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Clinical trial condition

Lipid Metabolism, Inborn Errors

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

833 public trials 155 recruiting studies
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Lipid Metabolism, Inborn Errors trial results

Active, Not Recruiting

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07082543

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations
  • UCSF Children's Hospital and Research Center at Oakland Oakland, California, United States
  • University of Minnesota Medical School Minneapolis, Minnesota, United States
  • Mayo Clinic Children's Center - PIN Rochester, Minnesota, United States
  • Children's Medical Center Dallas Dallas, Texas, United States
View all 26 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

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

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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Active, Not Recruiting

A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease

NCT03737214

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of Alabama at Birmingham - Nephrology Research Clinic Birmingham, Alabama, United States
  • University of California Irvine Irvine, California, United States
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • University of Florida Clinical and Translational Science Institute, UF Clinical Research Center Gainesville, Florida, United States
View all 42 locations
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Active, Not Recruiting

Early Check: Expanded Screening in Newborns

NCT03655223

Conditions
17 Alpha-Hydroxylase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, +456 more
Locations
  • RTI International Research Triangle Park, North Carolina, United States
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Active, Not Recruiting

Cerliponase Alfa Observational Study in the US

NCT04476862

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +10 more
Locations
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • Children's Hospital of Orange County Orange, California, United States
  • Children's Hospital of Colorado Aurora, Colorado, United States
  • Children's National Hospital Washington D.C., District of Columbia, United States
View all 16 locations
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