# A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)

> **NCT03392987** · PHASE2 · COMPLETED · sponsor: **Orchard Therapeutics** · enrollment: 10 (actual)

## Conditions studied

- Lysosomal Storage Disease
- Metachromatic Leukodystrophy

## Interventions

- **GENETIC:** OTL-200

## Key facts

- **NCT ID:** NCT03392987
- **Lead sponsor:** Orchard Therapeutics
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2018-01-25
- **Primary completion:** 2022-04-06
- **Final completion:** 2026-03-10
- **Target enrollment:** 10 (ACTUAL)
- **Last updated:** 2026-03-16

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT03392987

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT03392987, "A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT03392987. Licensed CC0.

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