This study examines a lentiviral gene therapy delivered by direct injection (into the spinal fluid and into a vein) in the inherited disease metachromatic leukodystrophy (MLD). Patients aged 1 month and over whose MLD diagnosis is confirmed by ARSA gene analysis may take part. It is a study without an assigned phase and is currently still enrolling 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. MLD patient age \>= 1 month 2. ARSA gene sequence analysis to confirm MLD mutations 3. Scoring system for brain MR Imaging confirmed MLD 4. Parent / guardian / patient signing informed consent 5. Patients and their families have a strong willingness to participate in clinical trials, and are willing to bear all the consequences caused by the failure of the trial, and sign an informed consent form Exclusion Criteria: 1. HIV positive patients 2. Patients who are 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 pre-injection site 5. Any condition that may increase the subjects' risk or interfere with the results of the trial. In addition to MLD, there are other neurological disorders.