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2 clinical studies listed.
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Tundra lists 2 GM1 Gangliosidosis, Type 2 clinical trials. Each listing includes eligibility criteria, study locations, and direct links to research sites in the Tundra directory.
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NCT04713475
Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis
PBGM01 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the brain and peripheral tissues. This study will assess in a 2 part design the safety, tolerability and efficacy of PBGM01 in patients with early onset infantile (Type 1) and late onset infantile (Type 2a) GM1 gangliosidosis
Gender: All
Ages: 1 Month - 24 Months
Updated: 2026-06-30
4 states
NCT07479953
Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial
This is a study for the administration of in utero AAV9 transfer in prenatally diagnosed Type I or Type II GM1.
Gender: All
Updated: 2026-04-13