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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

Brain Involvement in Dystrophinopathies Part 2

NCT04668716

Conditions
Autism Spectrum Disorder, Autistic Disorder, Becker Muscular Dystrophy, +14 more
Locations
  • Copenhagen Neuromuscular Center Copenhagen, Denmark
  • Imagine Institut Des Maladies Genetiques Necker Enfants Malades Fondation Paris, France
  • Universita Cattolica Del Sacro Cuore Rome, Italy
  • STICHTING KEMPENHAEGHE/Leiden University Medical Center Heeze, Netherlands
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Completed

Vitamin D3 For CGD Patients With BCGosis/Itis

NCT03984890

Conditions
BCG, Chronic Disease, Chronic-granulomatous Disease, +14 more
Locations
  • Children's Hospital of Fudan University Shanghai, Shanghai Municipality, China
  • Children's Hospital of Fudan University Shanghai, Shanghai Municipality, China
View Trial Details
Completed

THC Crossover Study

NCT04429568

Conditions
Behavior, Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, +34 more
Locations
  • Zuckerberg San Francisco General Hospital San Francisco, California, United States
  • University of California, San Francisco San Francisco, California, United States
View Trial Details
Completed

Dose Confirmation Trial of AAV5-hFIXco-Padua

NCT03489291

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +11 more
Locations
  • Phoenix Childrens Hospital Phoenix, Arizona, United States
  • University of California, Davis Sacramento, California, United States
  • University of California, San Diego San Diego, California, United States
  • University of Michigan Ann Arbor, Michigan, United States
View Trial Details
Completed

A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

NCT04007536

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +22 more
Locations
  • UCSF Benioff Children's Hospital Oakland, California, United States
  • UNC Children's Research Institute Chapel Hill, North Carolina, United States
  • UPMC | Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
  • Erasmus Medical Center Rotterdam, South Holland, Netherlands
View Trial Details
Completed

An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B

NCT02554773

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +8 more
Locations
  • Clinical Trial Site Ann Arbor, Michigan, United States
  • Clinical Trial Site Pittsburgh, Pennsylvania, United States
  • Clinical Trial Site Plovdiv, Bulgaria
  • Clinical Trial Site Sofia, Bulgaria
View Trial Details
Completed

A Study to Evaluate Ketamine for the Treatment of Rett Syndrome

NCT03633058

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +7 more
Locations
  • University of Alabama Birmingham School of Medicine Birmingham, Alabama, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Rush University Medical Center Chicago, Illinois, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
View Trial Details
Completed

A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

NCT03852498

Conditions
Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +22 more
Locations
  • Lucile Packard Children's Hospital Palo Alto, California, United States
  • Boston Children's Hospital/Massachusetts General Hospital Boston, Massachusetts, United States
  • University of Minnesota Minneapolis, Minnesota, United States
  • Hôpital Robert Debré Paris, France
View Trial Details

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