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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.

1,244 public trials 227 recruiting studies
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Genetic Diseases, X-Linked trial results

Completed

Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy

NCT02024932

Conditions
Bulbo-Spinal Atrophy, X-Linked, Central Nervous System Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +10 more
Locations
  • Novartis Investigative Site Orange, California, United States
  • National Institutes of Health Bethesda, Maryland, United States
  • Novartis Investigative Site Columbus, Ohio, United States
  • Novartis Investigative Site Copenhagen, Denmark
View Trial Details
Completed

Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome

NCT01253629

Conditions
Chromosome Disorders, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +10 more
Locations
  • Novartis Investigative Site Phoenix, Arizona, United States
  • Novartis Investigative Site Sacramento, California, United States
  • Novartis Investigative Site Decatur, Georgia, United States
  • Novartis Investigative Site Chicago, Illinois, United States
View all 31 locations
View Trial Details
Completed

Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

NCT03039686

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Neuromuscular Research Center Phoenix, Arizona, United States
  • Stanford University Palo Alto, California, United States
  • University of California Davis Medical Center Sacramento, California, United States
  • Yale University School of Medicine ; Pulmonary & Critical Care New Haven, Connecticut, United States
View all 44 locations
View Trial Details
Completed

Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease

NCT02194985

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Clinical Study Site Atlanta, Georgia, United States
  • Clinical Study Site Kansas City, Kansas, United States
  • Clinical Study Site Grand Rapids, Michigan, United States
  • Clinical Study Site New York, United States
View all 28 locations
View Trial Details
Completed

A Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B Patients

NCT02035605

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +8 more
Locations
  • Clinical Trial Site Pittsburgh, Pennsylvania, United States
  • Clinical Trial Site Plovdiv, Bulgaria
  • Clinical Trial Site Sofia, Bulgaria
  • Clinical Trial Site Varna, Bulgaria
View all 14 locations
View Trial Details
Completed

Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia B

NCT01425723

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +8 more
Locations
  • Research Site Phoenix, Arizona, United States
  • Research Site Sacramento, California, United States
  • Research Site Aurora, Colorado, United States
  • Research Site Atlanta, Georgia, United States
View all 49 locations
View Trial Details
Completed

Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B

NCT01440946

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations
  • Research Site Phoenix, Arizona, United States
  • Research Site Sacramento, California, United States
  • Research Site Atlanta, Georgia, United States
  • Research Site Honolulu, Hawaii, United States
View all 15 locations
View Trial Details
Completed

Phase I/IIa Study of FIXFc in Hemophilia B Patients

NCT00716716

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations
  • RUSH University Medical Center Chicago, Illinois, United States
  • Indiana Hemophilia & Thrombosis Center Indianapolis, Indiana, United States
  • Brigham & Women's Hospital Boston, Massachusetts, United States
  • University of North Carolina Medical School Chapel Hill, North Carolina, United States
View Trial Details

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