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: 06 Mar 2017

At a glance

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

    • 06 Jun 2016 Planned End Date changed from 1 Feb 2019 to 1 Jun 2018.
    • 06 Jun 2016 Planned primary completion date changed from 1 Feb 2019 to 1 May 2018.
    • 06 Jun 2016 Status changed from recruiting to active, no longer recruiting.
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