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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.

449 public trials 107 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

Investigating the Long-term Efficacy and Safety of Two Doses of NN-220 (Somatropin) in Short Stature Due to Noonan Syndrome

NCT01927861

Conditions
Cardiovascular Abnormalities, Cardiovascular Diseases, Congenital Abnormalities, +11 more
Locations
  • Novo Nordisk Investigational Site Asahikawa, Hokkaido, Japan
  • Novo Nordisk Investigational Site Fukuoka, Japan
  • Novo Nordisk Investigational Site Fukuoka, Japan
  • Novo Nordisk Investigational Site Iruma-gun, Saitama, Japan
View all 27 locations
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Completed

Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

NCT01826487

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +7 more
Locations
  • University of California, Los Angeles Los Angeles, California, United States
  • UC Davis Medical Center Sacramento, California, United States
  • Children's Hospital Colorado - Center for Cancer and Blood Disorders Aurora, Colorado, United States
  • Child Neurology Center of Northwest Florida Gulf Breeze, Florida, United States
View all 55 locations
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Completed

Efficacy and Safety of Tigerase® vs. Pulmozyme® in Patients With Cystic Fibrosis

NCT04468100

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +8 more
Locations
  • Regional Clinical Hospital Barnaul, Altayskiy Kray, Russia
  • Republican Clinical hospital named after G.G. Kuvatov Ufa, Bashkortostan Republic, Russia
  • Crimean federal university named after V.I.Vernadsky Simferopol, Crimea Republic, Russia
  • Sverdlovsk Regional clinical hospital No. 1 Yekaterinburg, Sverdlovsk Oblast, Russia
View all 14 locations
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Completed

Farber Disease Natural History Study

NCT03233841

Conditions
ASAH1 Mutation, Acid Ceramidase Deficiency, Brain Diseases, +27 more
Locations
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Children's National Health System Washington D.C., District of Columbia, United States
  • Hospital de Niños de la Santisima Trinidad Córdoba, CP, Argentina
  • Montreal Children's Hospital Montreal, Quebec, Canada
View all 15 locations
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Completed

Saline Hypertonic in Preschoolers

NCT02378467

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +7 more
Locations
  • University of Alabama-Birmingham: Pulmonary, Allergy and Critical Care Medicine Birmingham, Alabama, United States
  • Stanford University Palo Alto, California, United States
  • Children's Hospital of Colorado Aurora, Colorado, United States
  • Nemours Children's Hospital Orlando, Florida, United States
View all 25 locations
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Completed

Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

NCT02112994

Conditions
Cholesterol Ester Storage Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +10 more
Locations
  • Chicago, Illinois, United States
  • Shreveport, Louisiana, United States
  • Cincinnati, Ohio, United States
  • Westmead, Australia
View all 19 locations
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