Cyclophosphamide and Prednisone With or Without Immunoglobulin in Treating Abnormal Muscle Movement in Children With Neuroblastoma

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

A 12-Month Study To Evaluate The Safety And Tolerability Of Pregabalin As Add-On Therapy In Pediatric Subjects 1 Month To 16 Years Of Age With Partial Onset Seizures And Pediatric And Adult Subjects 5 To 65 Years Of Age With Primary Generalized Tonic-Clonic Seizures

The Childhood and Adolescent Migraine Prevention Study

A Study Investigating the Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy

Post Approval Study of Liposorber LA-15 System for the Treatment of Focal Segmental Glomerulosclerosis in Children

Apixaban for the Acute Treatment of Venous Thromboembolism in Children

A Study of the Safety and Effectiveness of Apixaban in Preventing Blood Clots in Children With Leukemia Who Have a Central Venous Catheter and Are Treated With Asparaginase

Exercise Versus DDAVP in Patients With Mild Hemophilia A

Computational Simulation of Patellar Instability

The Pediatric Anesthesia Quality Improvement Project

Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)

Study of Perampanel as Adjunctive Treatment for Inadequately Controlled Seizures Associated With Lennox-Gastaut Syndrome

A Trial to Evaluate the Safety and Efficacy of Elamipretide in Subjects With Primary Mitochondrial Myopathy Followed by an Open-Label Extension

Efficacy & Safety Of Zomig Nasal Spray For Acute Migraine Treatment In Subjects 6 To 11 Years, With OLE

Study Assessing the Safety, Tolerability, Pharmacokinetics, Food Effect, and Drug-Drug Interactions of PTI-801 in Healthy Volunteers, and Safety, Tolerability, and Pharmacokinetics of PTI-801 in Subjects With Cystic Fibrosis

A Study to Assess the Efficacy, Safety, and Tolerability of Cannabidiol Oral Solution With Vigabatrin as Initial Therapy in Participants With Infantile Spasms

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

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

Timing of Inguinal Hernia Repair in Premature Infants

Cannabidiol Oral Solution in Pediatric Participants With Treatment-Resistant Childhood Absence Seizures

Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Pediatric Participants With Treatment-Resistant Childhood Absence Seizures

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

I Change Adherence & Raise Expectations

Pediatric Vasculitis Initiative

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

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

Continued Study of Artificial Vision: Evaluation of the BrainPort® System and Investigation of Visual Ambulation

Building Evidence for Effective Palliative/End of Life Care for Teens With Cancer

Standardized Treatment of Pulmonary Exacerbations II

Pharmacokinetic Study With an Oral Suspension of Perampanel as Adjunctive Therapy in Pediatric Subjects With Epilepsy

A Study of AeroVanc for the Treatment of MRSA Infection in CF Patients

Ultrasound Guided Adductor Canal Block Versus Femoral Nerve Block for Anterior Cruciate Ligament Repair in Pediatrics

A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

Evaluation of the Duration of Therapy for Thrombosis in Children

Indirect Intracranial Pressure Measurement in Patients With Suspected or Documented Concussion

HeadStart4: Newly Diagnosed Children (

Study Assessing PTI-428 Safety, Tolerability, Pharmacokinetics and Effect in Subjects With Cystic Fibrosis

Non-invasive Intracranial Pressure (ICP) Monitoring in Pediatric Patients