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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

Active, Not Recruiting

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

NCT04713475

Conditions
Beta-Galactosidase-1 (GLB1) Deficiency, Brain Diseases, Brain Diseases, Metabolic, +23 more
Locations
  • Benioff Children's Hospital Oakland, California, United States
  • University of Minnesota Minneapolis, Minnesota, United States
  • Children's Hospital at St. Peter's University Hospital New Brunswick, New Jersey, United States
  • The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
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Active, Not Recruiting

A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease

NCT05280548

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of Alabama -The Kirklin Clinic- Site Number : 8400010 Birmingham, Alabama, United States
  • University of California Los Angeles Medical Center- Site Number : 8400008 Los Angeles, California, United States
  • Emory University School of Medicine - Atlanta- Site Number : 8400009 Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 8400005 Chicago, Illinois, United States
View all 54 locations
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Active, Not Recruiting

Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan

NCT06052800

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Investigational Site Number : 1580004 Taichung, Taiwan
  • Investigational Site Number : 1580005 Tainan, Taiwan
  • Investigational Site Number : 1580001 Taipei, Taiwan
  • Investigational Site Number : 1580003 Taipei, Taiwan
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Active, Not Recruiting

German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa

NCT06095713

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Fabry disease center Berlin - Charité - Universitätsmedizin Berlin Berlin, Germany
  • Fabry disease center Cologne, Universitätsklinikum Köln Cologne, Germany
  • Fabry disease center Hamburg, Universitätsklinikum Hamburg Hamburg, Germany
  • Fabry disease center Hannover, Universitätsklinikum Hannover Hanover, Germany
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Active, Not Recruiting

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

NCT04411654

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • UCSF Benioff Children's Hospital, 747 52nd St Oakland, California, United States
  • University of Minnesota Masonic Children's Hospital, 2450 Riverside Avenue Minneapolis, Minnesota, United States
  • Children's Hospital of Pittsburgh, 4401 Penn Avenue Pittsburgh, Pennsylvania, United States
  • Lysosomal & Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
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Active, Not Recruiting

A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

NCT05206773

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011 Birmingham, Alabama, United States
  • UCLA Medical Center_Investigational Site Number: 8400006 Los Angeles, California, United States
  • University of California Irvine Medical Center- Site Number : 8400019 Orange, California, United States
  • Advent Health Orlando_Investigational Site Number: 8400008 Orlando, Florida, United States
View all 58 locations
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