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NOT YET RECRUITING
NCT07479953
PHASE1

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

Sponsor: Tippi Mackenzie

View on ClinicalTrials.gov

Summary

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

Official title: A Phase I Study of Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis

Key Details

Gender

All

Age Range

Any - Any

Study Type

INTERVENTIONAL

Enrollment

5

Start Date

2026-06-01

Completion Date

2055-06-01

Last Updated

2026-04-13

Healthy Volunteers

No

Interventions

DRUG

Gene Transfer with an AAV9 Vector Expressing Human ß-galactosidase

Prenatal administration of an AAV9 Vector Expressing Human ß-galactosidase