NCT04283227

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

An Open Label, Non-randomized Trial to Evaluate the Safety and Efficacy of a Single Infusion of OTL-200 in Patients With Late Juvenile (LJ) Metachromatic Leukodystrophy (MLD).

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Orchard Therapeutics · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Conditions

• Lysosomal Storage Diseases
• Metachromatic Leukodystrophy

Interventions

Type	Name	Description
GENETIC	OTL-200	All subjects will receive OTL-200 gene therapy and will be followed up for 8 years following treatment with OTL-200.

Timeline

• Start date: 2022-01-17
• Primary completion: 2026-01-31
• Completion: 2031-03-31
• First posted: 2020-02-25
• Last updated: 2025-09-05

Locations

1 site across 1 country: Italy