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RECRUITING
NCT03727555
NA

IT and IV Lentiviral Gene Therapy for X-ALD

Sponsor: Shenzhen Geno-Immune Medical Institute

View on ClinicalTrials.gov

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

Interventions

GENETIC

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