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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.

12,593 public trials 2,156 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

ASIA Down Syndrome Acute Lymphoblastic Leukemia 2016

NCT03286634

Conditions
Abnormalities, Multiple, Acute Lymphoblastic Leukemia, Childhood Cancer, +20 more
Locations
  • Prince of Wales Hospital Shatin, New Territories, Hong Kong
  • Kagoshima University Hospital Kagoshima, Japan
  • University of Malaya Medical Centre Kuala Lumpur, Malaysia
  • Subang Jaya Medical Centre Subang Jaya, Malaysia
View Trial Details
Active, not recruiting

Gout in the ED and Improving Research Participation

NCT04075903

Conditions
Arthritis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Crystal Arthropathies, +9 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • University of Iowa Hospitals and Clinics Iowa City, Iowa, United States
  • Massachusetts General Hospital Boston, Massachusetts, United States
  • MetroHealth Medical Center Cleveland, Ohio, United States
View Trial Details
Active, not recruiting

Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants

NCT06738017

Conditions
Alpha 1-Antitrypsin Deficiency, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, +9 more
Locations
  • University of California, San Diego San Diego, California, United States
  • Saint Louis University St Louis, Missouri, United States
  • Medpace Clinical Pharmacology Unit Cincinnati, Ohio, United States
  • The Medical University of South Carolina Charleston, South Carolina, United States
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Active, not recruiting

Proof-of-concept Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of SION-719 When Added to Trikafta

NCT07108153

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Cystic Fibrosis (CF), +6 more
Locations
  • National Jewish Health Denver, Colorado, United States
  • University of Iowa Iowa City, Iowa, United States
  • Johns Hopkins Baltimore, Maryland, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
View all 13 locations
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Active, not recruiting

To Evaluate the Safety and Efficacy of GS1191-0445 Injection in the Treatment of Severe Hemophilia A

NCT07548411

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations
  • Union Hospital, Tongji Medical College, Huazhong University of Science and Technology Wuhan, Hubei, China
  • Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College Tianjin, Tianjin Municipality, China
View Trial Details
Active, not recruiting

Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa

NCT06079372

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Hypophosphatasia, +4 more
Locations
  • Research Site Hartford, Connecticut, United States
  • Research Site Baltimore, Maryland, United States
  • Research Site Minneapolis, Minnesota, United States
  • Research Site Kansas City, Missouri, United States
View all 21 locations
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