Gene Therapy for Metachromatic Leukodystrophy (MLD)

Plain-language summary

This study evaluates a gene therapy (OTL-200) in children with Metachromatic Leukodystrophy (MLD, an inherited metabolic disease affecting the nervous system). It included pre-symptomatic late-infantile or early-symptomatic early-juvenile MLD patients. It was a Phase 1/Phase 2 study and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Completed

Sponsor

Orchard Therapeutics

Intervention

OTL-200 Gene Therapy

Start date

4/9/2010

Estimated completion

9/19/2025

Countries

Italy

Related genes

Locations

• Milan, Italy

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.