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

Key facts

Sponsor

National Human Genome Research Institute (NHGRI)

Principal Investigator

Cynthia J Tifft, M.D.

National Human Genome Research Institute (NHGRI)

Intervention

AAV9-GLB1(biological)

Enrollment

54 enrolled

Eligibility

12 years · All sexes

Timeline

2019 – 2028

Study locations (1)

• National Institutes of Health Clinical Center, Bethesda, Maryland, United States

Collaborators

Sio Gene Therapies

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

Other trials for Gangliosidosis

Additional recruiting or active studies for the same condition.

• RECRUITINGPHASE3NCT07082543
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• RECRUITINGNCT00668187
  A Natural History Study of the Gangliosidoses
  University of Minnesota

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