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Conditions: Cardiomyopathy, Dilated
Interventions: Not listed
Lead sponsor: Carelon Research; Other
Eligibility: Up to 22 Years
Enrollment: 131 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2009
U.S. locations: 7
States / cities: Boston, Massachusetts • St Louis, Missouri • New York, New York + 4 more

Conditions: Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Rhabdoid Tumor, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Recurrent WHO Grade 2 Glioma, Refractory Ependymoma, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Rhabdoid Tumor, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Refractory WHO Grade 2 Glioma, Wilms Tumor
Interventions: Ivosidenib; Drug
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 12 Months to 21 Years
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2027
U.S. locations: 171
States / cities: Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 129 more

Conditions: Iron Overload
Interventions: Not listed
Lead sponsor: Boston Children's Hospital; Other
Eligibility: 5 Years and older
Enrollment: 38 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2010
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Heterozygous or Homozygous Familial Hypercholesterolemia
Interventions: Inclisiran; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 12 Years to 100 Years
Enrollment: 195 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2032
U.S. locations: 5
States / cities: Boca Raton, Florida • New York, New York • Cincinnati, Ohio + 2 more

Conditions: Mucopolysaccharidosis Type IIIA
Interventions: DNL126; Drug
Lead sponsor: Denali Therapeutics Inc.; Industry
Eligibility: 0 Years to 18 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2028
U.S. locations: 4
States / cities: Oakland, California • Iowa City, Iowa • Chapel Hill, North Carolina + 1 more

Conditions: Neuroblastoma, Sarcoma
Interventions: Cabozantinib; Drug
Lead sponsor: Nationwide Children's Hospital; Other
Eligibility: 18 Months to 40 Years
Enrollment: 86 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2029
U.S. locations: 6
States / cities: Birmingham, Alabama • Aurora, Colorado • Washington D.C., District of Columbia + 3 more

Conditions: Fanconi Anemia Complementation Group A
Interventions: RP-L102; Biological
Lead sponsor: Rocket Pharmaceuticals Inc.; Industry
Eligibility: 1 Year and older
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2026
U.S. locations: 2
States / cities: Stanford, California • Minneapolis, Minnesota

Conditions: Achondroplasia
Interventions: Not listed
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: Up to 17 Years
Enrollment: 363 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2021
U.S. locations: 9
States / cities: Los Angeles, California • Oakland, California • Wilmington, Delaware + 6 more

Conditions: Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission, Myelodysplastic Syndromes, Chronic Myeloid Leukemia, Hemophagocytic Lymphohistiocytoses, Primary Immunodeficiency Diseases, Hemoglobinopathies, Severe Aplastic Anemia, Cytopenia, Bone Marrow Failure Syndrome, Severe Chronic Active Epstein-Barr Virus Infection
Interventions: CliniMACS; Device
Lead sponsor: Baylor College of Medicine; Other
Eligibility: Up to 55 Years
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2026
U.S. locations: 2
States / cities: Houston, Texas

Conditions: Friedreich Ataxia, Cardiomyopathies, Cardiac Hypertrophy, Myocardial Fibrosis
Interventions: AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN, Prednisone; Biological · Drug
Lead sponsor: Weill Medical College of Cornell University; Other
Eligibility: 12 Years to 50 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2029
U.S. locations: 1
States / cities: New York, New York

Conditions: Congenital Hypothyroidism
Interventions: Tirosint®-SOL, Levothyroxine Sodium; Drug
Lead sponsor: IBSA Institut Biochimique SA; Industry
Eligibility: 1 Day to 9 Months
Enrollment: 34 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 10
States / cities: Los Angeles, California • Orange, California • San Francisco, California + 7 more

Conditions: Osteosarcoma, Ewing Sarcoma, Rhabdomyosarcoma, Relapsed Pediatric Solid Tumor, Refractory Pediatric Solid Tumor, Melanoma, Hepatoblastoma, Hepatocellular Carcinoma, Neuroblastoma, Wilms Tumor
Interventions: Cryoablation Therapy, Nivolumab, Ipilimumab; Procedure · Drug
Lead sponsor: Children's National Research Institute; Other
Eligibility: 1 Year to 39 Years
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2024
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Diffuse Astrocytoma, Anaplastic Astrocytoma, Astrocytoma, Oligodendroglioma, Childhood, Anaplastic Oligodendroglioma, Glioblastoma, Pilocytic Astrocytoma, Giant Cell Astrocytoma, Pleomorphic Xanthoastrocytoma, Anaplastic Pleomorphic Xanthoastrocytoma, Angiocentric Glioma, Chordoid Glioma of Third Ventricle, Gangliocytoma, Ganglioglioma, Anaplastic Ganglioglioma, Dysplastic Gangliocytoma of Cerebrellum, Desmoplastic Infantile Astrocytoma and Ganglioglioma, Papillary Glioneuronal Tumor, Rosette-forming Glioneuronal Tumor, Central Neurocytoma, Extraventricular Neurocytoma, Cerebellar Liponeurocytoma, Neurofibromatosis Type 1
Interventions: dabrafenib, trametinib; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 1 Year to 99 Years
Enrollment: 163 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2026
U.S. locations: 12
States / cities: Phoenix, Arizona • Washington D.C., District of Columbia • Miami, Florida + 8 more

Conditions: Dermatitis Atopic
Interventions: Standard of care; Other
Lead sponsor: Sanofi; Industry
Eligibility: 0 Years to 11 Years
Enrollment: 1,860 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2035
U.S. locations: 46
States / cities: Birmingham, Alabama • Montgomery, Alabama • Phoenix, Arizona + 35 more

Conditions: Pediatric: Genetic Syndrome
Interventions: rapid whole genomic sequencing (rWGS); Diagnostic Test
Lead sponsor: Tufts Medical Center; Other
Eligibility: 1 Day to 1 Year
Enrollment: 400 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2022
U.S. locations: 6
States / cities: San Diego, California • Boston, Massachusetts • New York, New York + 3 more

Conditions: Lysosomal Acid Lipase Deficiency
Interventions: There is no intervention in the study; Other
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 2 Years to 16 Years
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2017
U.S. locations: 2
States / cities: Atlanta, Georgia • Houston, Texas

Conditions: Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer, Liver Cancer, Neuroblastoma, Ovarian Cancer, Sarcoma, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions: ABT-751; Drug
Lead sponsor: National Institutes of Health Clinical Center (CC); NIH
Eligibility: Up to 18 Years
Enrollment: 90 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2010
U.S. locations: 3
States / cities: Chicago, Illinois • Bethesda, Maryland • Philadelphia, Pennsylvania

Conditions: Hematopoietic and Lymphatic System Neoplasm, Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Histiocytic and Dendritic Cell Neoplasm, Recurrent Langerhans Cell Histiocytosis, Recurrent Lymphoma, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Rhabdoid Tumor, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Recurrent WHO Grade 2 Glioma, Refractory Ependymoma, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Histiocytic and Dendritic Cell Neoplasm, Refractory Langerhans Cell Histiocytosis, Refractory Lymphoma, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Rhabdoid Tumor, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Refractory WHO Grade 2 Glioma, Wilms Tumor
Interventions: Computed Tomography, Magnetic Resonance Imaging, Positron Emission Tomography, Radionuclide Imaging, Selpercatinib, X-Ray Imaging; Procedure · Drug
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 12 Months to 21 Years
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2026
U.S. locations: 172
States / cities: Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 129 more

Conditions: Atopic Dermatitis
Interventions: Infrequent versus Frequent Soaking Baths; Behavioral
Lead sponsor: MaineHealth; Other
Eligibility: 6 Months to 11 Years
Enrollment: 63 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011 – 2017
U.S. locations: 1
States / cities: Portland, Maine

Conditions: Retinoblastoma, Clear Cell Sarcoma, Renal Cell Carcinoma, Rhabdoid Tumor, Wilms Tumor, Hepatoblastoma, Neuroblastoma, Germ Cell Tumors, Ewings Sarcoma, Non-rhabdomyosarcoma Soft Tissue Sarcoma, Osteosarcoma, Rhabdomyosarcoma
Interventions: Simvastatin, Cyclophosphamide, Topotecan, Myeloid growth factor; Drug
Lead sponsor: Emory University; Other
Eligibility: 1 Year to 29 Years
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2019
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Hunter Syndrome
Interventions: No treatment; Other
Lead sponsor: Shire; Industry
Eligibility: Up to 70 Years · Male only
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2013
U.S. locations: 5
States / cities: Decatur, Georgia • Chicago, Illinois • Minneapolis, Minnesota + 2 more

Conditions: Spinal Muscular Atrophy, Charcot-Marie-Tooth Disease, Muscular Dystrophy, Spinal Muscular Atrophy With Respiratory Distress 1, Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Neuromuscular Disease, Peroneal Muscular Atrophy
Interventions: sample collection; Other
Lead sponsor: Nemours Children's Clinic; Other
Eligibility: 1 Month and older
Enrollment: 230 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2024
U.S. locations: 3
States / cities: Wilmington, Delaware • Jacksonville, Florida • Orlando, Florida

Conditions: Wolfram Syndrome, Diabetes Mellitus, Optic Nerve Atrophy, Ataxia
Interventions: dantrolene sodium; Drug
Lead sponsor: Washington University School of Medicine; Other
Eligibility: 5 Years to 60 Years
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2023
U.S. locations: 1
States / cities: St Louis, Missouri

Conditions: Advanced Malignant Solid Neoplasm, Malignant Solid Neoplasm, Recurrent Ependymoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Primary Central Nervous System Neoplasm, Recurrent Rhabdoid Tumor, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Refractory Ependymoma, Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Primary Central Nervous System Neoplasm, Refractory Rhabdoid Tumor, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Wilms Tumor
Interventions: Biospecimen Collection, Bone Marrow Aspiration and Biopsy, Bone Scan, Computed Tomography, Ensartinib, Laboratory Biomarker Analysis, Magnetic Resonance Imaging, Pharmacological Study, Positron Emission Tomography, Radionuclide Imaging, X-Ray Imaging; Procedure · Drug · Other
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 12 Months to 21 Years
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2026
U.S. locations: 122
States / cities: Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 95 more

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Variability of Ventricular Mass, Volume, & Ejection Fraction in Pediatric Cardiomyopathy Patients-Pediatric Heart Network

Ivosidenib in Treating Patients With Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With IDH1 Mutations (A Pediatric MATCH Treatment Trial)

Iron Overload in Stem Cell Transplant Recipients

Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20, or ORION-19 Studies

Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

A Study of Cabozantinib as a Maintenance Agent to Prevent Progression or Recurrence in High-Risk Pediatric Solid Tumors

Gene Therapy for Fanconi Anemia, Complementation Group A

A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia

Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion

Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia

Use of Tirosint®-SOL or Tablet Formulations of Levothyroxine in Pediatric Patients With Congenital Hypothyroidism (CH)

Neoadjuvant Dual Checkpoint Inhibition and Cryoablation in Relapsed/Refractory Pediatric Solid Tumors

Pediatric Long-Term Follow-up and Rollover Study

Observational Evaluation of Atopic Dermatitis in Pediatric Patients

Genomic Medicine for Ill Neonates and Infants (The GEMINI Study)

Screening for Lysosomal Acid Lipase Deficiency

ABT-751 in Treating Young Patients With Refractory Solid Tumors

Selpercatinib for the Treatment of Advanced Solid Tumors, Lymphomas, or Histiocytic Disorders With Activating RET Gene Alterations, a Pediatric MATCH Treatment Trial

Twice Daily Versus Twice Weekly Soak-and-Seal Baths in Pediatric Atopic Dermatitis

Simvastatin With Topotecan and Cyclophosphamide in Relapsed and/or Refractory Pediatric Solid and CNS Tumors

Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome

Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases

A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome

Ensartinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With ALK or ROS1 Genomic Alterations (A Pediatric MATCH Treatment Trial)