Recruiting studies: Gm2 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 →

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Phase 1 · 1 location(s) · Joanne Kurtzberg, MD · NCT02254863 A Natural History Study of the Gangliosidoses
Phase N/A · 1 location(s) · University of Minnesota · NCT00668187 A 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. · NCT07399704 The Myelin Disorders Biorepository Project
Phase N/A · 23 location(s) · Children's Hospital of Philadelphia · NCT03047369 Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders
Phase N/A · 1 location(s) · National Human Genome Research Institute (NHGRI) · NCT00029965 Longitudinal Study of Neurodegenerative Disorders
Phase N/A · 1 location(s) · University of Pittsburgh · NCT03333200 A 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. · NCT07054515 A 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.