Actively Recruiting
Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD
Led by Shenzhen Geno-Immune Medical Institute · Updated on 2025-07-01
10
Participants Needed
1
Research Sites
278 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 1 month or older
- Confirmed MLD mutations by ARSA gene sequence analysis
- Ability to undergo brain MRI scanning
- Signed informed consent by parent, guardian, or patient
- Strong willingness to participate and accept trial outcomes
You will not qualify if you...
- HIV positive status
- Uncontrolled infections, malignant tumors, heart abnormalities, liver or kidney dysfunction
- Inability to perform MRI
- Infection or skin disease at infusion site
- Other neurological disorders besides MLD that increase risk or interfere with trial results
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Shenzhen Geno-Immune Medical Institute
Shenzhen, Guangdong, China, 518000
Actively Recruiting
Research Team
L
Lung-Ji Chang, Ph.D
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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