TRIALSITEIQ PUBLIC FEASIBILITY SCORECARD · GRADE C

NCT03952637: 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

Sponsor: National Human Genome Research Institute (NHGRI) · Phase: PHASE1/PHASE2 · Area: CARDIOLOGY_METABOLIC · Target Enrollment: 54 pts

Competing Trials Overlap

3 Active Trials

Excess Burn Exposure

$9,030,400

Rescue Site Savings

+$5,954,420

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