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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

Recruiting

ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders

NCT04398628

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +34 more
Locations
  • Arizona Hemophilia and Thrombosis Treatment Center at Phoenix Children's Hospital Phoenix, Arizona, United States
  • Arkansas Center for Bleeding Disorders Little Rock, Arkansas, United States
  • Orthopaedic Institute for Children HTC Los Angeles, California, United States
  • Childrens Hospital Los Angeles Los Angeles, California, United States
View all 71 locations
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Recruiting

Clinical Trial in Patients With Barth Syndrome- 4TAZPower

NCT07531251

Conditions
Abnormalities, Multiple, Barth Syndrome, Cardiovascular Abnormalities, +12 more
Locations
  • Trial Not Offered in the U.S Needham, Massachusetts, United States
  • Metabolics and Genetics in Canada (MAGIC) Calgary, Alberta, Canada
  • Bristol Royal Hospital for Children Upper Maudlin Street Paul O'Gorman Building Bristol, United Kingdom
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Recruiting

Rare Kidney Stone Consortium Patient Registry

NCT00588562

Conditions
APRT Deficiency, Adenine phosphoribosyltransferase deficiency, Carbohydrate Metabolism, Inborn Errors, +19 more
Locations
  • Dent Disease Registry -Mayo Clinic Rochester, Minnesota, United States
  • Primary Hyperoxaluria Registry - Mayo Clinic Rochester, Minnesota, United States
  • Cystinuria Registry - New York University New York, United States
  • APRT Registry - Landspitali Universtiy Hospital Reykjavik, Iceland
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Recruiting

A Study of Patients With Fabry Disease (US Specific)

NCT06906367

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • UAB Nephrology Research Clinic at Paula Building Birmingham, Alabama, United States
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • Emory Genetics Atlanta, Georgia, United States
  • Washington University School of Medicine St Louis, Missouri, United States
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Recruiting

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

NCT06379789

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations
  • Orthopaedic Hemophilia Treatment Center Los Angeles, California, United States
  • David Geffen School of Medicine at UCLA Los Angeles, California, United States
  • Children's Hospital Los Angeles Los Angeles, California, United States
  • University of California Davis Sacramento, California, United States
View all 44 locations
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Recruiting

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

NCT06138639

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • University of California, Los Angeles Medical Center Los Angeles, California, United States
  • University of California, Davis Sacramento, California, United States
  • University of California San Diego, California, United States
View all 15 locations
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Recruiting

Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting

NCT07109375

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • AUSL Toscana Sud-Est - Ospedale San Donato Arezzo, Italy
  • Azienda Ospedaliera Universitaria Consorziale Policlinico Di Bari Bari, Italy
  • Irccs Azienda Ospedaliero-Universitaria Di Bologna - Policlinico Di Sant'Orsola Bologna, Italy
  • A.O. Sant'Anna e San Sebastiano Caserta, Italy
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