A Multicenter, Open-Label Study of Sebelipase Alfa in Patients With Lysosomal Acid Lipase Deficiency

Trial Profile

A Multicenter, Open-Label Study of Sebelipase Alfa in Patients With Lysosomal Acid Lipase Deficiency

Active, no longer recruiting
Phase of Trial: Phase II

Latest Information Update: 27 Nov 2017

At a glance

  • Drugs Sebelipase alfa (Primary)
  • Indications Wolman disease
  • Focus Adverse reactions
  • Sponsors Alexion Pharmaceuticals; Synageva BioPharma
  • Most Recent Events

    • 25 Nov 2017 This trial has been completed in Germany.
    • 24 Oct 2017 Long term results of the study assessing Sebelipase Alfa treatment safety over 96 weeks in a diverse population of children and adults with lysosomal acid lipase deficiency, presented at The Liver Meeting 2017: 68th Annual Meeting of the American Association for the Study of Liver Diseases.
    • 22 Dec 2016 Planned End Date changed from 1 Feb 2018 to 1 Dec 2017.
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