A Phase 2, Open Label, Multicenter Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Sebelipase Alfa in Infants with Rapidly Progressive Lysosomal Acid Lipase Deficiency

Trial Profile

A Phase 2, Open Label, Multicenter Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Sebelipase Alfa in Infants with Rapidly Progressive Lysosomal Acid Lipase Deficiency

Active, no longer recruiting
Phase of Trial: Phase II

Latest Information Update: 30 Sep 2017

At a glance

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

    • 05 Sep 2017 Planned End Date changed from 1 Jun 2018 to 1 Apr 2018.
    • 05 Sep 2017 Planned primary completion date changed from 1 May 2018 to 1 Apr 2018.
    • 06 Jun 2016 Planned End Date changed from 1 Feb 2019 to 1 Jun 2018.
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