# Gene Therapy for Metachromatic Leukodystrophy (MLD)

> **NCT01560182** · PHASE1,PHASE2 · COMPLETED · sponsor: **Orchard Therapeutics** · enrollment: 20 (actual)

## Conditions studied

- Lysosomal Storage Disease
- Metachromatic Leukodystrophy

## Interventions

- **GENETIC:** OTL-200 Gene Therapy

## Key facts

- **NCT ID:** NCT01560182
- **Lead sponsor:** Orchard Therapeutics
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2010-04-09
- **Primary completion:** 2018-04-09
- **Final completion:** 2025-09-19
- **Target enrollment:** 20 (ACTUAL)
- **Last updated:** 2025-12-05

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01560182

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01560182, "Gene Therapy for Metachromatic Leukodystrophy (MLD)". Retrieved via AI Analytics 2026-06-03 from https://api.ai-analytics.org/clinical/NCT01560182. Licensed CC0.

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