Actively Recruiting
Direct Lentiviral Injection Gene Therapy for MLD
Led by Shenzhen Geno-Immune Medical Institute · Updated on 2025-06-26
10
Participants Needed
1
Research Sites
291 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 functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct gene transfer clinical protocol.
CONDITIONS
Official Title
Direct Lentiviral Injection Gene Therapy for MLD
Who Can Participate
Eligibility Criteria
You may qualify if you...
- MLD patient age >= 1 month
- ARSA gene sequence analysis confirming MLD mutations
- Brain MRI scoring system confirming MLD
- Parent, guardian, or patient signing informed consent
- Strong willingness by patients and families to participate and accept consequences of trial outcomes, with signed informed consent
You will not qualify if you...
- HIV positive patients
- Uncontrolled viral, bacterial, or fungal infections
- Malignant tumors
- Heart abnormalities
- Liver dysfunction
- Renal insufficiency
- Unable to perform MRI
- Infection or skin disease at pre-injection site
- Any condition increasing risk or interfering with trial results, including other neurological disorders besides MLD
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Lung-Ji Chang
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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