Skip to main content
OpenTrials

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
Refine in advanced search Browse all conditions

Genetic Diseases, Inborn trial results

Completed

Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients

NCT06406153

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • Clinical Research Development Unit of Akbar Hospital, Faculty of Medicine Mashhad, Iran
  • Growth and Development Research Center, Childrens Medical Center Tehran, Iran
  • Loghman Hospital Tehran, Iran
  • Mofid Childrens Hospital Tehran, Iran
View Trial Details
Completed

Instrumented Data Exchange for Ataxia Study

NCT04268147

Conditions
Ataxia, Brain Diseases, Central Nervous System Diseases, +21 more
Locations
  • University of California-Los Angeles Los Angeles, California, United States
  • The University of Chicago Chicago, Illinois, United States
  • Johns Hopkins Medicine Baltimore, Maryland, United States
  • Mass General Hospital Boston, Massachusetts, United States
View Trial Details
Completed

A Study of B244 Delivered as a Topical Spray to Assess Safety in Pediatric Subjects With Atopic Dermatitis

NCT03775434

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +10 more
Locations
  • Dermatology Trial Associates Bryant, Arkansas, United States
  • L.A. Universal Research Center, Inc. Los Angeles, California, United States
  • The Indiana Clinical Trials Center Plainfield, Indiana, United States
  • Minnesota Clinical Study Center Fridley, Minnesota, United States
View Trial Details
Completed

Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency

NCT04442347

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
Locations
  • University of Florida Gainesville, Florida, United States
  • M Health Fairview Masonic Children's Hospital Minneapolis, Minnesota, United States
  • The Mount Sinai Hospital New York, United States
  • Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
View Trial Details
Completed

Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis Subjects Without an F508del Mutation

NCT05274269

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations
  • Medizinische Universität Innsbruck Innsbruck, Austria
  • Cliniques Universitaires de Bruxelles Hopital Erasme Brussels, Belgium
  • Universitair Ziekenhuis Brussel - Campus Jette Brussels, Belgium
  • Universitair Ziekenhuis Antwerpen (UZA) - Antwerp University Hospital Edegem, Belgium
View all 90 locations
View Trial Details

Related conditions