NCT01560182 - Gene Therapy for Metachromatic Leukodystrophy (MLD) | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

April 9, 2010

Primary Completion Date

April 9, 2018

Study Completion Date

September 19, 2025

Conditions

Lysosomal Storage DiseaseMetachromatic Leukodystrophy

Interventions

GENETIC

OTL-200 Gene Therapy

Autologous hematopoietic stem/progenitor cells collected from the bone marrow and transduced ex vivo with a Lentiviral vector encoding the human ARSA cDNA

Trial Locations (1)

20132

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