Skip to main content
OpenTrials

Clinical trial condition

Genetic Diseases, X-Linked

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

1,244 public trials 227 recruiting studies
Refine in advanced search Browse all conditions

Genetic Diseases, X-Linked trial results

Completed

Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency

NCT02991144

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
Locations
  • The Children's Hospital Colorado Aurora, Colorado, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
  • Icahn School of Medicine at Mount Sinai New York, United States
  • University Hospital Cleveland Medical Center/Case Western Reserve University Cleveland, Ohio, United States
View Trial Details
Completed

Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

NCT03167255

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • UC Davis Sacramento, California, United States
  • Lurie Children's Hospital Chicago, Illinois, United States
  • Washington University St Louis, Missouri, United States
  • Duke University Medical Center Durham, North Carolina, United States
View Trial Details
Completed

Tamoxifen in Duchenne Muscular Dystrophy

NCT03354039

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Hôpitaux Raymond Poincaré Garches, France
  • Hôpital de Hautepierre Strasbourg, France
  • DRK Klinik Berlin Westend Berlin, Germany
  • Universitätsklinikum Essen Essen, Germany
View Trial Details
Completed

Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)

NCT01998633

Conditions
Blood Protein Disorders, Chronic Active Epstein-Barr Virus Infection, Chronic Disease, +26 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • Children's National Medical Center Washington D.C., District of Columbia, United States
  • Children's Healthcare of Atlanta Atlanta, Georgia, United States
  • Ann and Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
View all 22 locations
View Trial Details
Completed

A Study of JR-141 in Patients With Mucopolysaccharidosis II

NCT03568175

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
Locations
  • Fukui Clinical site Fukui, Japan
  • Fukuoka Clinical site 2 Fukuoka, Japan
  • Fukuoka Clinical site Fukuoka, Japan
  • Gifu Clinical site Gifu, Japan
View all 19 locations
View Trial Details
Completed

A Study of JR-141 in Patients With Mucopolysaccharidosis Type II

NCT03128593

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
Locations
  • Gifu Clinical site Gifu, Japan
  • Fukuoka Clinical site Kurume, Japan
  • Tokyo clinical site1 Minato, Japan
  • Osaka Clinical site1 Osaka, Japan
View Trial Details
Completed

AAV Gene Therapy Screening/Observational Protocol (ECLIPSE)

NCT04272554

Conditions
Blood Coagulation Disorder, Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, +9 more
Locations
  • Phoenix Childrens Hospital Phoenix, Arizona, United States
  • Children's Hospital of Los Angeles Los Angeles, California, United States
  • University of California - Davis Sacramento, California, United States
  • University of South Florida Tampa, Florida, United States
View all 56 locations
View Trial Details

Related conditions