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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.

12,530 public trials 2,140 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

Uganda Sickle Surveillance Study (US-3)

NCT06301893

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • Makerere University Kampala, Uganda
  • Minister of Health Kampala, Uganda
  • National Coordinator EID Program Kampala, Uganda
  • Sickle Cell Clinic Department of Pediatrics & Child Health Mulago Hospital Kampala, Uganda
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Recruiting

Phase 3 Extension Study of ADX-324 in Participants With Hereditary Angioedema (HAE)

NCT07428499

Conditions
Angioedema, Angioedemas, Hereditary, Cardiovascular Diseases, +17 more
Locations
  • ADARx Clinical Site Litchfield Park, Arizona, United States
  • ADARx Clinical Site Little Rock, Arkansas, United States
  • ADARx Clinical Site San Diego, California, United States
  • ADARx Clinical Site Walnut Creek, California, United States
View all 48 locations
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Recruiting

A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725

NCT06539507

Conditions
Abnormalities, Multiple, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +10 more
Locations
  • Stanford University School of Medicine Palo Alto, California, United States
  • Therapeutics Clinical Research San Diego, California, United States
  • Yale Center for Clinical Investigation New Haven, Connecticut, United States
  • Northwestern Dermatology CTU Chicago, Illinois, United States
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Recruiting

STOP-HAE: A Phase 3 Study of ADX-324 in HAE

NCT06960213

Conditions
Angioedema, Angioedemas, Hereditary, Cardiovascular Diseases, +17 more
Locations
  • ADARx Clinical Site Litchfield Park, Arizona, United States
  • ADARx Clinical Site Little Rock, Arkansas, United States
  • ADARx Clinical Site San Diego, California, United States
  • ADARx Clinical Site Walnut Creek, California, United States
View all 51 locations
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Recruiting

A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia

NCT06953583

Conditions
Brain Diseases, Central Nervous System Diseases, Cerebellar Diseases, +11 more
Locations
  • UCLA Neurology Outpatient Clinic at Westwood Los Angeles, California, United States
  • Norman Fixel Institute for Neurological Diseases UF Health Gainesville, Florida, United States
  • USF Health Morsani College of Medicine Department of Neurology Tampa, Florida, United States
  • Children's Hospital of Philadelphia - Buerger Center for Advanced Pediatric Care - PIN Philadelphia, Pennsylvania, United States
View all 34 locations
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Recruiting

The HOPE Biobank Resource (BMT CTN 2402 HOPE)

NCT07227155

Conditions
Anemia, Anemia, Aplastic, Aplastic Anemias, +8 more
Locations
  • University of Florida Gainesville, Florida, United States
  • Oregon Health and Science University Portland, Oregon, United States
  • Vanderbilt University Medical Center Nashville, Tennessee, United States
  • Baylor College of Medicine Houston, Texas, United States
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