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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.

12,522 public trials 2,142 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia

NCT06597006

Conditions
Aortic Valve Disease, Aortic Valve Stenosis, Cardiovascular Diseases, +18 more
Locations
  • UC San Francisco Medical Center San Francisco, California, United States
  • Childrens National Hospital Washington D.C., District of Columbia, United States
  • Washington Univ School Of Medicine St Louis, Missouri, United States
  • Primary Childrens Medical Center Salt Lake City, Utah, United States
View all 19 locations
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Recruiting

A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)

NCT07159841

Conditions
Adrenal Gland Diseases, Adrenal Hyperplasia, Congenital, Adrenogenital Syndrome, +17 more
Locations
  • UCSF Ron Conway Gateway Medical Building San Francisco, California, United States
  • Boston's Children's Hospital Boston, Massachusetts, United States
  • University of Michigan Ann Arbor, Michigan, United States
  • University of Minnesota Minneapolis, Minnesota, United States
View all 43 locations
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Recruiting

A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)

NCT06439082

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +9 more
Locations
  • University Of Alabama Birmingham, Alabama, United States
  • Ctr for Inherited Blood Disorders Orange, California, United States
  • Childrens National Hospital Washington D.C., District of Columbia, United States
  • University of Florida Jacksonville, Florida, United States
View all 32 locations
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