An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 (Sebelipase Alfa) in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency

Trial Profile

An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 (Sebelipase Alfa) in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency

Active, no longer recruiting
Phase of Trial: Phase II/III

Latest Information Update: 12 Mar 2018

At a glance

  • Drugs Sebelipase alfa (Primary)
  • Indications Wolman disease
  • Focus Registrational; Therapeutic Use
  • Acronyms VITAL
  • Sponsors Alexion Pharmaceuticals; Synageva BioPharma
  • Most Recent Events

    • 12 Mar 2018 The study has been completed in Ireland (end dare: 2018-01-03).
    • 12 Mar 2018 The study has been completed in France (end dare: 2018-01-03).
    • 06 Feb 2018 According to an Alexion Pharmaceuticals media release,data from the study were presented at WORLDSymposium(T) 2018.
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