Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial

Clinical trial NCT07479953 · NOT_YET_RECRUITING

Trial details

NCT number: NCT07479953
Sponsor: Tippi Mackenzie
Status: NOT_YET_RECRUITING
Phase: Phase 1
Study type: INTERVENTIONAL

GM1 Gangliosidoses GM1 Gangliosidosis, Type I GM1 Gangliosidosis, Type 2

Start date: 2026-06-01
Primary completion: 2038-06-01
Enrollment: 5

View on ClinicalTrials.gov

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