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Phase 1/2 Dose-Escalation Study of SPK-3006 in Adults With Late-Onset Pompe Disease
2023-509303-32-00Phase 2Authorised
- Sponsor
- Genentech
- Start
- Oct 2021
- Modality
- Gene Therapies
- Countries
- 2
01
Overview
Phase 2, open-label, single-arm trial of vanglusagene ensiparvovec in Pompe disease, sponsored by Genentech. Primary endpoints: Incidence of adverse events and serious adverse events, Vector shedding and liver function tests and 1 more. Planned start Oct 2021; status: authorised.
02
Drugs
vanglusagene ensiparvovec
03
Conditions
Pompe disease
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