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: 30 Sep 2017

At a glance

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

    • 22 Sep 2017 The study has been completed in United Kingdom (End date : 2016-03-21)
    • 06 Oct 2016 According to an Alexion Pharmaceuticals media release, new data from the study were presented at the 5th World Congress of Pediatric Gastroenterology, Hepatology and Nutrition (WCPGHAN).
    • 16 Apr 2016 Planned End Date changed from 1 Dec 2015 to 1 Apr 2018.
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top