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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

Completed

Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease

NCT00113035

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • Tower Hematology/Oncology Medical Group Beverly Hills, California, United States
  • Children's National Medical Center Washington D.C., District of Columbia, United States
  • Washington University Medical Center St Louis, Missouri, United States
  • Mount Sinai School of Medicine New York, United States
View Trial Details
Completed

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

NCT00158600

Conditions
Acid Maltase Deficiency Disease, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations
  • Tower Hematology Oncology Medical Group Beverly Hills, California, United States
  • Children's National Medical Center Washington D.C., District of Columbia, United States
  • Washington University Medical Center St Louis, Missouri, United States
  • Mount Sinai School of Medicine New York, United States
View Trial Details
Completed

Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease

NCT00364858

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Children's National Medical Center Washington D.C., District of Columbia, United States
  • University Research Foundation for Lysosomal Storage Disease, Inc. Coral Springs, Florida, United States
  • Emory University Atlanta, Georgia, United States
  • Children's Memorial Hospital Chicago, Illinois, United States
View all 26 locations
View Trial Details
Completed

A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease

NCT00144781

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +12 more
Locations
  • Hospital Infantil Joana de Gusmao Florianópolis, Santa Catarina, Brazil
  • Universidade Federal de Minas Gerais Belo Horizonte, Brazil
  • Hospital de Clinical de Porto Alegre Porto Alegre, Brazil
  • Universidade Federal de Sao Paulo São Paulo, Brazil
View Trial Details
Completed

A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old

NCT00146757

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +12 more
Locations
  • Hôpital E. Herriot Lyon, France
  • Johannes Gutenberg Universität Kinderklinik, Mainz, Germany
  • Sophia Children's Hospital Rotterdam, Netherlands
  • Willink Biochemical Genetics Unit Royal Hospital for Children Manchester, United Kingdom
View Trial Details
Completed

A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

NCT00196716

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • II. interní klinika 1. LF UK Prague, Czechia
  • Tartu University Clinics, Department of Internal Medicine Tartu, Estonia
  • Klinika Chorob Metabolicznych, Instytut "Pomnik-Centrum Zdrowia Dziecka" Warsaw, Poland
  • Detská fakultná nemocnica Kramáre I. Interná klinika Bratislava, Slovakia
View Trial Details
Completed

Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

NCT00146770

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +12 more
Locations
  • University of South Alabama Mobile, Alabama, United States
  • Emory University School of Medicine Atlanta, Georgia, United States
  • University of Rochester Rochester, New York, United States
  • University of North Carolina at Chapel Hill Chapel Hill, North Carolina, United States
View all 25 locations
View Trial Details
Completed

A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

NCT00081497

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • Cedars-Sinai Medical Center Los Angeles, California, United States
  • University of San Francisco San Francisco, California, United States
  • University of Connecticut Health Partners West Hartford, Connecticut, United States
View all 25 locations
View Trial Details
Completed

A Study of Fabrazyme in Pediatric Patients With Fabry Disease

NCT00074958

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of Arizona Tucson, Arizona, United States
  • Hopital Edouard Herriot Lyon, France
  • Hopital de la Timone Enfants Marseille, France
  • Hopital Europeen Georges Pompidou Paris, France
View Trial Details

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