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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

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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Completed

Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

NCT05758922

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +22 more
Locations
  • Hospital Pequeno Principe Curitiba, Brazil
  • Hospital de Clinicas de Porto Alegre Porto Alegre, Brazil
  • Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira Rio de Janeiro, Brazil
View Trial Details
Completed

Prevalence of Fabry's Disease in a Population of Patients With Chronic Pain

NCT02450604

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +25 more
Locations
  • Cs douleur chronique GHSR, CHU Sud Réunion Saint-Pierre, La Réunion, France
  • CeRCa, Unité de Neuromyologie, CHU de Fort de France FORT de France, Martinique, France
  • Centre Douleurs Chroniques, CH de la Côte Basque Bayonne, France
  • Centre Douleurs Chroniques, CHU de Bordeaux Bordeaux, France
View all 21 locations
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Completed

An Efficacy and Safety Study of HGT-1110 in Participants With Metachromatic Leukodystrophy

NCT01887938

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • The Children's Hospital at Westmead Westmead, Australia
  • Hospital de Clinicas de Porto Alegre Porto Alegre, Brazil
  • Detska Interni Klinika, Lf Mu A Fn Brno Brno, Czechia
  • Rigshospitalet Copenhagen, Denmark
View all 15 locations
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Completed

Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)

NCT04046224

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of California, Irvine Irvine, California, United States
  • University of South Florida Tampa, Florida, United States
  • Emory University School of Medicine Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
View all 18 locations
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Completed

Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease

NCT03566017

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 San Diego La Jolla, California, United States
  • University of California Irvine Center Orange, California, United States
View all 30 locations
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