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Clinical trial

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.

NCT01801709 is registered in ClinicalTrials.gov with Institut National de la Santé Et de la Recherche Médicale, France as lead sponsor.

NCT NCT01801709
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Registry record

As filed in ClinicalTrials.gov.

Title
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.
Lead sponsor
Institut National de la Santé Et de la Recherche Médicale, France
Phase
Phase 1/2
Overall status
Completed
Start date
June 30, 2014
Completion date
December 20, 2022
Enrollment
5 (actual)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

metachromatic leukodystrophy