MedTrace Logo

Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD

NCT07046338 · Status: RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2025-07-01

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 transduce patient-derived hematopoietic stem cells (HSCs), with the goal of achieving therapeutic gene correction through transplantation of genetically modified HSCs. The primary objectives are to evaluate the safety and efficacy of the gene therapy clinical protocol.

Conditions

  • Metachromatic Leukodystrophy (MLD)

Interventions

GENETIC

Lentiviral TYF-ARSA correction of patient's autologous HSCs

Infusion of lentiviral TYF-ARSA modified autologous HSCs at 1\~10x10\^6 gene-modified cells per kg body weight.

Sponsors & Collaborators

  • Shenzhen Geno-Immune Medical Institute

    lead OTHER

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
2025-06-01
Primary Completion
2025-06-01
Completion
2030-09-30

Countries

  • China

Study Locations

More Related Trials

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 NCT07046338 on ClinicalTrials.gov