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Clinical trial condition

Lysosomal Storage Diseases

Explore clinical trials studying Lysosomal Storage Diseases. Study availability and eligibility vary by location and protocol.

710 public trials 130 recruiting studies
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Lysosomal Storage Diseases trial results

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
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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

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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Active, Not Recruiting

A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

NCT04848779

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations
  • Advanced Medical Genetics- Site Number : 8400002 Hawthorne, New York, United States
  • Duke University Medical Center- Site Number : 8400004 Durham, North Carolina, United States
  • Cincinnati Children's Hospital Medical Center- Site Number : 8400001 Cincinnati, Ohio, United States
  • Le Bonheur Children's Hospital- Site Number : 8400005 Memphis, Tennessee, United States
View all 15 locations
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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
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