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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

NCT07479953 is registered in ClinicalTrials.gov with Tippi Mackenzie as lead sponsor.

NCT NCT07479953
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Registry record

As filed in ClinicalTrials.gov.

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
Lead sponsor
Tippi Mackenzie
Phase
Phase 1
Overall status
Not yet recruiting
Start date
January 1, 2027
Completion date
June 1, 2055
Enrollment
5 (estimated)
Sites
Not reported

Conditions

As the registry lists them; a condition with its own page is linked.

gm1 gangliosidoses, gm1 gangliosidosis, type 2, gm1 gangliosidosis, type i