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

> **NCT01801709** · PHASE1,PHASE2 · COMPLETED · sponsor: **Institut National de la Santé Et de la Recherche Médicale, France** · enrollment: 5 (actual)

## Conditions studied

- Metachromatic Leukodystrophy

## Interventions

- **GENETIC:** intracerebral administration of AAVrh.10cuARSA

## Key facts

- **NCT ID:** NCT01801709
- **Lead sponsor:** Institut National de la Santé Et de la Recherche Médicale, France
- **Sponsor class:** OTHER_GOV
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2014-06
- **Primary completion:** 2016-06
- **Final completion:** 2022-12-20
- **Target enrollment:** 5 (ACTUAL)
- **Last updated:** 2026-03-02

## Collaborators

- [object Object]
- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01801709

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01801709, "Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy". Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/clinical/NCT01801709. Licensed CC0.

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