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.
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.