Actively Recruiting
IT and IV Lentiviral Gene Therapy for X-ALD
Led by Shenzhen Geno-Immune Medical Institute · Updated on 2025-09-09
30
Participants Needed
1
Research Sites
174 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 X-linked adrenoleukodystrophy using a high-safety, high-efficiency, self-inactivating lentiviral vector (LV) TYF-ABCD1 to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the intrathecal and intravenous lentiviral gene transfer clinical protocol.
CONDITIONS
Official Title
IT and IV Lentiviral Gene Therapy for X-ALD
Who Can Participate
Eligibility Criteria
You may qualify if you...
- X-ALD patients aged 1 year or older
- Diagnosis of ALD brain involvement confirmed by plasma VLCFA levels
- MRI imaging to examine central nervous system damage
- Neurological function score of 1 or higher
- Informed consent signed by patient, parent, or guardian
- Willingness to participate and accept possible trial outcomes
You will not qualify if you...
- HIV positive patients
- Patients stable after treatment with statins, Lorenzo's oil, or VLCFA-reducing diet
- Severe infections, malignant tumors, heart abnormalities, liver dysfunction, or kidney insufficiency
- Unable to undergo MRI
- Infection or skin disease at injection site
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
R
Rui Zhang, MSc
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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