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A Single Center, Open-Label, Non-Randomized, Uncontrolled, Multiple-Dose, Dose Escalation Study of the Safety, Pharmacokinetics, Efficacy and Long Term Safety of HGT-1111 (Recombinant Human Arylsulfatase A [rhASA, Metazym]) for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

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Trial Profile

A Single Center, Open-Label, Non-Randomized, Uncontrolled, Multiple-Dose, Dose Escalation Study of the Safety, Pharmacokinetics, Efficacy and Long Term Safety of HGT-1111 (Recombinant Human Arylsulfatase A [rhASA, Metazym]) for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 07 Aug 2015

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At a glance

  • Drugs Cerebroside-sulfatase (Primary)
  • Indications Metachromatic leukodystrophy
  • Focus Therapeutic Use
  • Sponsors Shire

    • Most Recent Events

    • 31 Aug 2018 Biomarkers information updated
    • 07 May 2014 New trial record
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