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LEAP2MONO: venglustat versus imiglucerase in Gaucher disease type 3 patients stable on enzyme replacement therapy
2024-514381-39-00Phase 3Authorised
- Sponsor
- Sanofi
- Start
- Dec 2022
- Modality
- Small Molecules
- Countries
- 11
01
Overview
Phase 3, randomised, double-blind trial of venglustat in Gaucher disease type 3 (2 arms), sponsored by Sanofi. Primary endpoints: Change in modified SARA total score and Change in RBANS total scale index score. Planned start Dec 2022; status: authorised.
02
Drugs
venglustat
03
Conditions
Gaucher disease type 3
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