Clinical Trial

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

Name: Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial — clinical trial record (NCT07479953)
Creator: DataLookout
License: https://creativecommons.org/licenses/by/4.0/

Not Yet Recruiting Phase 1

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Summary

This is a study for the administration of in utero AAV9 transfer in prenatally diagnosed Type I or Type II GM1.

Protocol Amendment History 1 amendment

This ClinicalTrials.gov record has been amended once since 2026-03-14.

Trial Details

NCT Number	NCT07479953
Lead Sponsor	Tippi Mackenzie
Conditions	GM1 Gangliosidoses, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2
Enrollment	5 participants
Start Date	2026-06-01
Primary Completion	2038-06-01 (estimated)
Study Completion	2055-06-01 (estimated)
Updated on ClinicalTrials.gov	2026-04-13