CompletedPHASE1, PHASE2NCT01801709

Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy

Studying Metachromatic leukodystrophy

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Key facts

Sponsor
Institut National de la Santé Et de la Recherche Médicale, France
Principal Investigator
Patrick Aubourg, MD-PhD
Assistance Publique - Hôpitaux de Paris and Institut National de la Santé et de la Recherche Médicale
Intervention
intracerebral administration of AAVrh.10cuARSA(genetic)
Enrollment
5 enrolled
Eligibility
5 years · All sexes
Timeline
2014 – 2022

Study locations (1)

Collaborators

European Leukodystrophy Association · Assistance Publique - Hôpitaux de Paris

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

Open NCT01801709 on ClinicalTrials.gov

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