Gene Therapy for Metachromatic Leukodystrophy (TIGET-MLD)
This study is ongoing, but not recruiting participants.
IRCCS San Raffaele
Information provided by (Responsible Party):
Alessandra Biffi, IRCCS San Raffaele
First received: March 16, 2012
Last updated: April 20, 2015
Last verified: April 2015
No Study Results Posted on ClinicalTrials.gov for this Study
|Study Status:||This study is ongoing, but not recruiting participants.|
|Estimated Study Completion Date:||April 2023|
|Estimated Primary Completion Date:||April 2018 (Final data collection date for primary outcome measure)|
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Sessa M, Lorioli L, Fumagalli F, Acquati S, Redaelli D, Baldoli C, Canale S, Lopez ID, Morena F, Calabria A, Fiori R, Silvani P, Rancoita PM, Gabaldo M, Benedicenti F, Antonioli G, Assanelli A, Cicalese MP, Del Carro U, Sora MG, Martino S, Quattrini A, Montini E, Di Serio C, Ciceri F, Roncarolo MG, Aiuti A, Naldini L, Biffi A. Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial. Lancet. 2016 Jul 30;388(10043):476-87. doi: 10.1016/S0140-6736(16)30374-9.