clinical trial · NCT01560182
Gene Therapy for Metachromatic Leukodystrophy (MLD)
Lysosomal Storage DiseaseMetachromatic LeukodystrophyOTL-200 Gene Therapy
brief summary
This Phase I/II clinical trial consists of the application of lentiviral vector-based gene therapy to patients affected by Metachromatic Leukodystrophy (MLD), a rare inherited Lysosomal Storage Disorder (LSD) resulting from mutations in the gene encoding the Arylsulfatase A (ARSA) enzyme. The medicinal product consists of autologous CD34+ hematopoietic stem/progenitor cells in which a functional ARSA cDNA is introduced by means of 3rd generation VSV-G pseudotyped lentiviral vectors.
started
Apr 9, 2010
primary completion
Apr 9, 2018
completion
Sep 19, 2025
last updated
Dec 5, 2025
official title
A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol