NCT07479953

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

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

• Status: Not Yet Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 5 (estimated)
• Sponsor: Tippi Mackenzie · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

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

Conditions

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

Interventions

Type	Name	Description
DRUG	Gene Transfer with an AAV9 Vector Expressing Human ß-galactosidase	Prenatal administration of an AAV9 Vector Expressing Human ß-galactosidase

Timeline

• Start date: 2026-06-01
• Primary completion: 2038-06-01
• Completion: 2055-06-01
• First posted: 2026-03-18
• Last updated: 2026-04-13

Regulatory

• FDA-regulated drug study