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Clinical trial

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).

NCT04283227 is registered in ClinicalTrials.gov with Orchard Therapeutics as lead sponsor.

NCT NCT04283227
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Registry record

As filed in ClinicalTrials.gov.

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
Phase
Phase 3
Overall status
Active, not recruiting
Start date
January 17, 2022
Completion date
March 31, 2031
Enrollment
6 (actual)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

lysosomal storage diseases, metachromatic leukodystrophy