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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

Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

NCT05164055

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations
  • Investigational Site Number : 2500004 Bordeaux, France
  • Investigational Site Number : 2500005 Brest, France
  • Investigational Site Number : 2500008 Clermont-Ferrand, France
  • Investigational Site Number : 2500009 Lille, France
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Active, Not Recruiting

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

NCT03911505

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • University of Florida Clinical Research Center Gainesville, Florida, United States
  • Wolfson Children's Hospital Jacksonville, Florida, United States
  • Woodruff Memorial Research Building Atlanta, Georgia, United States
  • St. Louis Children's Hospital St Louis, Missouri, United States
View all 17 locations
View Trial Details
Active, Not Recruiting

A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

NCT04251026

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +22 more
Locations
  • UCSF Benioff Children's Hospital Oakland, California, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • UNC Children's Research Institute Chapel Hill, North Carolina, United States
  • UPMC | Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
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