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A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant Human GAA) in Patients With Late-onset Pompe Disease

Trial Profile

A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant Human GAA) in Patients With Late-onset Pompe Disease

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 12 Jul 2019

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At a glance

  • Drugs Reveglucosidase alfa (Primary)
  • Indications Glycogen storage disease type II
  • Focus Adverse reactions; First in man; Pharmacodynamics
  • Sponsors BioMarin Pharmaceutical
  • Most Recent Events

    • 12 Jul 2019 This trial has been completed in United Kingdom, according to European Clinical Trials Database.
    • 12 Feb 2014 New trial record
    • 28 Jan 2014 Status changed from "active, no longer recruiting" to "completed" as reported by the European Clinical Trials Database.
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