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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.

12,656 public trials 2,147 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in Treatment of Adults With Pachyonychia Congenita

NCT03920228

Conditions
Abnormalities, Multiple, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +8 more
Locations
  • Arizona Research Center Phoenix, Arizona, United States
  • Stanford University Palo Alto, California, United States
  • Yale University New Haven, Connecticut, United States
  • International Dermatology Research Miami, Florida, United States
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Completed

FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE)

NCT02922751

Conditions
Abnormalities, Multiple, Alagille Syndrome, Alpha1 Anti-Trypsin Deficiency, +26 more
Locations
  • Children's Hospital Los Angeles Los Angeles, California, United States
  • University of California at San Francisco (UCSF) San Francisco, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Children's Healthcare of Atlanta (Emory University) Atlanta, Georgia, United States
View all 13 locations
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Completed

To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)

NCT01257737

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +13 more
Locations
  • UCLA Pediatrics/Genetics Los Angeles, California, United States
  • Stanford University School of Medicine Palo Alto, California, United States
  • Denver Children's Hospital Aurora, Colorado, United States
  • Children's National Medical Center Washington D.C., District of Columbia, United States
View all 16 locations
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