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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.

12,584 public trials 2,158 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

NCT05329649

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +10 more
Locations
  • Levine Children's Hospital - Hematology Charlotte, North Carolina, United States
  • The Children's Hospital of Philadelphia - Hematology Philadelphia, Pennsylvania, United States
  • St. Jude Children's Research Hospital Memphis, Tennessee, United States
  • TriStar Medical Group Children's Specialists - Pediatric Oncology Nashville, Tennessee, United States
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Active, not recruiting

Early Check: Expanded Screening in Newborns

NCT03655223

Conditions
17 Alpha-Hydroxylase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, +456 more
Locations
  • RTI International Research Triangle Park, North Carolina, United States
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Active, not recruiting

A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body

NCT06421636

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +8 more
Locations
  • Weill Cornell Medicine New York, United States
  • K Eristavi National Center of Experimental and Clinical Surgery Tbilisi, Georgia
  • Medinvest Institute of Hematology and Transfusiology Limited Tbilisi, Georgia
  • Children's Hospital Agia Sophia Athens, Attica, Greece
View all 26 locations
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Active, not recruiting

A Study of KT-621 Administered Orally to Participants With Moderate to Severe Atopic Dermatitis

NCT07217015

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +9 more
Locations
  • Kymera Investigative Site Birmingham, Alabama, United States
  • Kymera Investigative Site North Little Rock, Arkansas, United States
  • Kymera Investigative Site Freemont, California, United States
  • Kymera Investigative Site Los Angeles, California, United States
View all 68 locations
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Active, not recruiting

Cerliponase Alfa Observational Study in the US

NCT04476862

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +10 more
Locations
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • Children's Hospital of Orange County Orange, California, United States
  • Children's Hospital of Colorado Aurora, Colorado, United States
  • Children's National Hospital Washington D.C., District of Columbia, United States
View all 16 locations
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