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Clinical trial condition

Genetic Diseases, Inborn

Explore clinical trials studying Genetic Diseases, Inborn. Study availability and eligibility vary by location and protocol.

449 public trials 107 recruiting studies
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Genetic Diseases, Inborn trial results

Active, Not Recruiting

VIGOR: Virtual Genome Center for Infant Health

NCT05205356

Conditions
Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, +7 more
Locations
  • USA Children's and Women's Hospital Mobile, Alabama, United States
  • Holtz Children's Hospital at Jackson Memorial Medical Center Miami, Florida, United States
  • Boston Medical Center Boston, Massachusetts, United States
  • Baystate Medical Center Springfield, Massachusetts, United States
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Active, Not Recruiting

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

NCT05356195

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +8 more
Locations
  • TriStar Medical Group Children's Specialists - Pediatric Oncology Nashville, Tennessee, United States
  • Hospital for Sick Children - Hematology Toronto, Canada
  • University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology Düsseldorf, Germany
  • IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica Rome, Italy
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Active, Not Recruiting

A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome

NCT05898620

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +12 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Nicklaus Children's Hospital Research Institute Miami, Florida, United States
View all 16 locations
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Active, Not Recruiting

A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

NCT05126758

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +7 more
Locations
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • UCSD Altman Clinical and Translational Research Institute La Jolla, California, United States
  • Children's Hospital of Los Angeles, Division of Neurology Los Angeles, California, United States
View all 20 locations
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