Congenital Clinical Trials in Boston, Massachusetts
27 recruitingBoston, Massachusetts
Showing 1–20 of 27 trials
Recruiting
Phase 2Phase 3
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
Congenital Adrenal HyperplasiaClassic Congenital Adrenal Hyperplasia
Crinetics Pharmaceuticals Inc.153 enrolled35 locationsNCT07159841
Recruiting
Project: Every Child for Younger Patients With Cancer
Malignant Solid NeoplasmNeuroendocrine NeoplasmCentral Nervous System Neoplasm+15 more
Children's Oncology Group75,000 enrolled278 locationsNCT02402244
Recruiting
Phase 1Phase 2
A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
Regeneron Pharmaceuticals30 enrolled16 locationsNCT05788536
Recruiting
Not Applicable
Starlight Cardiovascular Lifeline Ductus Arteriosus Stent IDE Study
Congenital Heart DiseaseCongenital Heart Disease (CHD)
Starlight Cardiovascular Inc35 enrolled8 locationsNCT07114718
Recruiting
Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy
LAMA2-MD (Merosin Deficient Congenital Muscular Dystrophy, MDC1A)
Nationwide Children's Hospital44 enrolled14 locationsNCT06503367
Recruiting
Muscle Health Measurements Using Electrical Impedance Myography
Muscular DystrophiesMyositisCongenital Myopathy+4 more
Beth Israel Deaconess Medical Center150 enrolled1 locationNCT07502989
Recruiting
Phase 2
Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
deaf-blindRetinitis pigmentosa (RP)Retinal Disease+4 more
Laboratoires Thea81 enrolled27 locationsNCT06627179
Recruiting
Molecular and Genetic Studies of Congenital Myopathies
Nemaline MyopathyCentronuclear MyopathyCentral Core Disease+5 more
Boston Children's Hospital4,000 enrolled1 locationNCT00272883
Recruiting
Advanced SPinal Innovations With Robotics and Enabling Technology Registry
SpondylolisthesisAdolescent Idiopathic ScoliosisCongenital Scoliosis+3 more
Boston Children's Hospital700 enrolled10 locationsNCT05071144
Recruiting
Not Applicable
COMPASSION S3 - Evaluation of the SAPIEN 3 Transcatheter Heart Valve in Patients With Pulmonary Valve Dysfunction
Complex Congenital Heart DefectDysfunctional RVOT ConduitPulmonary Valve Insufficiency+1 more
Edwards Lifesciences108 enrolled25 locationsNCT02744677
Recruiting
Extracellular Vesicle Micro RNA Profiling in Congenital Heart Disease: Fetal-Maternal Regulation in Neonatal Thrombosis
ThrombosisCongenital Heart DiseaseSingle Ventricle
Boston Children's Hospital10 enrolled1 locationNCT06434207
Recruiting
Phase 2
A Study to Evaluate Tabelecleucel in Participants With Epstein Barr Virus (EBV) Associated Diseases
LeiomyosarcomaSolid Organ Transplant ComplicationsLymphoproliferative Disorders+8 more
Pierre Fabre Medicament190 enrolled40 locationsNCT04554914
Recruiting
Genetic Studies of Strabismus, Congenital Cranial Dysinnervation Disorders (CCDDs), and Their Associated Anomalies
Facial PalsyCongenital Fibrosis of Extraocular MusclesDuane Retraction Syndrome+23 more
Boston Children's Hospital20,000 enrolled1 locationNCT03059420
Recruiting
Phase 4
De-implementing Inhaled Nitric Oxide for Congenital Diaphragmatic Hernia
Congenital Diaphragmatic Hernia
The University of Texas Health Science Center, Houston600 enrolled19 locationsNCT05213676
Recruiting
Evaluation of Pro-Inflammatory Leukocyte Activity in Patients Undergoing Cardiac Surgery
CoagulationCongenital Heart Disease
Boston Children's Hospital100 enrolled1 locationNCT04299828
Recruiting
Genetic Studies of Strabismus, Nystagmus, and Associated Disorders
StrabismusNystagmus, Congenital
Boston Children's Hospital400 enrolled1 locationNCT04770519
Recruiting
The Myelin Disorders Biorepository Project
AdrenoleukodystrophyCockayne SyndromeRefsum Disease+63 more
Children's Hospital of Philadelphia12,000 enrolled23 locationsNCT03047369
Recruiting
Clinical and Basic Investigations Into Congenital Disorders of Glycosylation
Congenital Disorders of Glycosylation
Icahn School of Medicine at Mount Sinai500 enrolled12 locationsNCT04199000
Recruiting
Phase 2
Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)
Rare DiseasesCongenital DisordersMicrovillus Inclusion Disease
Napo Pharmaceuticals, Inc.12 enrolled3 locationsNCT06721871
Recruiting
Identification of Biomarkers for Patients with Vascular Anomalies
Vascular AnomalyKlippel Trenaunay SyndromeKaposiform Hemangioendothelioma+4 more
Children's Hospital Medical Center, Cincinnati1,000 enrolled2 locationsNCT03001180