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Clinical trial

A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis

NCT03952637 is registered in ClinicalTrials.gov with National Human Genome Research Institute (NHGRI) as lead sponsor.

NCT NCT03952637
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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis
Lead sponsor
National Human Genome Research Institute (NHGRI)
Phase
Phase 1/2
Overall status
Recruiting
Start date
August 19, 2019
Completion date
January 1, 2030
Enrollment
54 (estimated)
Sites
1

Conditions

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

gangliosidosis, gm1, lysosomal diseases