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Direct Lentiviral Injection Gene Therapy for MLD

NCT03725670 · Status: NOT_YET_RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2026-09-01

No results posted yet for this study

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.

Conditions

  • Metachromatic Leukodystrophy (MLD)

Interventions

GENETIC

Intrathecal and intravenous LV gene therapy

Direct IT and IV LV gene therapy to deliver high levels of LVs at 1-2×10\^9 transduction units/ml which carry a normal ARSA gene

Sponsors & Collaborators

  • Shenzhen Geno-Immune Medical Institute

    lead OTHER

Principal Investigators

  • Lung-Ji Chang, Ph.D · Shenzhen Geno-Immune Medical Institute

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Month
Max Age
50 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2027-05-31
Primary Completion
2028-06-01
Completion
2028-12-31

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03725670 on ClinicalTrials.gov