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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

Recruiting

A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)

NCT06192576

Conditions
Acid Sphingomyelinase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +19 more
Locations
  • Ann and Robert H Lurie Children's Hospital of Chicago- Site Number: 001002 Chicago, Illinois, United States
  • Children's Hospital Medical Center- Site Number: 001003 Cincinnati, Ohio, United States
  • Nationwide Children's Hospital - PIN- Site Number : 1001-1 Columbus, Ohio, United States
  • Pulse InfoFrame US Inc.- Site Number: 001001 Philadelphia, Pennsylvania, United States
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Recruiting

The Myelin Disorders Biorepository Project

NCT03047369

Conditions
4H Syndrome, ADLD, AGS, +173 more
Locations
  • Children's Hospital of Los Angeles Los Angeles, California, United States
  • Children's Hospital of Orange County Orange, California, United States
  • Stanford University (Lucile Packard Children's Hospital) Palo Alto, California, United States
  • University of California, Davis (UC Davis Health) Sacramento, California, United States
View all 23 locations
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Recruiting

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

NCT06270316

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • The Kirklin Clinic Of university of Alabama Birmingham Hospital Birmingham, Alabama, United States
  • Emory University School of Medicine Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • MHealth Fairview University of Minnesota Medical Center East Bank Minneapolis, Minnesota, United States
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