I Change Adherence & Raise Expectations

Natural History Study of SCID Disorders

Safety Study of Unlicensed, Investigational Cord Blood Units Manufactured by the NCBP for Unrelated Transplantation

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

Congenital Sucrase-Isomaltase Deficiency (CSID) Genetic Prevalence Study (GPS)

Patients Treated for Chronic Granulomatous Disease (CGD) Since 1995

KingVision Video Laryngoscopy vs Direct Laryngoscopy - Equivalence Trial

Efficacy Study Of Tofacitinib In Pediatric JIA Population

Observational Study of Pediatric Rheumatic Diseases: The CARRA Registry

Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

A Phase 2 Study of Ruxolitinib With Chemotherapy in Children With Acute Lymphoblastic Leukemia

FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE)

Long-term Safety Study of AR101 in Subjects Who Participated in a Prior AR101 Study (ARC008)

A Long-term Follow-up Study to Evaluate the Impact of Lumicitabine on the Incidence of Asthma and/or Wheezing in Infants and Children With a History of Respiratory Syncytial Virus Infection

Low Dose Oral Methotrexate in Pediatric Crohn's Disease Patients Initiating Anti-Tumor Necrosis Factor (Anti-TNF) Therapy

Safety and Efficacy Study of Viaskin Peanut in Peanut-allergic Young Children 1-3 Years of Age

Modified Measles Virus (MV-NIS) for Children and Young Adults With Recurrent Medulloblastoma or Recurrent ATRT

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Subjects With Transfusion-Dependent β-Thalassemia, Who Have a β0/β0 Genotype

A Study of Intravenous and Oral Isavuconazonium Sulfate in Pediatric Patients

Neurocognition in Congenital Central Hypoventilation Syndrome (CCHS)

Sickle Cell Pro-Inflammatory Response to Interval Training Study

A Study of Safety and Efficacy of MK-1986 (Tedizolid Phosphate) and Comparator in Participants From Birth to

MIPE for Pilonidal Disease

A Phase I/II Study of the Photon Radiosurgery System

Lenalidomide and Radiation Therapy in Treating Young Patients With Pontine Glioma or High-Grade Glioma

Comparison of Three Treatments for Lower Extremity Apophysitis

PNOC 001: Phase II Study of Everolimus for Recurrent or Progressive Low-grade Gliomas in Children

Pediatric Obesity Weight Evaluation Registry (POWER) Study

Coordinated Healthcare Interventions for Childhood Asthma Gaps in Outcomes

Remodulin as Add-on Therapy for the Treatment of Persistent Pulmonary Hypertension of the Newborn

Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy

Binocular Dig Rush Game Treatment for Amblyopia

Pharmacokinetic, Pharmacodynamic, Safety, and Efficacy Study of Rivaroxaban for Thromboprophylaxis in Pediatric Participants 2 to 8 Years of Age After the Fontan Procedure

Expanded Access Protocol for Tabelecleucel in Subjects With EBV-Associated Viremia or Malignancies

National Collaborative to Improve Care of Children With Complex Congenital Heart Disease

Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease

Efficacy, Safety and Pharmacokinetics of Topical Timolol in Infants With Infantile Hemangioma (IH)

Study Comparing Efficacy and Safety of Defibrotide vs Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients

CHaractErizing CFTR Modulated Changes in Sweat Chloride and Their Association With Clinical Outcomes

A Study to Evaluate the Efficacy and Safety of Pimodivir in Combination With the Standard-of-Care Treatment in Adolescent, Adult, and Elderly Non-Hospitalized Participants With Influenza A Infection Who Are at Risk of Developing Complications

Stress Hydrocortisone In Pediatric Septic Shock

Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex

MK-7625A Versus Meropenem in Pediatric Participants With Complicated Urinary Tract Infection (cUTI) (MK-7625A-034)

A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia

A Study to Evaluate the Efficacy and Safety of Daratumumab in Pediatric and Young Adult Participants Greater Than or Equal to (>=)1 and Less Than or Equal to (

Defining the Skin and Blood Biomarkers of Ichthyosis

TODAY! A Mobile App Study

A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia

Local Version of the Multi-center PREVENT Study Evaluating Cardio-respiratory Instability in Premature Infants

Pediatric Acute Gastrointestinal Bleeding Registry

A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma

Pharmacokinetics and Safety of Commonly Used Drugs in Lactating Women and Breastfed Infants

Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome

An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy

Biliary Atresia Study in Infants and Children

Evaluating the Genetic Causes and Progression of Cholestatic Liver Diseases (LOGIC)

PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry

Bortezomib and Combination Chemotherapy in Treating Young Patients With Relapsed Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma

Patients Treated for SCID (1968-Present)

Evaluation of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC)

Quantitative Detection of Circulating Donor-Specific DNA in Organ Transplant Recipients (DTRT-Multi-Center Study)

Reducing Emotional Distress, Enhancing Function and Improving Network Engagement in Adolescent and Young Adult Oncology

A Study of Ramucirumab (LY3009806) in Children With Refractory Solid Tumors

Simple Bone Cysts in Kids

Home Away From Home - Quality of Life Surveys

Dose-finding, Pharmacokinetics, and Safety of VABOMERE in Pediatric Subjects With Bacterial Infections

Trial of Heat Shock Protein Peptide Complex-96 (HSPPC-96) Vaccine

Circulating microRNAs as Disease Markers in Pediatric Cancers

Safety and Efficacy of 2 Treatment Regimens of Aztreonam for Inhalation Solution in Children With Cystic Fibrosis and New Onset Pseudomonas Aeruginosa Infection

Gene Transfer Clinical Study in X-Linked Myotubular Myopathy

Registry of Patients Treated With Systemic Mold-Active Triazoles

A Study of Ribociclib and Everolimus Following Radiation Therapy in Children With Newly Diagnosed Non-biopsied Diffuse Pontine Gliomas (DIPG) and RB+ Biopsied DIPG and High Grade Gliomas (HGG)

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy (DMD)

Safety, Tolerability, Efficacy and Pharmacokinetics of Copanlisib in Pediatric Patients

Study to Evaluate Safety and Antiviral Activity of Doses of JNJ-53718678 in Children (>=28 Days to

Characterization of Lipoprotein Composition and Function in Pediatric Psoriasis Before and After Treatment

Long-term Safety With Vedolizumab Intravenous (IV) in Pediatric Participants With Ulcerative Colitis (UC) or Crohn's Disease (CD)

Genetic Analysis of Pediatric Psoriasis

Non-Invasive Diagnosis of Pediatric Pulmonary Invasive Mold Infections

A Prospective Database of Infants With Cholestasis

The Treatment of Type I Open Fractures in Pediatrics

Temozolomide, Cixutumumab, and Combination Chemotherapy in Treating Patients With Metastatic Rhabdomyosarcoma

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

Haplo-identical SCT for HR Hematologic Malignancies w/Post-Transplant In-Vivo T-cell Depletion

Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)

Longitudinal Changes in Exercise Capacity in Children and Young Adults With Sickle Cell Anemia

Longitudinal Study of Biomarkers

Effects of Medical Complexity Using GMA on Lurie Children's In- and Outpatients

A Study to Assess the Efficacy and Safety of IONIS-SMN Rx in Infants With Spinal Muscular Atrophy

The Safety and Efficacy of Supraglottic Airway Use in Children

Phase 1 Pediatric Pharmacokinetics/Pharmacodynamics (PK/PD) Study

Expanded Access Protocol to Provide Brincidofovir for the Treatment of Serious Adenovirus Infection or Disease

Trial of Panobinostat in Children With Diffuse Intrinsic Pontine Glioma

Effectiveness Trial of an E-Health Intervention To Support Diabetes Care in Minority Youth (3Ms)

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

Study to Assess Safety and Preliminary Activity of Eribulin Mesylate in Pediatric Participants With Relapsed/Refractory Rhabdomyosarcoma (RMS), Non-rhabdomyosarcoma Soft Tissue Sarcoma (NRSTS) and Ewing Sarcoma (EWS)

Inhaled Tissue Plasminogen Activator for Acute Plastic Bronchitis

Study of Lenvatinib in Combination With Everolimus in Recurrent and Refractory Pediatric Solid Tumors, Including Central Nervous System Tumors

Abatacept for GVHD Prophylaxis After Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell Disease

A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Patients With Developmental and/or Epileptic Encephalopathies