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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

Completed

Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency

NCT01757184

Conditions
Cholesterol Ester Storage Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +10 more
Locations
  • Tucson, Arizona, United States
  • Palo Alto, California, United States
  • San Francisco, California, United States
  • Wilmington, Delaware, United States
View all 40 locations
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Completed

An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia

NCT03510715

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Genetic Diseases, Inborn, +10 more
Locations
  • Investigational Site Number 0760001 São Paulo, Brazil
  • Investigational Site Number 1240001 Québec, Canada
  • Investigational Site Number 2080001 Viborg, Denmark
  • Investigational Site Number 4840006 Oaxaca City, Mexico
View Trial Details
Completed

Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease

NCT02194985

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Clinical Study Site Atlanta, Georgia, United States
  • Clinical Study Site Kansas City, Kansas, United States
  • Clinical Study Site Grand Rapids, Michigan, United States
  • Clinical Study Site New York, United States
View all 28 locations
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Completed

The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment

NCT02763215

Conditions
Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations
  • Clinical Trial Site New Haven, Connecticut, United States
  • Clinical Trial Site Chicago, Illinois, United States
  • Clinical Trial Site Ann Arbor, Michigan, United States
  • Clinical Trial Site Nashville, Tennessee, United States
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