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A 4-part, Open-label, Multicenter, Multinational Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic, and Exploratory Efficacy of Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

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Trial Profile

A 4-part, Open-label, Multicenter, Multinational Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic, and Exploratory Efficacy of Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

Status: Recruiting
Phase of Trial: Phase II

Latest Information Update: 18 Jul 2024

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At a glance

  • Drugs Venglustat (Primary) ; Imiglucerase
  • Indications Gaucher's disease type I; Gaucher's disease type III
  • Focus Adverse reactions; Biomarker; Pharmacodynamics
  • Acronyms LEAP
  • Sponsors Sanofi Genzyme

    • Most Recent Events

    • 12 Sep 2023 Planned number of patients changed from 15 to 18.
    • 12 Sep 2023 Planned number of patients changed from 15 to 18.
    • 13 Feb 2023 Results published in the Brain

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