A Phase I/II, Open Labeled, Monocentric Study of Direct Intracranial Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human ARSA cDNA to Children With Metachromatic Leukodystrophy.

Trial Profile

A Phase I/II, Open Labeled, Monocentric Study of Direct Intracranial Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human ARSA cDNA to Children With Metachromatic Leukodystrophy.

Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 25 Jul 2017

At a glance

  • Drugs Arylsulfatase A gene therapy (Primary)
  • Indications Metachromatic leukodystrophy
  • Focus Adverse reactions; Therapeutic Use
  • Acronyms TG-MLD
  • Sponsors INSERM
  • Most Recent Events

    • 20 Jul 2017 Planned End Date changed from 1 Apr 2018 to 1 Apr 2019.
    • 20 Jul 2017 Status changed from recruiting to active, no longer recruiting.
    • 11 Mar 2013 New trial record
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