Direct Lentiviral Injection Gene Therapy for MLD

Recruiting now · Not applicable

Conditions studied: Metachromatic Leukodystrophy (MLD)

In brief

This is a Phase I/II clinical trial of gene therapy for treating Metachromatic leukodystrophy (MLD) using a safety and efficacy improved self-inactivating lentiviral vector TYF-ARSA to functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct gene transfer clinical protocol.

Key facts

Study ID

NCT03725670

Run by

Shenzhen Geno-Immune Medical Institute

People needed

10

Starts

2025-05-31

Expected to finish

2030-12-31

Last updated by the study team

2026-06-23

Who can join

Age: 0 and older, up to 50. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• MLD patient age >= 1 month
• ARSA gene sequence analysis to confirm MLD mutations
• Scoring system for brain MR Imaging confirmed MLD
• Parent / guardian / patient signing informed consent
• 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

You may not qualify if…

• HIV positive patients
• Patients who are experiencing uncontrolled viral, bacterial or fungal infections, malignant tumors, heart abnormalities, liver dysfunction, or renal insufficiency
• Cannot perform an MRI
• Infection or dermatosis at pre-injection site
• 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.

Where it is running

• Lung-Ji Chang — Shenzhen, Guangdong, China (enrolling)

Full record on ClinicalTrials.gov

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