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Clinical trial condition

Genetic Diseases, Inborn

Explore clinical trials studying Genetic Diseases, Inborn. Study availability and eligibility vary by location and protocol.

12,603 public trials 2,158 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia

NCT06832371

Conditions
Arterial Occlusive Diseases, Arteriosclerosis, Atherosclerosis, +18 more
Locations
  • CHRU Lille - Centre Hospitalier Universitaire de Lille Lille, France
  • Centre Hospitalier Universitaire de Lyon (CHU Lyon) Lyon, France
  • Hôpital de la Conception - Assistance Publique Hôpitaux de Marseille (AP-HM) Marseille, France
  • Hôpital La Pitié Salpêtrière - Groupe Hospitalier "La Pitié Salpêtrière - Charles Foix" Paris, France
View all 30 locations
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Active, not recruiting

Study to Evaluate Safety of RTx-015 Injection in Retinitis Pigmentosa or Choroideremia Patients (ENVISION)

NCT06460844

Conditions
Choroid Diseases, Choroideremia, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +9 more
Locations
  • Retina Vitreous Associates Medical Group Beverly Hills, California, United States
  • UCI Alpha Clinic Orange, California, United States
  • UPMC Vision Institute Pittsburgh, Pennsylvania, United States
  • Retina Consultants of Texas Research Centers Bellaire, Texas, United States
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Active, not recruiting

A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)

NCT05876780

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Limb Girdle Muscular Dystrophy, +7 more
Locations
  • Nationwide Children's Hospital Columbus, Ohio, United States
  • St. Jude Children's Research Hospital Memphis, Tennessee, United States
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Active, not recruiting

A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A

NCT04158934

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +6 more
Locations
  • UF Health Shands Hospital Gainesville, Florida, United States
  • SHAT of Oncohaematology Diseases Sofia, Bulgaria
  • Clinical Hospital Sveti Duh Zagreb, Croatia
  • University hospital centre Zagreb Zagreb, Croatia
View all 36 locations
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Active, not recruiting

Epidemiology of Silent and Overt Strokes in Sickle Cell Disease

NCT03376893

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +16 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • Washington University School of Medicine St Louis, Missouri, United States
  • Vanderbilt University Medical Center Nashville, Tennessee, United States
  • Vanderbilt University Medical Center Nashville, Tennessee, United States
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Active, not recruiting

Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE

NCT06669754

Conditions
Angioedema, Angioedemas, Hereditary, Cardiovascular Diseases, +14 more
Locations
  • Study Site Little Rock, Arkansas, United States
  • Study Site San Diego, California, United States
  • Study site Santa Monica, California, United States
  • Study site Walnut Creek, California, United States
View all 56 locations
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