Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD

Plain-language summary

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.

Trial details

Phase

Not Applicable

Status

Recruiting

Sponsor

Shenzhen Geno-Immune Medical Institute

Intervention

Lentiviral TYF-ARSA correction of patient's autologous HSCs

Start date

6/1/2025

Estimated completion

9/30/2030

Countries

China

Related genes

Locations

• Shenzhen, China

Eligibility criteria

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.