NCT03392987 - A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD) | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

January 25, 2018

Primary Completion Date

April 6, 2022

Study Completion Date

January 31, 2026

Conditions

Lysosomal Storage DiseaseMetachromatic Leukodystrophy

Interventions

GENETIC

OTL-200

OTL-200 is an autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with lentiviral vector that encodes for the human ARSA cDNA sequence

Trial Locations (1)

20132

Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET), Milan