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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.

12,674 public trials 2,143 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis

NCT03912233

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations
  • Keck Medical Center of University of Southern California Los Angeles, California, United States
  • Kaiser Permanente Oakland, California, United States
  • University of Kentucky. Lexington, Kentucky, United States
  • Tulane Medical Center New Orleans, Louisiana, United States
View all 26 locations
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Completed

An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia

NCT02604433

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +9 more
Locations
  • Children's Hospital of Los Angeles Los Angeles, California, United States
  • Children's Hospital and Research Center at Oakland Oakland, California, United States
  • Ann and Robert H Lurie Childrens Hospital of Chicago Chicago, Illinois, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
View all 76 locations
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