Direct Lentiviral TYF-ARSA Injection Gene Therapy for Metachromatic Leukodystrophy (MLD)
Status: Recruiting
Location: See location...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Not Applicable
SUMMARY
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 1 month
Maximum Age: 50
Healthy Volunteers: f
View:
• 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
Locations
Other Locations
China
Lung-Ji Chang
RECRUITING
Shenzhen
Contact Information
Primary
Lung-Ji Chang, Ph.D
c@szgimi.org
+86 0755-86573763
Time Frame
Start Date: 2025-05-31
Estimated Completion Date: 2030-12-31
Participants
Target number of participants: 10
Treatments
Experimental: Lentivirus-mediated delivery of ARSA to the CNS and the body
Intrathecal and intravenous injections with lentiviral TYF-ARSA vector carrying the functional gene
Related Therapeutic Areas
Sponsors
Leads: Shenzhen Geno-Immune Medical Institute