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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

Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)

NCT01510028

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • The Children's Hospital at Westmead Westmead, Australia
  • Rigshospitalet Copenhagen, Denmark
  • Hopital de Bicetre Le Kremlin-Bicêtre, Île-de-France Region, France
  • Center for Pediatric Clinical Studies Tübingen, Baden-Wurttemberg, Germany
View Trial Details
Completed

Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease

NCT01842841

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations
  • Hamamatsu University School of Medicine Hamamatsu, Shizuoka, Japan
  • Iwata City Hospital Ōkubo, Shizuoka, Japan
  • The Jikei University School of Medicine Minato-ku, Tokyo, Japan
  • Chiba Children's Hospital Chiba, Japan
View Trial Details
Completed

Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease

NCT01685216

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Alexandria University Hospital Alexandria, Egypt
  • Abu El Rich Hospital, Cairo University Hospital Cairo, Egypt
  • Children's Hospital, Ain Shams University Hospital Cairo, Egypt
  • KEM Hospital Research Centre Pune, Maharashtra, India
View Trial Details
Completed

An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease

NCT00635427

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Los Angeles Medical Center Los Angeles, California, United States
  • Children's Hospital Oakland Oakland, California, United States
  • Emory University School of Medicine Atlanta, Georgia, United States
  • Children's Memorial Hospital Chicago, Illinois, United States
View all 21 locations
View Trial Details
Completed

Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase

NCT00478647

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations
  • Regional Metabolic Center Los Angeles, California, United States
  • Children's Hospital Oakland Oakland, California, United States
  • Emory University Decatur, Georgia, United States
  • Feinberg School of Medicine Chicago, Illinois, United States
View all 15 locations
View Trial Details
Completed

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

NCT00069641

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +22 more
Locations
  • Children's Hospital Oakland Oakland, California, United States
  • St. Louis Children's Hospital, Washington University St Louis, Missouri, United States
  • University of North Carolina at Chapel Hill Chapel Hill, North Carolina, United States
  • Texas Children's Hospital, Baylor College of Medicine Houston, Texas, United States
View Trial Details
Completed

Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome

NCT01449240

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +17 more
Locations
  • Emory University Decatur, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • Children's Hospitals and Clinics of Minnesota Minneapolis, Minnesota, United States
  • University of North Carolina, Division of Genetics and Metabolism Chapel Hill, North Carolina, United States
View Trial Details
Completed

Safety Study of Replagal® Therapy in Children With Fabry Disease

NCT01363492

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Emory Division of Medical Genetics Decatur, Georgia, United States
  • Duke University Medical Center Durham, North Carolina, United States
  • Baylor University Medical Center Dallas, Texas, United States
  • University of Utah Hospital Salt Lake City, Utah, United States
View Trial Details

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