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Clinical trial condition

Sphingolipidoses

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

389 public trials 81 recruiting studies
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Sphingolipidoses trial results

Completed

A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

NCT03771898

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Los Angeles Biomedical Research Institute at Harbor-UCLA Torrance, California, United States
  • Childrens Hospital Colorado Aurora, Colorado, United States
  • Rare Disease Research, LLC Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
View all 30 locations
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Completed

Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3

NCT03485677

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations
  • Investigational Site Number : 0320001 Buenos Aires, Argentina
  • Investigational Site Number : 1240002 Calgary, Alberta, Canada
  • Investigational Site Number : 1240003 Vancouver, British Columbia, Canada
  • Investigational Site Number : 1240001 Toronto, Ontario, Canada
View all 21 locations
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Completed

A Gene Therapy Study in Patients With Gaucher Disease Type 1

NCT05324943

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Kaiser Permanente Los Angeles, California, United States
  • Lysosomal Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
  • Hospital de Clinicas de Porto Alegre (HCPA) Porto Alegre, Brazil
  • SphinCS Höchheim, Germany
View Trial Details
Completed

Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

NCT03614234

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +29 more
Locations
  • UAB Medicine Birmingham, Alabama, United States
  • Emory University School of Medicine Atlanta, Georgia, United States
  • University of Iowa Hospitals and Clinica Iowa City, Iowa, United States
  • Infusion Associates Grand Rapids, Michigan, United States
View all 14 locations
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