An Open-label, Multicenter, Multinational Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid Alpha-glucosidase Treatment in Patients Less Than 6 Months Old With Infantile-onset Pompe Disease

Trial Profile

An Open-label, Multicenter, Multinational Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid Alpha-glucosidase Treatment in Patients Less Than 6 Months Old With Infantile-onset Pompe Disease

Completed
Phase of Trial: Phase II/III

Latest Information Update: 19 May 2014

At a glance

  • Drugs Alglucosidase alfa (Primary)
  • Indications Glycogen storage disease type II
  • Focus Adverse reactions; Pharmacodynamics; Pharmacokinetics; Registrational; Therapeutic Use
  • Sponsors Genzyme Corporation
  • Most Recent Events

    • 07 Jun 2017 Biomarkers information updated
    • 19 May 2014 New trial record
    • 24 Jul 2006
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