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Venglustat in late-onset GM2 gangliosidosis
2019-002375-34Phase 3Terminated
- Sponsor
- Sanofi
- Start
- Nov 2019
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 3, randomised, double-blind trial in GM2 gangliosidosis, Tay-Sachs disease (TSD) and Sandhoff disease comparing Venglustat with Placebo (2 arms). Primary endpoint: Change in cerebrospinal fluid GM2 biomarker. Started Nov 2019; status: terminated.
02
Drugs
venglustat
03
Conditions
GM2 gangliosidosisTay-Sachs disease (TSD)Sandhoff disease
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