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Clinical trial condition

Lipidoses

Explore clinical trials studying Lipidoses. Study availability and eligibility vary by location and protocol.

453 public trials 89 recruiting studies
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Lipidoses trial results

Active, not recruiting

A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)

NCT05487599

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Duke University Health System Durham, North Carolina, United States
  • Lysosomal & Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
  • Westmead Hospital-Cnr Hawkesbury and Darcy Rds Westmead, New South Wales, Australia
  • SphinCS Clinical Science for LSD Höchheim, Germany
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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

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
View Trial Details
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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Active, not recruiting

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

NCT04713475

Conditions
Beta-Galactosidase-1 (GLB1) Deficiency, Brain Diseases, Brain Diseases, Metabolic, +23 more
Locations
  • Benioff Children's Hospital Oakland, California, United States
  • University of Minnesota Minneapolis, Minnesota, United States
  • Children's Hospital at St. Peter's University Hospital New Brunswick, New Jersey, United States
  • The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
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Active, not recruiting

A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease

NCT05280548

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of Alabama -The Kirklin Clinic- Site Number : 8400010 Birmingham, Alabama, United States
  • University of California Los Angeles Medical Center- Site Number : 8400008 Los Angeles, California, United States
  • Emory University School of Medicine - Atlanta- Site Number : 8400009 Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 8400005 Chicago, Illinois, United States
View all 54 locations
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