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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.

81 public trials 12 recruiting studies
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Lysosomal Storage Diseases trial results

Completed

Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency

NCT01488097

Conditions
Cholesterol Ester Storage Disease, Cholesterol Ester Storage Disease (CESD), Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +12 more
Locations
  • Eureka, California, United States
  • Sacramento, California, United States
  • San Francisco, California, United States
  • Minneapolis, Minnesota, United States
View Trial Details
Completed

The Classification and Cause of Leukodystrophies of Unknown Cause

NCT00001671

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Lysosomal Storage Disease, +4 more
Locations
  • University of California, San Francisco San Francisco, California, United States
  • Childrens National Medical Center Washington D.C., District of Columbia, United States
  • Institut National de la Sante' et de la Recherche Medicale Clermont-Ferrand, Cedex, France
  • Tel Aviv University Tel Aviv, Israel
View Trial Details
Completed

A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C

NCT02435030

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University Hospital Copenhagen (Rigshospitalet) Copenhagen, Denmark
  • CHU de Montpellier Montpellier, France
  • Hôpital Trousseau Paris, France
  • Villa Metabolica Mainz Mainz, Germany
View all 16 locations
View Trial Details
Completed

An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype

NCT01528917

Conditions
Cholesterol Ester Storage Disease, Cholesterol Ester Storage Disease(CESD), Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +11 more
Locations
  • Stanford University Stanford, California, United States
  • Children's Memorial Hospital Chicago, Illinois, United States
  • University of Minnesota Minneapolis, Minnesota, United States
  • Morgan Stanley Children's Hospital of New York-Presbyterian New York, United States
View all 16 locations
View Trial Details
Completed

A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I

NCT00852358

Conditions
Carbohydrate Metabolism, Inborn Errors, Cognition Disorders, Cognitive Decline, +17 more
Locations
  • Children's Hospital & Research Center Oakland Oakland, California, United States
  • Los Angeles Biomedical Institute at Harbor-UCLA Torrance, California, United States
  • University of Minnesota Minneapolis, Minnesota, United States
View Trial Details

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