Gene Therapy for Metachromatic Leukodystrophy (MLD)

Status Active, not recruiting

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT01560182
Study type	Interventional
Source	Orchard Therapeutics
Contact
Status	Active, not recruiting
Phase	Phase 1/Phase 2
Start date	April 9, 2010
Completion date	March 15, 2025

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See also
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