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Gene Therapy for Metachromatic Leukodystrophy (MLD)

NCT01560182 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2025-12-05

No results posted yet for this study

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.

Conditions

  • Lysosomal Storage Disease
  • Metachromatic Leukodystrophy

Interventions

GENETIC

OTL-200 Gene Therapy

Autologous hematopoietic stem/progenitor cells collected from the bone marrow and transduced ex vivo with a Lentiviral vector encoding the human ARSA cDNA

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

Max Age
7 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2010-04-09
Primary Completion
2018-04-09
Completion
2025-09-19

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 NCT01560182 on ClinicalTrials.gov