# Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation

> **NCT01303146** · PHASE2 · COMPLETED · sponsor: **Assistance Publique - Hôpitaux de Paris** · enrollment: 1 (actual)

## Conditions studied

- Metachromatic Leukodystrophy

## Interventions

- **DRUG:** rhARSA

## Key facts

- **NCT ID:** NCT01303146
- **Lead sponsor:** Assistance Publique - Hôpitaux de Paris
- **Sponsor class:** OTHER
- **Phase:** PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2008-10
- **Primary completion:** 2010-03
- **Final completion:** 2010-04
- **Target enrollment:** 1 (ACTUAL)
- **Last updated:** 2026-03-27

## Collaborators

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## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01303146

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01303146, "Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT01303146. Licensed CC0.

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