Clinical trial
Gene Therapy for Metachromatic Leukodystrophy (MLD)
NCT01560182 · Orchard Therapeutics, Industry · Completed
Key facts
| NCT number | NCT01560182 |
|---|---|
| Title | Gene Therapy for Metachromatic Leukodystrophy (MLD) |
| Official title | A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy |
| Lead sponsor | Orchard Therapeutics, Industry |
| Collaborators | Ospedale San Raffaele |
| Status | Completed |
| Phase | Phase 1/Phase 2 |
| Study type | Interventional |
| Conditions | Lysosomal Storage Disease, Metachromatic Leukodystrophy |
| Intervention type | Genetic |
| Interventions | OTL-200 Gene Therapy |
| Enrollment | 20 |
| Start date | Apr 9, 2010 |
| Primary completion | Apr 9, 2018 |
| Completion | Sep 19, 2025 |
| First posted | Mar 22, 2012 |
| Last update posted | Dec 5, 2025 |
| Countries | Italy |
At a glance
- 1–9 Study sites
- No Results posted
- None Linked FDA submissions
Counts are shown as ranges. Exact figures, trends and every underlying record are available in Orca1.
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Source: U.S. National Library of Medicine (NLM), ClinicalTrials.gov · Source date: