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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

A Study Evaluating the Long-Term Safety of Elexacaftor Combination Therapy

NCT04362761

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations
  • The Prince Charles Hospital Chermside, Australia
  • Institute for Respiratory Health Nedlands, Australia
  • Telethon Kids Institute, Perth Children's Hospital Nedlands, Australia
  • The Royal Children's Hospital Parkville, VIC, Australia
View all 29 locations
View Trial Details
Completed

Lamazym Aftercare Study

NCT01908725

Conditions
Alpha-Mannosidosis, Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +7 more
Locations
  • Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9 Copenhagen, Denmark
View Trial Details
Completed

A Study of AEB1102 (Pegzilarginase) in Patients With Arginase I Deficiency

NCT03378531

Conditions
Amino Acid Metabolism, Inborn Errors, Arginase I Deficiency, Brain Diseases, +11 more
Locations
  • Stanford University School of Medicine Stanford, California, United States
  • University of Florida Gainesville, Florida, United States
  • Icahn School of Medicine at Mount Sinai New York, United States
  • UTSW Dallas, Texas, United States
View Trial Details
Completed

Study to Gain More Information on How Safe and Effective Jivi Works in Patients With Severe Hemophilia A (Post-marketing Investigation)

NCT04085458

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +6 more
Locations
  • UMHAT Tsaritsa Joanna-ISUL EAD Sofia Sofia, Bulgaria
  • MHAT Sveta Marina EAD Varna, Bulgaria
  • Aarhus Universitetshospital, Skejby Arhus N, Denmark
  • LAIKO General Hospital of Athens Athens, Greece
View all 13 locations
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Completed

Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome

NCT02311673

Conditions
Abnormalities, Multiple, Body Weight, Chromosome Disorders, +18 more
Locations
  • University of California Irvine Irvine, California, United States
  • University of Florida Gainesville, Florida, United States
  • Kansas University Medical Center Kansas City, Kansas, United States
  • Winthrop University Hospital Mineola, New York, United States
View Trial Details

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