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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

Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency

NCT02292654

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
Locations
  • Investigational Site Number 840001 New York, United States
  • Investigational Site Number 076001 Porto Alegre, Brazil
  • Investigational Site Number 250002 Bron, France
  • Investigational Site Number 276001 Mainz, Germany
View Trial Details
Completed

A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients

NCT03811028

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • Children´s Hospital and research center Oakland, California, United States
  • University of North Carolina hospitals Chapel Hill, North Carolina, United States
  • Gazi University Hospital Ankara, Turkey (Türkiye)
View Trial Details
Completed

Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease

NCT02574286

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations
  • Cedars Sinai Medical Center Beverly Hills, California, United States
  • Kaiser Permanente Los Angeles, California, United States
  • Emory Genetics Atlanta, Georgia, United States
  • Ann and Robert H Lurie Childrens Hospital of Chicago Chicago, Illinois, United States
View all 15 locations
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Completed

Respiratory Cathepsins, Proteases Inhibitors and Glycosaminoglycans (GAG) in Mucopolysaccharidosis

NCT04112602

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +8 more
Locations
  • Metabolic Disease Competence Centre - Medical Genetics Department - University Hospital, Angers Angers, France
  • Metabolic Disease Reference Centre - Medical Genetics Department - University Hospital, Bordeaux Bordeaux, France
  • Metabolic Disease Competence Centre - Medical Genetics Department - University Hospital, Brest Brest, France
  • Metabolic Disease Competence Centre - Pediatrics Department - University Hospital, Rennes Rennes, France
View Trial Details
Completed

A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients

NCT03423186

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • Childrens's Hospital and Research Center Oakland, California, United States
  • University of North Carolina Hospitals Chapel Hill, North Carolina, United States
  • University Medical Center Hamburg-Eppendorf Hamburg, Germany
  • Gazi University Hospital Ankara, Turkey (Türkiye)
View Trial Details
Completed

Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2

NCT04470713

Conditions
AB Variant Gangliosidosis GM2, Brain Diseases, Brain Diseases, Metabolic, +24 more
Locations
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • Mayo Clinic - Rochester Rochester, Minnesota, United States
  • Lysosomal and Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
  • UCL Cliniques Universitaires Saint-Luc Brussels, Belgium
View all 17 locations
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