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Condition: Gangliosidosis Synced from ClinicalTrials.gov · July 29, 2026

Recruiting studies: Gangliosidosis

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

Longitudinal Study of Neurodegenerative Disorders
Phase N/A · 1 location(s) · University of Pittsburgh · NCT03333200Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders
Phase N/A · 1 location(s) · National Human Genome Research Institute (NHGRI) · NCT00029965A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pi
Phase 2 · 3 location(s) · Azafaros B.V. · NCT07399704A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Typ
Phase 1/Phase 2 · 1 location(s) · National Human Genome Research Institute (NHGRI) · NCT03952637A Natural History Study of the Gangliosidoses
Phase N/A · 1 location(s) · University of Minnesota · NCT00668187UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Phase 1 · 1 location(s) · Joanne Kurtzberg, MD · NCT02254863FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
Phase N/A · 1 location(s) · xCures · NCT06539169The Myelin Disorders Biorepository Project
Phase N/A · 23 location(s) · Children's Hospital of Philadelphia · NCT03047369A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niem
Phase 3 · 38 location(s) · Azafaros B.V. · NCT07054515A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niem
Phase 3 · 35 location(s) · Azafaros B.V. · NCT07082725
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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.