Skip to main content
OpenTrials

Clinical trial condition

Genetic Diseases, Inborn

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

12,603 public trials 2,158 recruiting studies
Refine in advanced search Browse all conditions

Genetic Diseases, Inborn trial results

Active, not recruiting

Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

NCT06545136

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Lysosomal Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
  • Hospital de Clinicas de Porto Alegre (HCPA) Porto Alegre, Brazil
  • Hospital Quironsalud Zaragoza Zaragoza, Spain
  • Salford Royal Hospital Salford, United Kingdom
View Trial Details
Active, not recruiting

Prospective Study of Pregnancy in Women With Cystic Fibrosis

NCT04828382

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +6 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • University of Arkansas for Medical Sciences Little Rock, Arkansas, United States
  • University of California San Diego La Jolla, California, United States
  • National Jewish Health Denver, Colorado, United States
View all 40 locations
View Trial Details
Active, not recruiting

Open-label Extension of the HOPE-2 Trial

NCT04428476

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • University of California, Davis Sacramento, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Washington University St Louis, Missouri, United States
  • Cincinnati Children's Hospital Medical Center Cincinnati, Ohio, United States
View Trial Details
Active, not recruiting

Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

NCT05164055

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations
  • Investigational Site Number : 2500004 Bordeaux, France
  • Investigational Site Number : 2500005 Brest, France
  • Investigational Site Number : 2500008 Clermont-Ferrand, France
  • Investigational Site Number : 2500009 Lille, France
View Trial Details
Active, not recruiting

A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

NCT05515536

Conditions
Brain Diseases, Central Nervous System Diseases, Cerebellar Diseases, +11 more
Locations
  • UCLA Los Angeles, California, United States
  • University of Florida Gainesville, Florida, United States
  • University of South Florida Tampa, Florida, United States
  • University of Iowa Iowa City, Iowa, United States
View all 14 locations
View Trial Details
Active, not recruiting

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

NCT03911505

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • University of Florida Clinical Research Center Gainesville, Florida, United States
  • Wolfson Children's Hospital Jacksonville, Florida, United States
  • Woodruff Memorial Research Building Atlanta, Georgia, United States
  • St. Louis Children's Hospital St Louis, Missouri, United States
View all 17 locations
View Trial Details
Active, not recruiting

Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease

NCT04120493

Conditions
Basal Ganglia Diseases, Brain Diseases, Central Nervous System Diseases, +14 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • University of Arizona (Surgical Site Only) Tucson, Arizona, United States
  • University of California, San Francisco San Francisco, California, United States
  • CenExel Rocky Mountain Clinical Research Englewood, Colorado, United States
View Trial Details

Related conditions