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Clinical trial condition

Lysosomal Storage Diseases

Explore clinical trials studying Lysosomal Storage Diseases. Study availability and eligibility vary by location and protocol.

710 public trials 130 recruiting studies
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Lysosomal Storage Diseases trial results

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
View Trial Details
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
View Trial Details
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
View Trial Details

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