Clinical trial
OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)
NCT04283227 · Orchard Therapeutics, Industry · Active, not recruiting
Key facts
| NCT number | NCT04283227 |
|---|---|
| Title | OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD) |
| Official title | An Open Label, Non-randomized Trial to Evaluate the Safety and Efficacy of a Single Infusion of OTL-200 in Patients With Late Juvenile (LJ) Metachromatic Leukodystrophy (MLD). |
| Lead sponsor | Orchard Therapeutics, Industry |
| Collaborators | Ospedale San Raffaele |
| Status | Active, not recruiting |
| Phase | Phase 3 |
| Study type | Interventional |
| Conditions | Lysosomal Storage Diseases, Metachromatic Leukodystrophy |
| Intervention type | Genetic |
| Interventions | OTL-200 |
| Enrollment | 6 |
| Start date | Jan 17, 2022 |
| Primary completion | Jan 31, 2026 |
| Completion | Mar 31, 2031 |
| First posted | Feb 25, 2020 |
| Last update posted | Sep 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: