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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.

1,242 public trials 227 recruiting studies
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Genetic Diseases, X-Linked trial results

Active, Not Recruiting

Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy

NCT06100887

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • UCLA Medical Center Los Angeles, California, United States
  • UC Davis Medical Center Sacramento, California, United States
  • University of Florida Gainesville, Florida, United States
  • University of Massachusetts Memorial Medical Center Worcester, Massachusetts, United States
View Trial Details
Active, Not Recruiting

Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment

NCT06031259

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +17 more
Locations
  • M.A.G.I.C. Clinic Calgary, Alberta, Canada
  • University of British Columbia Vancouver, British Columbia, Canada
  • Hospital for Sick Children Toronto, Ontario, Canada
  • Hopital Femme Mere Enfant Bron, Auvergne-Rhône-Alpes, France
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
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Active, Not Recruiting

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

NCT06152237

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +9 more
Locations
  • Rush University Medical Center & Children's Hospital Chicago, Illinois, United States
  • Gillette Children's Specialty Healthcare Saint Paul, Minnesota, United States
  • Washington University, St. Louis St Louis, Missouri, United States
  • CHU Ste-Justine Montreal, Quebec, Canada
View Trial Details
Active, Not Recruiting

An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome

NCT05367960

Conditions
Chromosome Disorders, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +10 more
Locations
  • Amnova Clinical Research Irvine, California, United States
  • Thompson Autism & Neurodevelopment Center - CHOC Orange, California, United States
  • MIND Institute UC Davis Medical Center Sacramento, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
View all 17 locations
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Active, Not Recruiting

A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome

NCT03837483

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +13 more
Locations
  • Children's Healthcare of Atlanta, Inc Atlanta, Georgia, United States
  • Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET) Milan, Italy
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Active, Not Recruiting

A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD）

NCT04587908

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • A site selected by Taiho Pharmaceutical Co., Ltd. Aichi, Japan
  • A site selected by Taiho Pharmaceutical Co., Ltd. Fukuoka, Japan
  • A site selected by Taiho Pharmaceutical Co., Ltd. Hokkaido, Japan
  • A site selected by Taiho Pharmaceutical Co., Ltd. Osaka, Japan
View Trial Details

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