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Phase 3 study of venglustat versus continued imiglucerase in Gaucher disease type 3 patients stable on enzyme replacement therapy
jRCT2021220029Phase 3Active, not recruiting
- Sponsor
- Sanofi
- Start
- Jun 2023
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Small Molecules
01
Overview
Phase 3, randomised, double-blind trial in Gaucher disease type 3 comparing Venglustat arm with Imiglucerase active comparator arm (2 arms). Primary endpoints: Change in modified SARA total score and Change in RBANS total scale index score. Started Jun 2023; status: active, not recruiting.
02
Drugs
venglustatimiglucerase
03
Conditions
Gaucher disease type 3
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