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Clinical trial condition

Sphingolipidoses

Explore clinical trials studying Sphingolipidoses. Study availability and eligibility vary by location and protocol.

394 public trials 77 recruiting studies
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Sphingolipidoses 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
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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

Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)

NCT03500094

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Clinical Study Site Tampa, Florida, United States
  • Clinical Study Site Atlanta, Georgia, United States
  • Clinical Study Site Minneapolis, Minnesota, United States
  • Clinical Study Site Columbia, Missouri, United States
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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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