This study examines a gene therapy in which a patient's own stem cells are modified using a lentiviral approach to correct the ARSA gene in people with metachromatic leukodystrophy (MLD). It is open to patients aged 1 month and older whose MLD diagnosis has been confirmed by ARSA gene sequence analysis. It is a study without an assigned phase and is currently recruiting participants.
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: 1. age \>= 1 month 2. ARSA gene sequence analysis to confirm MLD mutations 3. Brain MR Imaging 4. Parent / guardian / patient signing informed consent 5. Patients and their families have a strong willingness to participate in clinical trials, are willing to bear all the consequences caused by the failure of the trial, and sign the informed consent Exclusion Criteria: 1. HIV positive 2. Experiencing uncontrolled viral, bacterial or fungal infections, malignant tumors, heart abnormalities, liver dysfunction, or renal insufficiency 3. Cannot perform an MRI 4. Infection or dermatosis at infusion site 5. Any condition that may increase the subject's risk or interfere with the results of the trial, e.g. in addition to MLD, there are other neurological disorders.