRG stage L1 disease who progress to stage M without systemic therapy may enroll within 4 weeks of progression to stage M
* FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Patients initially recognized to have high-risk disease must have had no prior systemic therapy (other than topotecan/cyclophosphamide initiated on an emergent basis and within allowed timing); patients observed or treated with a single cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (e.g., as per ANBL0531, ANBL1232 or similar) for what initially appeared to be non-high risk disease but subsequently found to meet the criteria will also be eligible; patients who receive localized emergency radiation to sites of life-threatening or function-threatening disease prior to or immediately after establishment of the definitive diagnosis will be eligible
* FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 or a serum creatinine based on age/sex as follows:
* 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
* FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age, and
* FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) \< 10 x ULN; for the purposes of this study, ULN for SGPT (ALT) is 45
* FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Shortening fraction of \>= 27% by echocardiogram, or ejection fraction of \> 50% by echocardiogram or radionuclide angiogram
* FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: No known contraindication to peripheral blood stem cell (PBSC) collection; examples of contraindications might be a weight or size less than the collecting institution finds feasible, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and/or the apheresis procedure
* PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): See ANBL2131 (NCT06172296) protocol for eligible high-risk neuroblastoma diagnoses
* PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): In addition, all patients transferring from ANBL2131 (NCT06172296) to ANBL1531 (NCT03126916) Arm E must have tumors with an ALK aberration
* PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Given the lack of data with lorlatinib in infant populations, patients transferring from ANBL2131 (NCT06172296) to ANBL1531 (NCT03126916) must be \> 1 year of age at time of transfer to ANBL1531 (NCT03126916). Patients \< 1 year of age found to have a qualifying ALK alteration as part of ANBL2131 (NCT06172296) may continue to participate in ANBL2131 (NCT06172296)
* PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Patients initially recognized to have high-risk disease must have received no more than one cycle of topotecan/cyclophosphamide either after enrollment to ANBL2131 (NCT06172296) or started emergently prior to enrollment to ANBL2131 (NCT06172296)
* PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Patients may have received up to one cycle of intermediate risk chemotherapy prior to initial enrollment to ANBL2131 (NCT06172296)
* PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Patients may have received localized emergency radiation to sites of life-threatening or function-threatening disease prior to or immediately after establishment of the definitive diagnosis
* PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): In order to facilitate patient transfer and ensure timely distribution of lorlatinib, there are no blood count requirements to meet at time of transfer from ANBL2131 (NCT06172296) to ANBL1531 ((NCT03126916) Arm E. Note the blood count criteria that must be met prior to start of Induction cycle 2 on Arm E. Lorlatinib therapy should start no sooner than day 1 of Induction cycle 2
* PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): No known irreversible grade 2 or greater atrioventricular (AV) block
* PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Due the potential psychiatric risks from lorlatinib, patients should not have a personal history of a serious psychiatric disorder requiring pharmacologic intervention or severe enough to be considered life-threatening
* PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): No known contraindication to PBSC collection. Examples of contraindications might be a weight or size less than the collecting institution deems feasible, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and/or the apheresis procedure
Exclusion Criteria:
* FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Patients with INRG stage L2 tumors without amplification of MYCN regardless of tumor histology (may meet criteria for high risk classification but are not eligible for this trial)
* FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Patients with bone marrow failure syndromes
* FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Patients for whom targeted radiopharmaceutical therapy would be contraindicated due to underlying medical disorders
* FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: 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
* FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Lactating females who plan to breastfeed their infants
* FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
* PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Patients who have previously received treatment with lorlatinib or other ALK inhibitor
* PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Patients who have undergone treatment arm randomization callback or started induction cycle 2 on ANBL2131 (NCT06172296)
* PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Patients who have an INRG Stage L2 tumor without amplification of MYCN regardless of tumor histology (may meet criteria for high risk classification but are not eligible for this trial)
* PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Patients with bone marrow failure syndromes
* PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): 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
* PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Lactating females who plan to breastfeed their infants
* PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation PROCEDURE: Autologous Hematopoietic Stem Cell Transplantation, PROCEDURE: Biospecimen Collection, PROCEDURE: Bone Marrow Aspiration and Biopsy, DRUG: Busulfan, DRUG: Carboplatin, DRUG: Cisplatin, PROCEDURE: Computed Tomography, DRUG: Cyclophosphamide, DRUG: Dexrazoxane Hydrochloride, BIOLOGICAL: Dinutuximab, DRUG: Doxorubicin Hydrochloride, PROCEDURE: Echocardiography Test, DRUG: Etoposide Phosphate, RADIATION: External Beam Radiation Therapy, RADIATION: Iobenguane I-123, RADIATION: Iobenguane I-131, DRUG: Isotretinoin, DRUG: Lorlatinib, PROCEDURE: Magnetic Resonance Imaging, DRUG: Melphalan Hydrochloride, PROCEDURE: Multigated Acquisition Scan, PROCEDURE: Positron Emission Tomography, BIOLOGICAL: Sargramostim, PROCEDURE: Therapeutic Conventional Surgery, DRUG: Thiotepa, DRUG: Topotecan Hydrochloride, DRUG: Vincristine Sulfate
Ganglioneuroblastoma, Ganglioneuroblastoma, Nodular, Neuroblastoma
A Study With Eptinezumab in Children and Adolescents (6 to 17 Years) With Chronic or Episodic Migraine (REJOIN)
Michelle Shin - michelle.shin@cchmc.org
ALL
6 years to 17 years old
PHASE3
NCT05164172
Inclusion Criteria:
\- The participant must have completed Week12 (completion) visit of either Study19356A (CM) or Study19357A (EM) immediately prior to enrolment into this OLE study.
Exclusion Criteria:
* The participant has an adverse event or other safety concerns that are deemed related to double-blind treatment received in the lead-in study and is considered a potential safety risk by the investigator.
* During lead-in Study19356A or Study19357A:
* participant experienced ananaphylactic reaction or another severe and/or serious hypersensitivity reaction to the investigational medicinal product (IMP) infusion, as assessed by the investigator
* the participant had a serum alanine aminotransferase (ALT) or aspartate aminotransferase(AST) value \>5 times the upper limit of the reference range that was confirmed by testing \<2 weeks later.
* the participant had a serum ALT or AST value \>3times the upper limit of the reference range and a serum total bilirubin value \>2times the upper limit of the reference range. DRUG: Eptinezumab
Migraine
Long-term Safety Study of Rimegepant in Pediatric Subjects for the Acute Treatment of Migraine
Michelle Michelle Shin - Michelle.Shin@cchmc.org
ALL
6 years to 17 years old
PHASE3
NCT04743141
Inclusion Criteria:
• History of migraine (with or without aura) for ≥ 6 months before Screening.
• History of 1 to 8 moderate or severe attacks per month during the 2 months prior to screening.
• 1 or more migraine days requiring treatment during the Observation Phase.
• Prophylactic migraine medication is permitted if the dose has been stable for at least 12 weeks prior to the Screening Visit.
• Ability to distinguish between migraine and other types of headaches.
• Weight \> 15 kg. For EU countries only: Participants 12 to \< 18 years of age must have a body weight of \>25kg.
• Adequate venous access for blood sampling.
• Male and female participants 6 to \< 18 years of age (participants must not reach their 18th birthday on or before the Baseline visit).
Exclusion Criteria:
• History of cluster headache or hemiplegic migraine headache.
• Confounding and clinically significant pain syndrome.
• Current uncontrolled and/or untreated psychiatric condition for a minimum of 6 months prior to the Screening Visit (lifetime history of psychosis and/or mania are excluded).
• History of suicidal behavior or at risk of self-harm/harm to others.
• History of major psychiatric disorder.
• Current diagnosis or history of substance abuse
• Reported current use of or tested positive at Screening for drugs of abuse.
DRUG: Rimegepant (PF-07899801)
Acute Treatment of Migraine
Long-term safety, acute treatment, migraine, phonophobia, photophobia, nausea, pediatric, children, adolescent
Simple Bone Cysts in Kids (SBoCK)
Krista Carpenter - krista.carpenter@cchmc.org
ALL
2 years to 21 years old
PHASE3
NCT02193841
Inclusion Criteria:
* Patients with a confirmed simple bone cyst in a long bone (ie. arm or leg) within last 3 months
* At least 3 weeks must have elapsed since last fracture
* At least 3 months must have elapsed since last cyst treatment
* Patients and/or their legal representatives willing to provide written informed consent (and assent, when appropriate)
* Patients with stabilizing implants in the bone where the cyst is located
Exclusion Criteria:
* Patients with bone disease (ie. osteogenesis imperfecta, cancer, osteoporosis, Paget's disease)
* Pregnant or breastfeeding female
* Patients with cysts crossing the growth plate (area where bone grows) PROCEDURE: Curettage with puncture (C & P), DEVICE: Curette, DEVICE: Vitoss morsels
Bone Cyst
Simple bone cyst, Unicameral bone cyst
A Study of Combination Chemotherapy for Patients With Newly Diagnosed DAWT and Relapsed FHWT
- cancer@cchmc.org
ALL
Up to 30 years old
PHASE2
NCT04322318
Inclusion Criteria:
* Patients with newly diagnosed stages 2 - 4 diffuse anaplastic Wilms tumor must be enrolled on APEC14B1, consented to Part A - Eligibility Screening, and have received an initial stratum assignment showing DAWT (if anaplasia first identified at diagnostic, pre-treatment nephrectomy or biopsy) or a final stratum assignment showing DAWT (if anaplasia first noted at delayed nephrectomy) prior to enrollment on AREN1921. Prior enrollment on APEC14B1 is not an eligibility requirement for patients with relapsed favorable histology Wilms tumor.
* Patients must be =\< 30 years old at study enrollment
* Patients with the following diagnoses are eligible for this study:
* Newly diagnosed stages 2 - 4 diffuse anaplastic Wilms tumor as confirmed by central review
* Favorable histology Wilms tumor at first relapse. Relapsed FHWT patients must have previously achieved remission for their initial FHWT diagnosis to be eligible for this study. The relapse risk groups are defined as follows, regardless of radiation therapy:
* Standard-Risk relapse: Patients who received two chemotherapy agents for frontline therapy; primarily actinomycin D and vincristine
* High-Risk relapse: Patients who received three chemotherapy agents for frontline therapy; primarily vincristine, actinomycin D and doxorubicin or vincristine, actinomycin D and irinotecan
* Very High-Risk relapse: Patients who received four or more chemotherapy agents as part of initial therapy; primarily regimen M or its variations
* Patients with newly diagnosed DAWT must have had histologic verification of the malignancy. For relapsed FHWT patients, biopsy to prove recurrence is encouraged, but not required
* Note: For relapsed FHWT patients, an institutional pathology report confirming favorable histology Wilms tumor (from relapse, if available, or from original diagnosis) must be available for upload prior to initiation of protocol therapy
* Patients with newly diagnosed Stages 2 - 4 diffuse anaplastic Wilms tumor must be enrolled on AREN1921 within 2 weeks of the tumor-directed surgery or biopsy procedure that first confirms a diagnosis of DAWT, whether at initial diagnostic procedure or delayed nephrectomy (such surgery/biopsy is day 0). For patients who received prior therapy for presumed favorable histology Wilms tumor, later confirmed to have diffuse anaplastic Wilms tumor at subsequent review of the initial biopsy
* Patients with newly diagnosed DAWT who undergo upfront nephrectomy must have at least 1 lymph node sampled prior to study enrollment
* 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 must have a life expectancy of \>= 8 weeks
* Diffuse Anaplastic Wilms Tumor: Patients with diffuse anaplastic histology must have had no prior systemic therapy, except in the following situations:
* Patients with diffuse anaplastic Wilms tumor who received no more than 12 weeks of pre nephrectomy chemotherapy for what was originally presumed to be favorable histology Wilms tumor, subsequently confirmed to be diffuse anaplastic Wilms tumor at delayed nephrectomy
* Patients with diffuse anaplastic Wilms tumor who received no more than 6 weeks of chemotherapy following upfront biopsy, initiated within 14 days of biopsy, for presumed favorable histology Wilms tumor based on institutional review, but subsequently corrected to diffuse anaplastic Wilms tumor based on the initial stratum assignment on APEC14B1-REN
* Treatment consisting of vincristine/doxorubicin/cyclophosphamide initiated on an emergent basis and within allowed timing as described
* Note: Patients who received prior therapy for presumed favorable histology Wilms tumor, later identified to have diffuse anaplastic Wilms tumor as per above, must begin study treatment starting at cycle 3 (week 7) of regimen UH 3. Patients who received emergency radiation to preserve organ function are eligible as noted. Patients who received radiation as part of standard of care for presumed newly diagnosed favorable histology Wilms tumor, along with chemotherapy as noted above, prior to identification of diffuse anaplasia, are also eligible
* Relapsed Favorable Histology Wilms Tumor: Patients must not have received prior chemotherapy for their relapsed favorable histology Wilms tumor diagnosis. In addition, patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study
* Myelosuppressive chemotherapy: Must not have received within 2 weeks of entry onto this study
* Radiation therapy (RT): \>= 2 weeks (wks) must have elapsed for local palliative RT (small port); \>= 6 months must have elapsed if prior craniospinal RT or if \>= 50% radiation of pelvis; \>= 6 wks must have elapsed if other substantial bone marrow (BM) radiation. Patients with relapsed favorable histology Wilms tumor who received emergency radiation to preserve organ function are eligible and do not need to washout with the above criteria
* Patients may not be receiving any other investigational agents (within 4 weeks prior to study enrollment)
* Peripheral absolute neutrophil count (ANC) \>= 750/uL (performed within 7 days prior to enrollment)
* Platelet count \>= 75,000/uL (transfusion independent) (performed within 7 days prior to enrollment)
* Hemoglobin \>= 8.0 g/dL (may receive red blood cell \[RBC\] transfusions) (performed within 7 days prior to enrollment)
* Patients with high-risk or very high-risk relapsed FHWT who will be treated with regimen ICE/Cyclo/Topo, must have renal function assessed by creatinine clearance or radioisotope glomerular filtration rate (GFR) and meet the following requirement:
* Creatinine clearance or radioisotope GFR \>= 60 mL/min/1.73 m\^2 (performed within 7 days prior to enrollment)
* Patients diagnosed with stage 2-4 DAWT or standard risk relapsed FHWT, who will be treated with regimen UH 3, may either obtain a creatinine clearance, radioisotope GFR (meeting the above criteria of GFR \>= 60 mL/min/1.73 m\^2), or an adequate serum creatinine as per the following table:
* Age: Maximum Serum Creatinine (mg/dL)
* 1 month to \< 6 months: 0.4 (male and female)
* 6 months to \< 1 year: 0.5 (male and female)
* 1 to \< 2 years: 0.6 (male and female)
* 2 to \< 6 years: 0.8 (male and female)
* 6 to \< 10 years: 1 (male and female)
* 10 to \< 13 years: 1.2 (male and female)
* 13 to \< 16 years: 1.5 (male), 1.4 (female)
* \>= 16 years: 1.7 (male), 1.4 (female)
* Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age or direct bilirubin =\< ULN for patients whose total bilirubin \> 1.5 x ULN (performed within 7 days prior to enrollment)
* Serum glutamic-oxaloacetic transaminase (SGOT) (aspartate aminotransferase \[AST\]) or serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) \< 2.5 x upper limit of normal (ULN) for age or =\< 5 x ULN for patients with liver metastases (performed within 7 days prior to enrollment)
* Shortening fraction of \>= 27% by echocardiogram, or ejection fraction of \>= 50% by radionuclide angiogram (obtained within 21 days prior to enrollment and start of protocol therapy)
Exclusion Criteria:
* Patients with a history of bilateral Wilms tumor (synchronous or metachronous)
* Patients with any uncontrolled, intercurrent illness including, but not limited to, ongoing or active infection, or symptomatic congestive heart failure (defined as grade 2 or higher heart failure per Common Terminology Criteria for Adverse Events \[CTCAE\] version 5.0)
* Relapsed FHWT patients who did not receive frontline chemotherapy (e.g., very low risk FHWT initially observed without chemotherapy) or received only one chemotherapy agent for frontline therapy
* For patients with high-risk or very high-risk relapsed FHWT:
* Patients with renal tubular acidosis (RTA) as evidenced by serum bicarbonate \< 16 mmol/L and serum phosphate =\< 2 mg/dL (or \< 0.8 mmol/L) without supplementation
* For stages 2-4 DAWT and standard-risk relapsed FHWT patients:
* Chronic inflammatory bowel disease and/or bowel obstruction
* Concomitant use of St. John's wort, which cannot be stopped prior to the start of trial treatment
* 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 PROCEDURE: Biopsy Procedure, PROCEDURE: Biospecimen Collection, PROCEDURE: Bone Scan, DRUG: Carboplatin, PROCEDURE: Computed Tomography, DRUG: Cyclophosphamide, DRUG: Doxorubicin, DRUG: Etoposide, DRUG: Ifosfamide, DRUG: Irinotecan, PROCEDURE: Magnetic Resonance Imaging, PROCEDURE: Positron Emission Tomography, RADIATION: Radiation Therapy, PROCEDURE: Surgical Procedure, DRUG: Topotecan, PROCEDURE: Transabdominal Ultrasound, DRUG: Vincristine, PROCEDURE: X-Ray Imaging
Anaplastic Kidney Wilms Tumor, Recurrent Kidney Wilms Tumor, Stage II Kidney Wilms Tumor, Stage III Kidney Wilms Tumor, Stage IV Kidney Wilms Tumor
Epilepsy Learning Healthcare System (ELHS) (ELHS)
Stephanie Loos - Stephanie.Loos@cchmc.org
ALL
NCT06265103
Inclusion Criteria:
* In order to be eligible to participate in this registry-based study, an individual must meet all of the following criteria:
* Patient is in an established care relationship with the ELHS site
Exclusion Criteria:
* An individual who meets any of the following criteria will be excluded from participation in this registry-based research study:
* Patients who are not currently in nor expect to be in an established care relationship with the ELHS site (for example, patients who are being seen at the center for a second opinion only).
* Patients who do not, after diagnostic evaluation, meet criteria for a diagnosis of epilepsy will not be analyzed in epilepsy-specific population groups. However, these non-epilepsy patients will not be excluded from the registry. OTHER: Clinical care and quality improvement
Epilepsy, Seizure Disorder, Neurologic Disorder, Rare Diseases
Focal seizures, Generalized seizures, Brain conditions, Developmental disorders, Rare diseases, Chronic condition, Health disparity, Seizure, Post-traumatic epilepsy (PTE), Post-stroke epilepsy
Infant Formula in Infants and Children With Cow's Milk Allergy
Jennifer Jennings - jennifer.jennings2@cchmc.org
ALL
3 months to 12 years old
NA
NCT06456541
Inclusion Criteria:
* Participant's parent(s) is/are willing for their child to undergo confirmatory testing or must have had at least one of the following within 6 months of enrollment:
• Physician diagnosis of IgE-mediated CMA, according to the participant's clinical history (e.g. gastrointestinal symptoms, hives, respiratory symptoms or angioedema) AND, detectable serum milk-specific IgE \>0.7 kUᴀ/L or positive skin prick test wheal ≥5 mm;
• Documentation of milk-specific serum IgE ≥15 kUᴀ/L or ≥ 5 kUᴀ/L if younger than 1 year;
• Documented cow's milk skin prick test wheal ≥8mm;
• Physician-supervised oral food challenge that elicited immediate, objective, allergic symptoms. * Participant's parent(s) agree for their child to stop oral steroid use within 14 days and antihistamine use within 7 days prior to confirmation of diagnosis and food challenges. * Participant had followed a strict cow's milk protein-free diet for at least 2 weeks prior to enrollment. * Parent(s) confirm their intention not to administer any products containing cow's milk protein during the study. * Participant is between 3 months and 12 years of age at enrollment. * Participant's parent(s) has voluntarily signed and dated an Informed Consent Form (ICF), approved by an Independent Ethics Committee/Institutional Review Board (IEC/IRB) and provided Health Insurance Portability and Accountability Act (HIPAA) (or other applicable privacy regulation) authorization prior to any participation in the study.
Exclusion Criteria:
* Participant is partially or exclusively breastfed at the time of enrollment.
* Participant is consuming an amino acid-based formula due to failure on an extensively hydrolyzed formula.
* Significant chronic medical diseases including major chromosomal or congenital anomalies, gastrointestinal diseases, or abnormalities other than CMA, immunodeficiencies, unstable asthma, eczema and/or food allergies treated with biologics (omalizumab or other monoclonal antibody), FPIES, eosinophilic esophagitis, and severe uncontrolled eczema.
* Previous severe anaphylactic reaction to cow's milk within the last two years.,
* An adverse medical history or current condition that is thought by the investigator to have potential for effects on tolerance or the hypoallergenicity test.
* Participant has an allergy or intolerance to any ingredient in the study product, as reported by the parent.
* Participant is consuming baked milk products.
* Use of and/or changing dose of high potency steroids.
* Participant is treated with any oral immunotherapy (sublingual, subcutaneous, and epicutaneous) for any food allergy. OTHER: Experimental Extensively Hydrolyzed Formula, OTHER: Placebo Extensively Hydrolyzed Formula
Cow's Milk Allergy
Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma
- cancer@cchmc.org
ALL
Up to 30 years old
PHASE3
NCT06172296
Inclusion Criteria:
* Patients must be enrolled on APEC14B1 and have consented to testing through the Molecular Characterization Initiative (MCI), prior to enrollment on ANBL2131
* ≤ 30 years at the time of initial diagnosis with high-risk disease
* \* Must have a diagnosis of neuroblastoma (NBL) or ganglioneuroblastoma (nodular) verified by tumor pathology analysis or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamines
* Newly diagnosed, high risk neuroblastoma (HRNBL) defined as one of the following:
* Any age with International Neuroblastoma Risk Group (INRG) Stage L2, MS, or M and MYCN amplification
* Age ≥ 547 days and INRG stage M regardless of biologic features (clinical MYCN testing not required prior to enrollment)
* Any age initially diagnosed with INRG Stage L1 MYCN amplified NBL who have progressed to stage M without systemic chemotherapy
* Age ≥ 547 days of age initially diagnosed with INRG Stage L1, L2, or MS who have progressed to stage M without systemic chemotherapy (clinical MYCN testing not required prior to enrollment)
* Patients must have a body surface area (BSA) ≥ 0.25 m\^2
* No prior anti-cancer therapy except as outlined below:
* Patients initially recognized to have high-risk disease treated with topotecan/cyclophosphamide initiated on an emergent basis and within allowed timing, and with consent
* Patients observed or treated with a single cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (e.g., as per ANBL0531, ANBL1232 or similar) for what initially appeared to be non-high-risk disease but subsequently found to meet the criteria
* Patients who received localized emergency radiation to sites of life threatening or function-threatening disease prior to or immediately after establishment of the definitive diagnosis
* Human immunodeficiency virus (HIV) -infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial
* A serum creatinine based on age/sex as follows:
* 1 month to \< 6 months: Male 0.4 mg/dL and female 0.4mg/dL
* 6 months to \< 1 year: Male 0.5 mg/dL and female 0.5 mg/dL
* 1 to \< 2 years: Male 0.6 mg/dL and female 0.6 mg/dL
* 2 to \< 6 years: Male 0.8 mg/dL and female 0.8 mg/dL
* 6 to \< 10 years: Male 1 mg/dL and female 1 mg/dL
* 10 to \< 13 years: Male 1.2 mg/dL and female 1.2 mg/dL
* 13 to \< 16 years: Male 1.5 mg/dL and female 1.4 mg/dL
* ≥ 16 years: Male 1.7 mg/dL and female 1.4 mg/dL
* The threshold creatinine values were derived from the Schwartz formula for estimating glomerular filtration rate (GFR) utilizing child length and stature data published by the Centers for Disease Control (CDC)
* or a 24-hour urine creatinine clearance ≥ 70 mL/min/1.73 m\^2 or
* or a GFR ≥ 70 mL/min/1.73 m\^2. 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
* Serum glutamic pyruvic transaminase (SGPT) (Alanine aminotransferase \[ALT\]) ≤ 10 x ULN\*
* Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U/L
* \* Shortening fraction of ≥ 27% by echocardiogram, or
* Ejection fraction of ≥ 50% by echocardiogram or radionuclide angiogram
* Ability to tolerate Peripheral Blood Stem Cell (PBSC) collection:
No known contraindication to PBSC collection. Examples of contraindications might be a weight or size less than the collecting institution finds feasible, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and/or the apheresis procedure
Exclusion Criteria:
* Patients who are 365-546 days of age with INRG Stage M and MYCN non-amplified NBL, irrespective of additional biologic features
* Patients ≥ 547 days of age with INRG Stage L2, MYCN non-amplified NBL, regardless of additional biologic features
* Patients with known bone marrow failure syndromes
* Patients on chronic immunosuppressive medications (e.g., tacrolimus, cyclosporine, corticosteroids) for reasons other than prevention/treatment of allergic reactions and adrenal replacement therapy are not eligible. Topical and inhaled corticosteroids are acceptable
* Patients with a primary immunodeficiency syndrome who require ongoing immune globulin replacement therapy
* 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 prior to enrollment 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
* 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 PROCEDURE: Biospecimen Collection, PROCEDURE: Bone Marrow Aspiration, PROCEDURE: Bone Marrow Biopsy, DRUG: Carboplatin, DRUG: Cisplatin, PROCEDURE: Computed Tomography, DRUG: Cyclophosphamide, BIOLOGICAL: Dinutuximab, DRUG: Doxorubicin, PROCEDURE: Echocardiography Test, DRUG: Etoposide, PROCEDURE: FDG-Positron Emission Tomography and Computed Tomography Scan, PROCEDURE: Hematopoietic Cell Transplantation, DRUG: Irinotecan, DRUG: Isotretinoin, PROCEDURE: Leukapheresis, PROCEDURE: Magnetic Resonance Imaging, DRUG: Melphalan, PROCEDURE: Multigated Acquisition Scan, RADIATION: Radiation Therapy, PROCEDURE: Radionuclide Imaging, OTHER: Survey Administration, DRUG: Temozolomide, DRUG: Thiotepa, DRUG: Topotecan, PROCEDURE: Tumor Resection, DRUG: Vincristine
Ganglioneuroblastoma, Nodular, Neuroblastoma
Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG- Infant
Elizabeth Smith, PhD - elizabeth.smith3@cchmc.org
ALL
6 months to 26 months old
NA
NCT06560242
Inclusion Criteria:
* Diagnoses of Fragile X Syndrome, Typical Development, or History of Premature Birth
* able to sit independently
* English is spoken at home
Exclusion Criteria:
* For all participants: no seizures in the past 6 months
* For typical development group and Fragile X group: not born prior to 32 weeks gestation OTHER: Speech discrimination
Fragile X Syndrome
Non-Invasive Diagnosis of Pediatric Pulmonary Invasive Mold Infections (DOMINIC)
Caitlin Brammer - caitlin.brammer@cchmc.org
ALL
120 days to 21 years old
NCT03827694
Inclusion Criteria:
* Males or females age \> 120 days and \< 22 years at any participating site
* Have at least one of the following conditions associated with a known high incidence of IFI: hematopoietic stem cell transplantation (HSCT), aplastic anemia, bone marrow failure, primary or acquired immune deficiency, or malignancy
* New (last 96 hours) radiographic evidence of at least one of the following: at least one nodular lesion greater than or equal to 5 mm in size, a wedge-shaped and segmental or lobar consolidation, a cavitary lesion, a lesion with a halo sign, a lesion with a reverse halo sign, or a lesion with an air crescent sign
* Prolonged neutropenia (absolute neutrophil count \< 500 cells/µl for a period of ≥ 5 consecutive days) in 30 days prior to and including the day of qualifying chest MRI or CT scan date OR currently receiving systemic therapy for acute or chronic graft-versus-host disease (GVHD) OR presence of neutrophil dysfunction because of underlying acquired or primary immune deficiency (e.g. chronic granulomatous disease) on the date of the qualifying chest MRI or CT scan
* Subject consent or parental/guardian permission (informed consent) and if appropriate, child assent
Exclusion Criteria:
* Weight \<3 kg, so as to not exceed 3 ml/kg in a single blood draw
* Previous inclusion in this study DIAGNOSTIC_TEST: Non-Invasive Testing for PIFI
Pulmonary Invasive Fungal Infections, Pulmonary Invasive Aspergillosis
Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy (MUSIC-DMD)
Jaynee Bartsch - jaynee.bartsch@cchmc.org
MALE
18 years and over
PHASE1
NCT06224660
Inclusion Criteria:
* Diagnosis of DMD with confirmatory genetic testing
* Cardiomyopathy with left ventricular scar in at least 3 of 16 segments
* Left ventricular ejection fraction \< 40%
* Individualized, optimized cardiac medical therapy and glucocorticoid treatment for at least 12 months prior to enrollment
* Willing and able to provide informed consent
Exclusion Criteria:
* Abnormal blood pressure
* Non-DMD-related liver function test elevations
* Cystatin C ≥ 1.2 mg/L
* Thrombocytopenia
* Anemia
* Inadequate pulmonary function GENETIC: SRD-001
DMD-Associated Dilated Cardiomyopathy
Simulation Trial of Telemedical Support for Paramedics (R01)
Bradford McClain - bradford.mcClain@cchmc.org
ALL
21 years and over
NA
NCT06441760
Inclusion Criteria:
* Certified Emergency Medical Technicians (EMTs), Advanced EMTs (AEMTs), and Paramedics (EMT-Ps) who provide direct scene response.
* Board-certified Pediatric Emergency Medicine (PEM) and Emergency Medicine (EM) physicians whose practice includes online medical support for EMS are eligible.
* The control arm will include physicians who provide radio/telephone support in usual care at each site. In the intervention arm, experts will be PEM with/without EMS board-certification as they have relevant pediatric training and experience.
Exclusion Criteria:
* EMS personnel providing interfacility transport and/or pediatric specialty transport
* Resident physicians-in-training
* Non-physician providers OTHER: Video teleconsultation, OTHER: Audio support
Emergencies, Cardiopulmonary Arrest, Acute Respiratory Failure, Status Epilepticus
Prehospital emergency care, Emergency medical services (EMS), Paramedics, Infant simulator mannequins, Telemedical support, Critically ill infants and children, Pediatric emergencies
Effects of Dexrazoxane Hydrochloride on Biomarkers Associated With Cardiomyopathy and Heart Failure After Cancer Treatment
- cancer@cchmc.org
ALL
NCT01790152
Inclusion Criteria:
Study Strata I, II, and III are closed for further patient entry as of March 31, 2021. The study remains open for existing medical record submission of Stratum IV
* STRATUM I AND STRATUM II: LEUKEMIA AND LYMPHOMA SURVIVORS
* Previously enrolled leukemia and lymphoma survivors, randomized to + or - DRZ on P9404, P9425, P9426, or DFCI 95-01 (high-risk patients only)
* STRATUM I: Alive and in continuous first complete remission from their original cancer (leukemia/lymphoblastic lymphoma \[P9404, high-risk DFCI 95-01\] or Hodgkin lymphoma \[P9425/P9426\])
* STRATUM I: Did not have progressive disease or induction failure requiring off-protocol therapy including hematopoietic cell transplantation
* STRATUM I: Must not have been diagnosed with any subsequent malignancy that required additional cardiotoxic therapies (i.e., radiotherapy to the chest \[also includes fields directed towards the neck, upper abdomen, or spine\], or additional anthracyclines or anthraquinones); patients with history of subsequent malignancy that did not require such therapies remain eligible
* STRATUM I: All patients and/or their parents or legal guardians must sign a written informed consent
* STRATUM II: Among leukemia and lymphoma patients randomized to + or - DRZ on P9404, P9425, P9426, and DFCI 95-01 (high risk patients only) who have relapsed or have experienced a subsequent malignancy that precludes eligibility since their original diagnosis, the study committee will review the available data (both from Children's Oncology Group's \[COG?s\] Statistics and Data Center \[SDC\] and the participating institution) to determine if individual patients are to be selected for Stratum 2; in recognition that local institutions sometimes have more updated relapse/subsequent cancer data than SDC, in cases where local data is more updated, local data will be used preferentially; the study will petition the Institutional Review Board (IRB) specifically for a waiver of consent to include any relapse and subsequent cancer data obtained from existing records for analysis of the secondary aims; patients selected for Stratum 2 will be those for whom late relapse or subsequent cancer is reported but who lack clear confirmation in existing records (either at SDC or at the local institution)
* STRATUM II: Alive, but have experienced relapse of their original cancer and/or have developed a subsequent cancer (other than non-melanomatous skin cancer) since their original diagnosis
* STRATUM II: All patients and/or their parents or legal guardians must sign a written informed consent
* STRATUM III: OSTEOSARCOMA SURVIVORS
* Previously enrolled osteosarcoma survivors treated on P9754 who are alive and able (themselves and/or parents/legal guardian) to provide written informed consent; note that relapse and subsequent malignancy are not exclusion criteria for P9754 survivors
* Comparison subjects for P9754 survivors will be eligible to be enrolled from any ALTE11C2 participating COG site (even if that institution did not participate on P9754), according to the following criteria:
* Newly diagnosed, previously untreated biopsy-proven moderate or high grade osteosarcoma without metastasis; patients with low grade osteosarcoma, parosteal or periosteal sarcoma are ineligible
* \< 31 years of age at time of initial osteosarcoma diagnosis
* Diagnosis occurred between January 1, 1999 through December 31, 2002; duration of therapy can extend beyond 2002
* No evidence of poor or low cardiac function at time of initial osteosarcoma diagnosis; if reports from the time are available: shortening fraction \>= 28% by echocardiogram and within the institutional normative range for age, or radionuclide angiogram ejection fraction \>= 50%; if imaging reports from the time are no longer available, there must be no documentation within available medical records that suggest poor or low cardiac function at time of diagnosis
* Comparison subject must have institutional records (e.g., clinic note, treatment summary, chemotherapy roadmap) documenting lifetime receipt of 450 to 600 mg/m\^2 of doxorubicin (doses within 10% are acceptable); this includes initial therapy as well as any subsequent therapy for relapse or second cancer, if relevant; as such, comparison subjects who have had osteosarcoma relapse or subsequent malignancies remain eligible so long as they meet all other eligibility criteria
* No anthracycline or anthraquinone aside from doxorubicin was ever given as part of initial or subsequent therapies
* No exposure to DRZ at any point in time
* All patients and/or their parents or legal guardians must sign a written informed consent
* STRATUM IV: CARDIOMYOPATHY CASES, NOT OTHERWISE ELIGIBLE FOR STRATUMS 1, 2, AND 3
* Individuals diagnosed with cancer prior to age 21 years, who required treatment with chemotherapy and/or radiotherapy, achieved initial remission, and remained alive after completing anti-cancer-therapy for at least 1 year
* Must have screening echocardiograms for heart function as part of cancer therapy and off-therapy evaluations available (Digital Imaging and Communications in Medicine \[DICOM\] format). Images from Video Home System (VHS) tapes and reports only (without images) are not suitable
* Cannot have a known history of congenital heart disease (patent foramen ovale remain eligible) or underlying genetic syndrome associated with abnormal cardiovascular development or health (e.g., down syndrome)
* Based on echocardiography, must have either left ventricular fractional shortening =\< 28.0% or ejection fraction =\< 50.0% on at least two occasions, with at least one of these measurements occurring after cancer therapy completion and be in the absence of sepsis or any uncontrolled infection
* If the fractional shortening or ejection fraction criteria is only met on one occasion, this must be after cancer therapy completion, be in the absence of sepsis or any uncontrolled infection, and the patient must have subsequently started on chronic medical therapy for cardiomyopathy (e.g., beta-blocker, angiotensin-converting enzyme \[ACE\]-inhibitor, angiotensin receptor blocker) lasting at least 6 months
* For all participants (stratums 1, 2, 3, and 4), all institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met OTHER: Assessment of Therapy Complications, OTHER: Laboratory Biomarker Analysis, OTHER: Quality-of-Life Assessment, OTHER: Questionnaire Administration
Hodgkin Lymphoma in Remission, Leukemia in Remission, Lymphoblastic Lymphoma, Osteosarcoma, Recurrent Leukemia, Recurrent Lymphoma, Recurrent Malignant Neoplasm
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