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Clinical trial condition

Metabolism, Inborn Errors

Explore clinical trials studying Metabolism, Inborn Errors. Study availability and eligibility vary by location and protocol.

2,496 public trials 457 recruiting studies
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Metabolism, Inborn Errors trial results

Active, Not Recruiting

Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa

NCT06079281

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Hypophosphatasia, +4 more
Locations
  • Research Site Indianapolis, Indiana, United States
  • Research Site Garden City, New York, United States
  • Research Site Durham, North Carolina, United States
  • Research Site Columbus, Ohio, United States
View all 65 locations
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Active, Not Recruiting

A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease

NCT04884815

Conditions
Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations
  • University of California Los Angeles Los Angeles, California, United States
  • Stanford University Redwood City, California, United States
  • University of California Davis Sacramento, California, United States
  • Northwestern University Chicago, Illinois, United States
View all 16 locations
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Active, Not Recruiting

Impact of Transferrin Saturation Guided Maintenance Treatment on Quality of Life in HFE Haemochromatosis

NCT04779593

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Haemochromatosis, +7 more
Locations
  • Hopital Avicenne Bobigny, France
  • CHU Dupuytren Limoges, France
  • GHBS site du Scorff Lorient, France
  • GHRMSA - Hôpital Emile Muller Mulhouse, France
View Trial Details
Active, Not Recruiting

Gene Transfer Study in Patients With Late Onset Pompe Disease

NCT04174105

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • University of California Irvine, Department of Neurology Orange, California, United States
  • Stanford University Palo Alto, California, United States
  • University of Utah, Division of Medical Genetics Salt Lake City, Utah, United States
  • Newcastle Upon Tyne Hospitals Foundation Trust Clinical Research Facility Newcastle upon Tyne, United Kingdom
View Trial Details
Active, Not Recruiting

A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)

NCT04573023

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
Locations
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • University of Minnesota Minneapolis, Minnesota, United States
View all 25 locations
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