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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 Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)

NCT04573023

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
Locations
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • University of Minnesota Minneapolis, Minnesota, United States
View all 25 locations
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Active, Not Recruiting

Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

NCT05345171

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
Locations
  • University of California Los Angeles, California, United States
  • University of Colorado Aurora, Colorado, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • University Hospitals Cleveland Medical Center Cleveland, Ohio, United States
View all 16 locations
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Active, Not Recruiting

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B

NCT03861273

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +8 more
Locations
  • UCSF IDS Pharmacy San Francisco, California, United States
  • University of California, San Francisco - Clinical Research Center San Francisco, California, United States
  • University of California, San Francisco - Outpatient Hematology Clinic San Francisco, California, United States
  • The Regents of the University of California, San Francisco campus San Francisco, California, United States
View all 67 locations
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Active, Not Recruiting

Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency

NCT03636438

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
Locations
  • The Children's Hospital Colorado Aurora, Colorado, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
  • Icahn School of Medicine New York, United States
  • University Hospital Cleveland Medical Center/Case Western Reserve University Cleveland, Ohio, United States
View Trial Details
Active, Not Recruiting

Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS

NCT03802799

Conditions
Chromosome Disorders, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +10 more
Locations
  • Southwest Autism Research and Resource Center Phoenix, Arizona, United States
  • Science 37 Culver City, California, United States
  • Amnova Clinical Research, LLC Irvine, California, United States
  • UC Davis Health System, MIND Institute Sacramento, California, United States
View all 26 locations
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Active, Not Recruiting

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

NCT05881408

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
  • Lucile Packard Children's Hospital Stanford Palo Alto, California, United States
  • University of California at Davis Medical Center Sacramento, California, United States
  • Rady Children's Hospital-San Diego San Diego, California, United States
View all 46 locations
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Active, Not Recruiting

Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)

NCT05291091

Conditions
Becker Muscular Dystrophy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +8 more
Locations
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • UC San Diego La Jolla, California, United States
  • UCLA Medical Center Los Angeles, California, United States
  • UC Irvine Medical Center Orange, California, United States
View all 51 locations
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Active, Not Recruiting

Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan

NCT06052800

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Investigational Site Number : 1580004 Taichung, Taiwan
  • Investigational Site Number : 1580005 Tainan, Taiwan
  • Investigational Site Number : 1580001 Taipei, Taiwan
  • Investigational Site Number : 1580003 Taipei, Taiwan
View Trial Details

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