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IT and IV Lentiviral Gene Therapy for X-ALD
Sponsor: Shenzhen Geno-Immune Medical Institute
Summary
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.
Official title: Intrathecal and Intravenous Lentiviral Gene Therapy for X-linked Adrenoleukodystrophy (X-ALD)
Key Details
Gender
All
Age Range
1 Year - 60 Years
Study Type
INTERVENTIONAL
Enrollment
30
Start Date
2025-08-31
Completion Date
2028-12-31
Last Updated
2025-09-09
Healthy Volunteers
No
Conditions
Interventions
Intrathecal and intravenous LV gene therapy
Direct IT and IV LV gene therapy to deliver high levels of LVs at 1-2×10\^9 multiplicity of infection/ml which carry normal ABCD1 gene
Locations (1)
Shenzhen Geno-immune Medical Institute
Shenzhen, Guangdong, China