An Open Label Multicenter Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 in Adult Subjects With Liver Dysfunction Due to Lysosomal Acid Lipase Deficiency Who Previously Received Treatment in Study LAL-CL01

Trial Profile

An Open Label Multicenter Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 in Adult Subjects With Liver Dysfunction Due to Lysosomal Acid Lipase Deficiency Who Previously Received Treatment in Study LAL-CL01

Active, no longer recruiting
Phase of Trial: Phase II

Latest Information Update: 08 Mar 2017

At a glance

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

    • 28 Mar 2016 According to Alexion Pharmaceuticals media release, Ministry of Health, Labour and Welfare (MHLW) has approved Kanuma (sebelipase alfa) for the treatment of patients of all ages in Japan with lysosomal acid lipase deficiency (LAL-D).
    • 03 Mar 2016 Long-term safety analysis from this trial were presented at the 12th Annual WORLDSymposium, according to an Alexion Pharmaceuticals media release.
    • 18 May 2015 Planned End Date changed from 1 Jan 2015 to 1 Jun 2017, as reported by ClinicalTrials.gov.
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