A Single Center, Open-label, Non-randomized, Uncontrolled, Multiple-dose, Dose Escalation Study of the Safety, Pharmacokinetics and Efficacy of Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

Trial Profile

A Single Center, Open-label, Non-randomized, Uncontrolled, Multiple-dose, Dose Escalation Study of the Safety, Pharmacokinetics and Efficacy of Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

Completed
Phase of Trial: Phase I

Latest Information Update: 29 Apr 2016

At a glance

  • Drugs Cerebroside-sulfatase (Primary)
  • Indications Metachromatic leukodystrophy
  • Focus Adverse reactions; Pharmacokinetics
  • Sponsors Shire
  • Most Recent Events

    • 07 Jun 2017 Biomarkers information updated
    • 20 Jun 2012 New trial record
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