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Gene Therapy for Metachromatic Leukodystrophy (MLD) | Clinical Trials | Clareo Health

COMPLETEDPHASE1/PHASE2

Gene Therapy for Metachromatic Leukodystrophy (MLD)

A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy

NCT01560182•Orchard Therapeutics

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.

Eligibility

Age

0 - 7 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Pre-symptomatic MLD patients with the late infantile variant;
•Pre- or early-symptomatic MLD patients with the early juvenile variant;
•Patients for whom parental/guardian signed informed consent has been obtained.

Exclusion Criteria

•HIV RNA and/or HCV RNA and/or HBV DNA positive patients;
•Patients affected by neoplastic diseases;
•Patients with cytogenetic alterations typical of MDS/AML;
•Patients with end-organ functions or any other severe disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study;
•Patients enrolled in other trials/other therapeutic approaches that might become available;
•Patient who underwent allogeneic hematopoietic stem cell transplantation in the previous six months;
•Patient who underwent allogenic hematopoietic stem cell transplantation with evidence of residual cells of donor origin.

Locations (1)

Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)

Milan, Italy

Key Dates

Start Date

April 9, 2010

Primary Completion Date

April 9, 2018

Completion Date

September 19, 2025

Last Updated

December 5, 2025

Enrollment

ACTUAL participants

Conditions

Lysosomal Storage DiseaseMetachromatic Leukodystrophy

Interventions

OTL-200 Gene Therapy

GENETIC

The Myelin Disorders Biorepository Project

NCT03047369

LeukodystrophyWhite Matter Disease+64 more

View study

RECRUITINGNA

Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD

NCT07046338

Metachromatic Leukodystrophy (MLD)

View study

ACTIVE_NOT_RECRUITINGPHASE1/PHASE2

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: December 5, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Gene Therapy for Metachromatic Leukodystrophy (MLD)

Inclusion Criteria

Exclusion Criteria

The Myelin Disorders Biorepository Project

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