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IT and IV Lentiviral Gene Therapy for X-ALD

NCT03727555 · Status: NOT_YET_RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 30

Last updated 2026-09-01

No results posted yet for this study

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.

Conditions

  • X-linked Adrenoleukodystrophy

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

Sponsors & Collaborators

  • Shenzhen Geno-Immune Medical Institute

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
60 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2027-06-01
Primary Completion
2028-05-31
Completion
2028-12-31

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03727555 on ClinicalTrials.gov