A Phase I/II clinical trial of hematopoietic stem cell gene therapy for the treatment of Metachromatic Leukodystrophy - TIGET-MLD

• Conditions: metachromatic leukodystrophy; MedDRA version: 9.1Level: PTClassification code 10024381

• Registration Number: EUCTR2009-017349-77-IT
• Lead Sponsor: FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-04-26
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Pre-symptomatic late infantile patients; Pre- or early-symptomatic early juvenile patients; Parental/guardian/patient signed informed consent.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Patients will be excluded from the present study according to the following crite-ria: HIV-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; Patients who underwent allogeneic hematopoietic stem cell transplantation in the previous 6 months; Patients who underwent allogeneic hematopoietic stem cell transplantation with evidence of residual cells of donor origin.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified