A Phase 2, Open-Label, Multi-Center Study Evaluating the Efficacy, Safety and Pharmacokinetics of Genz-112638 in Gaucher Type 1 Patients

Trial Profile

A Phase 2, Open-Label, Multi-Center Study Evaluating the Efficacy, Safety and Pharmacokinetics of Genz-112638 in Gaucher Type 1 Patients

Completed
Phase of Trial: Phase II

Latest Information Update: 12 Dec 2017

At a glance

  • Drugs Eliglustat (Primary)
  • Indications Gaucher's disease type I
  • Focus Therapeutic Use
  • Sponsors Genzyme Corporation
  • Most Recent Events

    • 12 Dec 2017 Results (8-year data) assessing improvements in hemoglobin concentration and platelet count from this and one other trial, presented at the 59th Annual Meeting and Exposition of the American Society of Hematology.
    • 25 Jun 2017 Final 8 year results from the study, presented at the 22nd Congress of the European Haematology Association.
    • 10 Jun 2017 Biomarkers information updated
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