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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

A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

NCT06150820

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • Emory Clinic Atlanta, Georgia, United States
  • University of Kansas Medical Center Kansas City, Kansas, United States
  • University of Michigan Ann Arbor, Michigan, United States
  • Children's Hospitals and Clinics of Minnesota Minneapolis, Minnesota, United States
View all 53 locations
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Active, Not Recruiting

Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)

NCT04127578

Conditions
Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +20 more
Locations
  • Mayo Clinic, 13400 E. Shea Boulevard Scottsdale, Arizona, United States
  • Esperanza Clinical, 25220 Hancock Avenue Murrieta, California, United States
  • Rocky Mountain Clinical Research - CenExel - PPDS, 701 East Hampden Avenue Suite 510 Englewood, Colorado, United States
  • K2 Medical Research, 101 Southhall Lane, Suite 150 Maitland, Florida, United States
View all 15 locations
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Active, Not Recruiting

Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

NCT05222906

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Yale University School of Medicine - Investigational Site Number: 8400003 New Haven, Connecticut, United States
  • University of Iowa - Investigational Site Number: 8400002 Iowa City, Iowa, United States
  • Texas Oncology - Medical City Dallas Site Number : 8400008 Dallas, Texas, United States
  • Lysosomal & Rare Disorders Research & Treatment Center, Inc - Investigational Site Number: 8400001 Fairfax, Virginia, United States
View all 22 locations
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Active, Not Recruiting

Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

NCT06181136

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • University of Iowa Stead Family Children's Hospital Iowa City, Iowa, United States
  • University of North Carolina at Chapel Hill Chapel Hill, North Carolina, United States
  • Baylor College of Medicine and Texas Children's Hospital Houston, Texas, United States
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Active, Not Recruiting

Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

NCT02843035

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations
  • Yale University School of Medicine Site Number : 840002 New Haven, Connecticut, United States
  • Baylor Institute of Metabolic Diseases Site Number : 840001 Dallas, Texas, United States
  • Lysosomal and Rare Disorders Research and Treatment Center, Inc Site Number : 840003 Fairfax, Virginia, United States
  • Investigational Site Number : 276001 Mainz, Germany
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Active, Not Recruiting

Specified Drug-use Survey of Leqvio for s.c. Injection.

NCT06275724

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Familial Hypercholesterolaemia, +12 more
Locations
  • Novartis Investigative Site Handa, Aichi-ken, Japan
  • Novartis Investigative Site Ichinomiya, Aichi-ken, Japan
  • Novartis Investigative Site Ichinomiya, Aichi-ken, Japan
  • Novartis Investigative Site Komaki, Aichi-ken, Japan
View all 107 locations
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Active, Not Recruiting

An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)

NCT06961344

Conditions
Acid-Base Imbalance, Acidosis, Acidosis, Lactic, +23 more
Locations
  • UC San Diego - Altman Clinical and Translational Research Institute La Jolla, California, United States
  • Children's Hospital of Colorado Aurora, Colorado, United States
  • Rare Disease Research Atlanta, Georgia, United States
  • Massachusetts General Hospital Boston, Massachusetts, United States
View all 16 locations
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Active, Not Recruiting

A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease

NCT04020055

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +32 more
Locations
  • Emory University Atlanta, Georgia, United States
  • The Cleveland Clinic Cleveland, Ohio, United States
  • Lysosomal and Rare Disorders Research and Treatment Center, Inc Fairfax, Virginia, United States
  • Royal Melbourne Hospital Parkville, Victoria, Australia
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