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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.

12,603 public trials 2,158 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

Antiviral Cellular Therapy for Enhancing T-cell Reconstitution Before or After Hematopoietic Stem Cell Transplantation

NCT03475212

Conditions
Adenoviridae Infections, Adenovirus Infection, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +12 more
Locations
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • City of Hope Duarte, California, United States
  • University of California, Los Angeles Los Angeles, California, United States
  • Children's Hospital Los Angeles Los Angeles, California, United States
View all 30 locations
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Active, not recruiting

PEP and O-PEP Device for Nebulization in Cystic Fibrosis

NCT06964074

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations
  • National Tuberculosis and Lung Diseases Research Institute Rabka-Zdrój, Malopolska, Poland
  • National Institute Tuberculosis and Lung Diesases Rabka-Zdrój, Małopolska, Poland
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Active, not recruiting

Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:

NCT02616484

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
Locations
  • Children's Hospital of Orange County Orange, California, United States
  • Stanford University Stanford, California, United States
  • Children's National Medical Center Washington D.C., District of Columbia, United States
  • University of Florida Gainesville, Florida, United States
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Active, not recruiting

Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)

NCT04239625

Conditions
Autosomal Recessive Stargardt Disease 1 (ABCA4-related), Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, +8 more
Locations
  • Alkeus Site Phoenix, Arizona, United States
  • Alkeus Site Los Angeles, California, United States
  • Alkeus Site Aurora, Colorado, United States
  • Alkeus Site Gainesville, Florida, United States
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Active, not recruiting

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

NCT04042025

Conditions
Central Nervous System Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +12 more
Locations
  • Stanford University Medical Center Palo Alto, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Ann Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • John Hopkins Hospital - David M. Rubenstein Child Health Building Baltimore, Maryland, United States
View all 31 locations
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Active, not recruiting

A Trial of the Safety and Efficacy of Single-Dose Administration of ANB-010 in Subjects With Hemophilia A

NCT06185335

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +6 more
Locations
  • State Autonomous Institution for Healthcare "Chelyabinsk Regional Clinical Hospital" Chelyabinsk, Russia
  • State budgetary healthcare institution Leningrad Regional Clinical Hospital Gatchina, Russia
  • Kuzbass Clinical Hospital named after S.V. Belyaev Kemerovo, Russia
  • Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology and Blood Transfusions of Federal Medical Biological agency" Kirov, Russia
View all 17 locations
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