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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

A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease

NCT03737214

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of Alabama at Birmingham - Nephrology Research Clinic Birmingham, Alabama, United States
  • University of California Irvine Irvine, California, United States
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • University of Florida Clinical and Translational Science Institute, UF Clinical Research Center Gainesville, Florida, United States
View all 42 locations
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Active, Not Recruiting

Early Check: Expanded Screening in Newborns

NCT03655223

Conditions
17 Alpha-Hydroxylase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, +456 more
Locations
  • RTI International Research Triangle Park, North Carolina, United States
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Active, Not Recruiting

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

NCT06280209

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Fondazione Serena ETS - Centro Clinico NeMO Milano Milan, Italy
  • UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore Rome, Italy
  • Leids Universitair Medisch Centrum Leiden, Netherlands
  • Hospital Sant Joan de Deu Barcelona, Spain
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Active, Not Recruiting

A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease

NCT05280548

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of Alabama -The Kirklin Clinic- Site Number : 8400010 Birmingham, Alabama, United States
  • University of California Los Angeles Medical Center- Site Number : 8400008 Los Angeles, California, United States
  • Emory University School of Medicine - Atlanta- Site Number : 8400009 Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 8400005 Chicago, Illinois, United States
View all 54 locations
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Active, Not Recruiting

NS-050/NCNP-03 in Boys With DMD (Meteor50)

NCT06053814

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Children's Hospital Los Angeles Los Angeles, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • University of Kansas Medical Center Kansas City, Kansas, United States
View all 19 locations
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Active, Not Recruiting

Defining Endpoints in Becker Muscular Dystrophy

NCT05257473

Conditions
Becker Muscular Dystrophy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +10 more
Locations
  • University of California, Irvine Orange, California, United States
  • University of Colorado Anschutz Medical Campus Aurora, Colorado, United States
  • University of Iowa Iowa City, Iowa, United States
  • University of Kansas Medical Center Kansas City, Kansas, United States
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