Gene Therapy for Metachromatic Leukodystrophy (MLD)

Orchard Therapeutics

Status and phase

Active, not recruiting

Phase 2

Phase 1

Conditions

Lysosomal Storage Disease

Metachromatic Leukodystrophy

Treatments

Genetic: OTL-200 Gene Therapy

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT01560182

Eudract 2009-017349-77 (Other Identifier)

201222

Details and patient eligibility

About

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.

Enrollment

20 patients

Sex

All

Ages

Under 7 years old

Volunteers

No 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.