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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.

12,522 public trials 2,142 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease

NCT02675959

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • University of California Los Angeles Los Angeles, California, United States
  • University of Florida Gainesville, Florida, United States
  • New York Medical College Valhalla, New York, United States
  • Medical College of Wisconsin Milwaukee, Wisconsin, United States
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Recruiting

Phase 1 Study to Evaluate the Safety and Tolerability of Intravenously Administered PYC-003

NCT06714006

Conditions
Abnormalities, Multiple, Autosomal Dominant Polycystic Kidney Disease (ADPKD), Ciliopathies, +12 more
Locations
  • South Coast Renal, Brockway House, Level 1, Suite 8, 82-86 Queen Street, Southport, Gold Coast, Australia
  • Concord Repatriation General Hospital Concord, New South Wales, Australia
  • Liverpool Hospital, Clinic G-Reception 133, Level 1, Clinical Building, Burnside Drive Liverpool, New South Wales, Australia
  • Scientia Clinical Research Randwick, New South Wales, Australia
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Recruiting

Cooperative Assessment of Late Effects for SCD Curative Therapies

NCT05153967

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +20 more
Locations
  • Children's National Medical Center Washington D.C., District of Columbia, United States
  • Emory University School of Medicine Atlanta, Georgia, United States
  • Johns Hopkins Hospital Baltimore, Maryland, United States
  • National Institutes of Health Clinical Center Bethesda, Maryland, United States
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Recruiting

Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders

NCT06081348

Conditions
22Q11 Deletion, 22q11 Deletion Syndrome, ADHD, +59 more
Locations
  • Alberta Children's Hospital - University of Calgary Calgary, Alberta, Canada
  • University of Alberta-Glenrose Edmonton, Alberta, Canada
  • Dalhousie University - IWK Health Centre Halifax, Nova Scotia, Canada
  • McMaster University Hamilton, Ontario, Canada
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Recruiting

A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD

NCT07038200

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, FSH Muscular Dystrophy, FSHD, +25 more
Locations
  • University of California Irvine Orange, California, United States
  • Stanford University Palo Alto, California, United States
  • University of Colorado Denver, Colorado, United States
  • University of Florida Gainesville, Florida, United States
View all 46 locations
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Recruiting

A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)

NCT07157254

Conditions
Abnormalities, Multiple, Angelman Syndrome, Central Nervous System Diseases, +7 more
Locations
  • Cedars Sinai Medical Center Los Angeles, California, United States
  • Rush University Medical Center Chicago, Illinois, United States
  • Clinical Trial Site Baltimore, Maryland, United States
  • Clinical Trial Site Kansas City, Missouri, United States
View all 21 locations
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