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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.

1,243 public trials 227 recruiting studies
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Genetic Diseases, X-Linked trial results

Completed

Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

NCT02369731

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +7 more
Locations
  • Krankenhaus der Barmherzigen Schwestern Linz Linz, Austria
  • Sozialmedizinisches Zentrum Süd Kaiser-Franz-Josef Spital mit Gottfried von Preyer´schen Kinderspital Vienna, Austria
  • Hospital das Clínicas da Universidade de São Paulo São Paulo, Brazil
  • The University Hospital Brno Brno, Czechia
View all 71 locations
View Trial Details
Completed

Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease

NCT05843916

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Instituto de Nefrología Pergamino S.R.L Pergamino, Buenos Aires, Argentina
  • Centro Médico Santa María de la Salud San Isidro, Buenos Aires, Argentina
  • Instituto de Investigaciones Clínicas Quilmes Buenos Aires, Argentina
  • Clínica Universitaria Reina Fabiola Córdoba, Argentina
View Trial Details
Completed

Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

NCT04049760

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of South Florida Tampa, Florida, United States
  • The Emory Clinic Atlanta, Georgia, United States
  • University of Minnesota Masonic Children's Hospital and Clinics Minneapolis, Minnesota, United States
  • Cincinnati Children's Hospital Cincinnati, Ohio, United States
View Trial Details
Completed

A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.

NCT04281537

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Amicus Therapeutics, Inc. Philadelphia, Pennsylvania, United States
  • Instituto de Genética e Erros Inatos do Metabolismo (IGEIM) São Paulo, Brazil
  • Keio University Hospital Tokyo, Japan
  • Yokohama Municipal Citizen's Hospital Yokohama, Japan
View Trial Details
Completed

A Randomized Study of BPN14770 in Male Adolescents (Aged 9 to < 18 Years) With Fragile X Syndrome

NCT05163808

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
  • UC Davis Sacramento, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
View all 17 locations
View Trial Details
Completed

Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

NCT05386680

Conditions
Atrophy, Bulbo-Spinal Atrophy, X-Linked, Central Nervous System Diseases, +19 more
Locations
  • Boston Childrens Hospital Boston, Massachusetts, United States
  • Child Hosp Of The Kings Daughters Norfolk, Virginia, United States
  • University of Wisconsin Madison Medical School Madison, Wisconsin, United States
  • Novartis Investigative Site Parkville, Victoria, Australia
View all 13 locations
View Trial Details

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