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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 to Evaluate GBT021601 in Single and Multiple Doses in Healthy Participants

NCT05036512

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • ICON Early Phase Services, LLC San Antonio, Texas, United States
  • Harry Perkins Institute of Medical Research Nedlands, Western Australia, Australia
  • Linear Clinical Research Nedlands, Western Australia, Australia
  • Oxford Compounding North Perth, Western Australia, Australia
View Trial Details
Completed

Pharmacokinetics (PK), Safety, Efficacy and Maximal Use PK of ARQ-151 in Adolescents/Children With Mild/Moderate Eczema

NCT04156191

Conditions
Atopic Dermatitis (Eczema), Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +10 more
Locations
  • Arcutis Clinical Site 07 Birmingham, Alabama, United States
  • Arcutis Clinical Site 11 Rancho Santa Margarita, California, United States
  • Arcutis Clinical Site 02 Coral Gables, Florida, United States
  • Arcutis Clinical Site 05 Hialeah, Florida, United States
View Trial Details
Completed

C-Trelin Orally Disintegrated(OD) Tablet 5mg in Ataxia Due to Spinocerebellar Degeneration

NCT04107740

Conditions
Ataxia, Brain Diseases, Central Nervous System Diseases, +13 more
Locations
  • Chonnam National University Hospital Gwangju, South Korea
  • Gangnam Severance Hospital Seoul, South Korea
  • Korea University Anam Hospital Seoul, South Korea
  • Korea University Guro Hospital Seoul, South Korea
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Completed

A Study of the Effect of IW-1701 (Olinciguat), a Stimulator of Soluble Guanylate Cyclase (sGC), on Patients With Sickle Cell Disease (SCD)

NCT03285178

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • Children's Hospital of Orange County Orange, California, United States
  • MedStar Health Research Institute, MedStar Washington Hospital Center Washington D.C., District of Columbia, United States
  • Howard University Center for Sickle Cell Disease Washington D.C., District of Columbia, United States
  • Innovative Medical Research of South Florida, Inc. Aventura, Florida, United States
View all 37 locations
View Trial Details
Completed

Study to Investigate Efficacy, Safety and Pharmacokinetics of BT595 in Subjects With PID

NCT02810444

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Immune System Diseases, +3 more
Locations
  • Investigational site # 0104 Birmingham, Alabama, United States
  • Investigational site # 0116 Los Angeles, California, United States
  • Investigational site # 0103 Centennial, Colorado, United States
  • Investigational site # 0114 Thornton, Colorado, United States
View all 19 locations
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