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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.

449 public trials 107 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

Evaluate the Effect of Obicetrapib in Patients With HeFH on Top of Maximum Tolerated Lipid-Modifying Therapies.

NCT05425745

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Familial Hypercholesterolemia, +14 more
Locations
  • Site 01022 Jonesboro, Arkansas, United States
  • Site 01015 Toluca Lake, California, United States
  • Site 01009 Sarasota, Florida, United States
  • Site 01023 Boise, Idaho, United States
View all 96 locations
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Completed

A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

NCT03406780

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +7 more
Locations
  • University of California, Davis Sacramento, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Nemours Children's Hospital Orlando, Florida, United States
  • University of Iowa Iowa City, Iowa, United States
View Trial Details
Completed

Gene Therapy Study in Severe Haemophilia A Patients (270-201)

NCT02576795

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Cardiovascular Diseases, +10 more
Locations
  • Queen Elizabeth Hospital Birmingham Birmingham, United Kingdom
  • Addenbrooke's Hospital Cambridge, United Kingdom
  • St. Thomas' Hospital London, United Kingdom
  • The Royal London Hospital London, United Kingdom
View Trial Details
Completed

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)

NCT03370913

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Cardiovascular Diseases, +9 more
Locations
  • Los Angeles Orthopedic Hospital, Orthopedic Hemophilia Treatment Center Los Angeles, California, United States
  • UC Davis Hemophilia Treatment Center Sacramento, California, United States
  • University of California San Diego, Hematology and Oncology, Hemophilia &Thrombosis Treatment Center San Diego, California, United States
  • UCSF Medical Center San Francisco, California, United States
View all 49 locations
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Completed

A Gene Transfer Study for Hemophilia A

NCT03003533

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations
  • University of California Davis - Hemostasis and Thrombosis Center Sacramento, California, United States
  • University of Florida Health Gainesville, Florida, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
  • Mississippi Center for Advanced Medicine Madison, Mississippi, United States
View all 16 locations
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