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Clinical trial condition

Genetic Diseases, X-Linked

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

33 public trials 10 recruiting studies
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Genetic Diseases, X-Linked trial results

Completed

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

NCT03734588

Conditions
Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, +18 more
Locations
  • Orthopaedic Institute for Children Los Angeles, California, United States
  • Illinois Bleeding and Clotting Disorders Institute Peoria, Illinois, United States
  • University of Michigan Ann Arbor, Michigan, United States
  • Mississippi Center for Advanced Medicine Madison, Mississippi, United States
View Trial Details
Completed

Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants

NCT03876301

Conditions
Blood Coagulation Disorder, Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, +9 more
Locations
  • University of California San Francisco San Francisco, California, United States
  • University of Florida Gainesville, Florida, United States
  • Emory University Atlanta, Georgia, United States
  • Children's Hospital of Michigan Detroit, Michigan, United States
View Trial Details
Completed

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

NCT03417245

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +8 more
Locations
  • Investigational Site Number 0140 Little Rock, Arkansas, United States
  • Investigational Site Number 128 Gainesville, Florida, United States
  • Investigational Site Number 103 Tampa, Florida, United States
  • Investigational Site Number 102 Chicago, Illinois, United States
View all 64 locations
View Trial Details
Completed

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors

NCT03417102

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +8 more
Locations
  • Investigational Site Number 0117 Phoenix, Arizona, United States
  • Investigational Site Number 0139 Los Angeles, California, United States
  • Investigational Site Number 0135 Orange, California, United States
  • Investigational Site Number 0137 San Diego, California, United States
View all 58 locations
View Trial Details
Completed

Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

NCT01826487

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +7 more
Locations
  • University of California, Los Angeles Los Angeles, California, United States
  • UC Davis Medical Center Sacramento, California, United States
  • Children's Hospital Colorado - Center for Cancer and Blood Disorders Aurora, Colorado, United States
  • Child Neurology Center of Northwest Florida Gulf Breeze, Florida, United States
View all 55 locations
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