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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.

12,656 public trials 2,147 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia)

NCT02624869

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Familial Hypercholesterolemia, +11 more
Locations
  • Research Site The Bronx, New York, United States
  • Research Site Cincinnati, Ohio, United States
  • Research Site Nashville, Tennessee, United States
  • Research Site Camperdown, New South Wales, Australia
View all 46 locations
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Completed

Trial Assessing Long Term USe of PCSK9 Inhibition in Subjects With Genetic LDL Disorders

NCT01624142

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Genetic Diseases, Inborn, +11 more
Locations
  • Research Site Los Angeles, California, United States
  • Research Site New York, United States
  • Research Site New York, United States
  • Research Site New York, United States
View all 44 locations
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Completed

A Study to Evaluate Ketamine for the Treatment of Rett Syndrome

NCT03633058

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +7 more
Locations
  • University of Alabama Birmingham School of Medicine Birmingham, Alabama, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Rush University Medical Center Chicago, Illinois, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
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Completed

A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

NCT03852498

Conditions
Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +22 more
Locations
  • Lucile Packard Children's Hospital Palo Alto, California, United States
  • Boston Children's Hospital/Massachusetts General Hospital Boston, Massachusetts, United States
  • University of Minnesota Minneapolis, Minnesota, United States
  • Hôpital Robert Debré Paris, France
View Trial Details

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