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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

A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

NCT05206773

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011 Birmingham, Alabama, United States
  • UCLA Medical Center_Investigational Site Number: 8400006 Los Angeles, California, United States
  • University of California Irvine Medical Center- Site Number : 8400019 Orange, California, United States
  • Advent Health Orlando_Investigational Site Number: 8400008 Orlando, Florida, United States
View all 58 locations
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Active, Not Recruiting

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

NCT06149403

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • University of Minnesota, Pediatrics Minneapolis, Minnesota, United States
  • Ospedale San Raffaele Milan, Italy
  • Princess Maxima Center Utrecht, Netherlands
  • UMC Utrecht Utrecht, Netherlands
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Active, Not Recruiting

Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment

NCT06031259

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +17 more
Locations
  • M.A.G.I.C. Clinic Calgary, Alberta, Canada
  • University of British Columbia Vancouver, British Columbia, Canada
  • Hospital for Sick Children Toronto, Ontario, Canada
  • Hopital Femme Mere Enfant Bron, Auvergne-Rhône-Alpes, France
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Active, Not Recruiting

Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

NCT06545136

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Lysosomal Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
  • Hospital de Clinicas de Porto Alegre (HCPA) Porto Alegre, Brazil
  • Hospital Quironsalud Zaragoza Zaragoza, Spain
  • Salford Royal Hospital Salford, United Kingdom
View Trial Details

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