OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)

Status Active, not recruiting

Clinical Trial Summary

OTL-200 is a cryopreserved dispersion for infusion containing autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene. MLD is an autosomal recessive lysosomal storage disorder (LSD) characterized by severe and progressive demyelination affecting the central and peripheral nervous system. The aim of this clinical study is to assess the pharmacodynamic effect and long-term clinical efficacy and safety of OTL-200 in Late Juvenile MLD patients.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04283227
Study type	Interventional
Source	Orchard Therapeutics
Contact
Status	Active, not recruiting
Phase	Phase 3
Start date	January 17, 2022
Completion date	March 31, 2031

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