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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.

77 public trials 13 recruiting studies
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Lysosomal Storage Diseases trial results

Completed

Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency

NCT01757184

Conditions
Cholesterol Ester Storage Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +10 more
Locations
  • Tucson, Arizona, United States
  • Palo Alto, California, United States
  • San Francisco, California, United States
  • Wilmington, Delaware, United States
View all 40 locations
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Completed

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

NCT02432144

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
Locations
  • Children's Hospital Oakland Oakland, California, United States
  • Children's Hospital of Orange County Orange, California, United States
  • University of Minnesota Minneapolis, Minnesota, United States
  • Baylor College of Medicine Houston, Texas, United States
View Trial Details
Completed

A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

NCT02230566

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +17 more
Locations
  • Children's Hospital Oakland Oakland, California, United States
  • Children's Hospital of Orange County Orange, California, United States
  • Miami Children's Hospital Miami, Florida, United States
  • University of Minnesota Minneapolis, Minnesota, United States
View Trial Details
Completed

Farber Disease Natural History Study

NCT03233841

Conditions
ASAH1 Mutation, Acid Ceramidase Deficiency, Brain Diseases, +27 more
Locations
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Children's National Health System Washington D.C., District of Columbia, United States
  • Hospital de Niños de la Santisima Trinidad Córdoba, CP, Argentina
  • Montreal Children's Hospital Montreal, Quebec, Canada
View all 15 locations
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Completed

Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

NCT02112994

Conditions
Cholesterol Ester Storage Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +10 more
Locations
  • Chicago, Illinois, United States
  • Shreveport, Louisiana, United States
  • Cincinnati, Ohio, United States
  • Westmead, Australia
View all 19 locations
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Completed

Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age

NCT02418455

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
Locations
  • Children's National Health System Washington D.C., District of Columbia, United States
  • New York University Langone Medical Center New York, United States
  • University of Utah Hospital Salt Lake City, Utah, United States
  • Centro Hospitalar do Porto Porto, Portugal
View Trial Details
Completed

Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency

NCT01307098

Conditions
Cholesterol Ester Storage Disease, Cholesterol Ester Storage Disease(CESD), Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +12 more
Locations
  • Stanford, California, United States
  • New York, United States
  • Pittsburgh, Pennsylvania, United States
  • Prague, Czechia
View Trial Details

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