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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.

12,597 public trials 2,159 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

A Phase 2 Study of Belzutifan (PT2977, MK-6482) for the Treatment of Von Hippel Lindau (VHL) Disease-Associated Renal Cell Carcinoma (RCC) (MK-6482-004)

NCT03401788

Conditions
Abnormalities, Multiple, Angiomatosis, Cardiovascular Diseases, +14 more
Locations
  • National Institutes of Health Clinical Center Bethesda, Maryland, United States
  • Massachusetts General Hospital Boston, Massachusetts, United States
  • University of Michigan Ann Arbor, Michigan, United States
  • University of Pennsylvania Medical Center Philadelphia, Pennsylvania, United States
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Active, not recruiting

Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema

NCT05897424

Conditions
Alpha 1-Antitrypsin Deficiency, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, +9 more
Locations
  • UAB Lung Health Center- Site Number : 105 Birmingham, Alabama, United States
  • St Joseph's Hospital and Medical Center- Site Number : 126 Phoenix, Arizona, United States
  • David Geffen School of Medicine at UCLA- Site Number : 124 Los Angeles, California, United States
  • University of California Davis Medical Center- Site Number : 110 Sacramento, California, United States
View all 40 locations
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Active, not recruiting

Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD

NCT03458832

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Facioscapulohumeral Muscular Dystrophy, Genetic Diseases, Inborn, +7 more
Locations
  • University of California Los Angeles Los Angeles, California, United States
  • University of Kansas Medical Center Kansas City, Kansas, United States
  • Kennedy Krieger Institute Baltimore, Maryland, United States
  • University of Rochester Medical Center Rochester, New York, United States
View all 14 locations
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Active, not recruiting

A Study About the Long-Term Safety of TAK-881 in People With Primary Immunodeficiency Diseases

NCT06076642

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Immune System Diseases, +3 more
Locations
  • University of California, Irvine Medical Sciences I Irvine, California, United States
  • Allergy & Asthma Clinical Research Walnut Creek, California, United States
  • National Jewish Medical And Research Center Denver, Colorado, United States
  • University of South Florida St. Petersburg, Florida, United States
View all 25 locations
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Active, not recruiting

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

NCT04910776

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations
  • Stanford Hospital- Site Number : 8400006 Stanford, California, United States
  • Children's Hospitals and Clinics of Minnesota- Site Number : 8400008 Minneapolis, Minnesota, United States
  • Advanced Medical Genetics- Site Number : 8400002 Hawthorne, New York, United States
  • Duke University Medical Center- Site Number : 8400004 Durham, North Carolina, United States
View all 16 locations
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