A Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease

Trial Profile

A Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease

Discontinued
Phase of Trial: Phase II

Latest Information Update: 27 Mar 2017

At a glance

  • Drugs Reveglucosidase alfa (Primary)
  • Indications Glycogen storage disease type II
  • Focus Therapeutic Use
  • Acronyms INSPIRE
  • Sponsors BioMarin Pharmaceutical
  • Most Recent Events

    • 15 Aug 2016 Status changed from recruiting to discontinued.
    • 11 Jan 2016 According to a BioMarin Pharmaceutical media release, this study was changed from a phase II/III to a phase II study to allow use of drug employing a new purification process.
    • 11 Jan 2016 Interim results (n=24) published in a BioMarin Pharmaceutical media release.
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