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Clinical trial condition

Sphingolipidoses

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

390 public trials 81 recruiting studies
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Sphingolipidoses trial results

Completed

Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks

NCT03180840

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • UAB Medicine Birmingham, Alabama, United States
  • Emory University School of Medicine Atlanta, Georgia, United States
  • University of Iowa Hospitals and Clinics Iowa City, Iowa, United States
  • Infusion Associates Grand Rapids, Michigan, United States
View all 14 locations
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Completed

Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients

NCT01678898

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • UC Davis Medical Center, MIND Institute Department of Pediatrics, Section of Genetics Sacramento, California, United States
  • Department of Human Genetics, Emory University School of Medicine Atlanta, Georgia, United States
  • University of Iowa Health Clinics Iowa City, Iowa, United States
  • University of Kansas Medical Center Kansas City, Kansas, United States
View all 14 locations
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Completed

Safety and Efficacy of PRX-102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)

NCT03018730

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Royal Melbourne Hospital Parkville, Victoria, Australia
  • Capital Health Halifax, Nova Scotia, Canada
  • Vseobecna fakultni nemocnice v Praze Prague, Czechia
  • Academisch Medisch Centrum Amsterdam, Netherlands
View Trial Details
Completed

Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function

NCT02795676

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +29 more
Locations
  • UAB Medicine Birmingham, Alabama, United States
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • University of California Irvine Center Orange, California, United States
  • University of California San Diego San Diego, California, United States
View all 29 locations
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Completed

Extension Study of PRX-102 for up to 60 Months

NCT01981720

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of Florida Gainesville, Florida, United States
  • University of Iowa Hospitals and Clinics Iowa City, Iowa, United States
  • Johns Hopkins University School of Medicine Baltimore, Maryland, United States
  • Institute of Metabolic Disease Dallas, Texas, United States
View Trial Details
Completed

A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)

NCT04106544

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
Locations
  • Investigational Site Number :8400002 Atlanta, Georgia, United States
  • Investigational Site Number :8400003 The Bronx, New York, United States
  • Investigational Site Number :8400001 Valhalla, New York, United States
  • Investigational Site Number :0320002 CABA, Argentina
View all 28 locations
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