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A Multinational Randomized Double-blind Placebo-controlled Study to Assess the Efficacy Pharmacodynamics Pharmacokinetics and Safety of Venglustat in Late-onset GM2

A Multinational Randomized Double-blind Placebo-controlled Study to Assess the Efficacy Pharmacodynamics Pharmacokinetics and Safety of Venglustat in Late-onset GM2

Recruiting
2 years and older
All
Phase 3
  • Simplified description (AI rewritten)
  • Technical description (Original)

The total duration is up to approximately 119 weeks, including a 60-day screening period, a 104-week treatment period, and a 6-week post-treatment safety observation period.

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Study details
    Tay-Sachs Disease Sandhoff Disease

NCT04221451

Genzyme, a Sanofi Company

19 February 2024

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