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Clinical trial

A Single Arm, Open Label, Clinical Study of Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Containing Human ARSA cDNA (OTL-200), for the Treatment of Early Onset Metachromatic Leukodystrophy (MLD)

NCT03392987 is registered in ClinicalTrials.gov with Orchard Therapeutics as lead sponsor.

NCT NCT03392987
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Registry record

As filed in ClinicalTrials.gov.

Title
A Single Arm, Open Label, Clinical Study of Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Containing Human ARSA cDNA (OTL-200), for the Treatment of Early Onset Metachromatic Leukodystrophy (MLD)
Lead sponsor
Orchard Therapeutics
Phase
Phase 2
Overall status
Completed
Start date
January 25, 2018
Completion date
March 10, 2026
Enrollment
10 (actual)
Sites
1

Conditions

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

lysosomal storage disease, metachromatic leukodystrophy