IT and IV Lentiviral Gene Therapy for X-ALD
Intrathecal and Intravenous Lentiviral Gene Therapy for X-linked Adrenoleukodystrophy (X-ALD)
Shenzhen Geno-Immune Medical Institute
30 participants
Aug 31, 2025
INTERVENTIONAL
Conditions
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.
Eligibility
Plain Language Summary
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Interventions
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)
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NCT03727555