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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 and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)

NCT04851873

Conditions
Atrophy, Bulbo-Spinal Atrophy, X-Linked, Central Nervous System Diseases, +19 more
Locations
  • Novartis Investigative Site Boston, Massachusetts, United States
  • Novartis Investigative Site St Louis, Missouri, United States
  • Novartis Investigative Site Randwick, New South Wales, Australia
  • Novartis Investigative Site Leuven, Belgium
View all 13 locations
View Trial Details
Completed

Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)

NCT05073133

Conditions
Atrophy, Bulbo-Spinal Atrophy, X-Linked, Central Nervous System Diseases, +18 more
Locations
  • Novartis Investigative Site CABA, Buenos Aires, Argentina
  • Novartis Investigative Site Buenos Aires, Argentina
  • Novartis Investigative Site Belo Horizonte, Minas Gerais, Brazil
  • Novartis Investigative Site Porto Alegre, Rio Grande do Sul, Brazil
View Trial Details
Completed

A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease

NCT04974749

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Peking Union Medical College Hospital Beijing, China
  • Xiangya Hospital, Central South University Changsha, China
  • West China Hospital, Sichuan University Chengdu, China
  • The Children's Hospital of Zhejiang University School of Medicine Hangzhou, China
View Trial Details
Completed

A Study to Evaluate the Real-world Usage and Effectiveness of Elocta and Alprolix in Patients With Haemophilia A or B

NCT03055611

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +10 more
Locations
  • Swedish Orphan Biovitrum Reserach site Berlin, Germany
  • Swedish Orphan Biovitrum Reserach Site Blaubeuren Abbey, Germany
  • Swedish Orphan Biovitrum Research Site Bonn, Germany
  • Swedish Orphan Biovitrum Research Site Delmenhorst, Germany
View all 22 locations
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Completed

A Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in France

NCT03655340

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +8 more
Locations
  • Swedish Orphan Biovitrum Research Site (CHU de Bordeaux) Bordeaux, France
  • Swedish Orphan Biovitrum Research Site Brest, France
  • Swedish Orphan Biovitrum Research Site Caen, France
  • Swedish Orphan Biovitrum Research Site Chambéry, France
View all 18 locations
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Completed

A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy

NCT03760029

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Children's Hospital of Chongqing Medical University Chongqing, Chongqing Municipality, China
  • Children's Hospital of Chongqing Medical University (Liangjiang Branch) Chongqing, Chongqing Municipality, China
  • The First Affiliated Hospital Of Fujian Medical University Fuzhou, Fujian, China
  • Peking University First Hospital Beijing, China
View Trial Details

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