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

NCT04283227 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2025-09-05

No results posted yet for this study

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

GENETIC

OTL-200

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

Sponsors & Collaborators

  • Ospedale San Raffaele

    collaborator OTHER

  • Orchard Therapeutics

    lead INDUSTRY

Principal Investigators

  • Orchard Clinical Trials · Orchard Therapeutics

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-01-17
Primary Completion
2026-01-31
Completion
2031-03-31

Countries

  • Italy

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04283227 on ClinicalTrials.gov