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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.

12,537 public trials 2,139 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

Descemet Endothelial Thickness Comparison Trial II

NCT05275972

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Corneal Diseases, Corneal Dystrophies, Hereditary, +6 more
Locations
  • Stanford University Palo Alto, California, United States
  • University of California Davis Sacramento, California, United States
  • University of Miami Palm Beach Gardens, Florida, United States
  • Dartmouth-Hitchcock Medical Center Lebanon, New Hampshire, United States
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Recruiting

A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment

NCT06247085

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
Locations
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • Yale University School of Medicine New Haven, Connecticut, United States
  • Emory University Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
View all 52 locations
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Recruiting

National Network for Cardiovascular Genomics: Advancing Cardiovascular Healthcare for Hereditary Diseases in Brazil's Unified Health System Through a Multicenter Registry

NCT06546137

Conditions
Abnormalities, Multiple, Aneurysm, Aortic Aneurysm, +70 more
Locations
  • Centro de Pesquisa Silvestre Santé Rio Branco, Acre, Brazil
  • Centro de Pesquisas Clínicas Dr. Marco Mota Maceió, Alagoas, Brazil
  • Hospital Universitário da Unifap Macapá, Amapá, Brazil
  • Hospital de Messejana Dr. Carlos Alberto Studart Gomes Fortaleza, Ceará, Brazil
View all 27 locations
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Recruiting

Safety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic Fibrosis

NCT06747858

Conditions
CFTR Gene Mutation, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, +6 more
Locations
  • University of Arizona Tucson, Arizona, United States
  • University of Arkansas for Medical Sciences Little Rock, Arkansas, United States
  • Children's Hospital of Los Angeles Los Angeles, California, United States
  • Children's National Hospital Washington D.C., District of Columbia, United States
View all 13 locations
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Recruiting

A Study Assessing KB407 for the Treatment of Cystic Fibrosis

NCT05504837

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations
  • University of Florida, Gainesville Gainesville, Florida, United States
  • St. Lukes CF Center of Idaho Boise, Idaho, United States
  • The Cystic Fibrosis Institute Northfield, Illinois, United States
  • New York Medical College/ Boston Children's Health Physicians Hawthorne, New York, United States
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