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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

Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I

NCT02371226

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
Locations
  • Children's Hospital Oakland Oakland, California, United States
  • Children's Hospital of Orange County Orange, California, United States
  • Emory Healthcare Decatur, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
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Completed

A Study to Assess the Pharmacokinetics and Safety of Co-administered Oral Galicaftor, Navocaftor, and ABBV-576 in Healthy Adults for the Treatment of Cystic Fibrosis

NCT05530278

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +6 more
Locations
  • Anaheim Clinical Trials LLC /ID# 248824 Anaheim, California, United States
  • Clinical Pharmacology of Miami /ID# 248823 Miami, Florida, United States
  • PPD Clinical Research Unit -Las Vegas /ID# 248853 Las Vegas, Nevada, United States
  • PPD Clinical Research Unit - Austin /ID# 248854 Austin, Texas, United States
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Completed

Ph 3 Efficacy and Safety of B-VEC for the Treatment of DEB

NCT04491604

Conditions
Collagen Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +12 more
Locations
  • Mission Dermatology Center Rancho Santa Margarita, California, United States
  • Stanford University Stanford, California, United States
  • Pediatric Skin Research, LLC Coral Gables, Florida, United States
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