RecruitingNot Applicable

IT and IV Lentiviral Gene Therapy for X-ALD

China30 participantsStarted 2025-08-31

Plain-language 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.

Who can participate

Age range

1 Year – 60 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. X-ALD patients ≥ 1 year of age
. ALD diagnosis of the brain: evaluation of the VLCFA value in plasma
. Central imaging of the MRI to examine the damage on the CNS.
. Neurological function score (NFS) ≥ 1
. Parent / guardian / patient signing informed consent
. Patients and their families have a strong willingness to participate in clinical trials, and are willing to bear all the consequences caused by the failure of the trial, and sign an informed consent form

Exclusion criteria

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Safety evaluation of IT and IV injections of lentiviral TYF-ABCD1, determined by number of participants with treatment-related adverse events (AEs), according to scheduled assessments, vital signs, & physical examinations as assessed by CTCAE v4.0.

Timeframe: Minimum 1 day, maximum 1 year follow up

Altered disease progression

Timeframe: Minimum 6 months, maximum 3 year follow up

Assess disease progression

Timeframe: Minimum 6 months, maximum 3 year follow up

Trial details

NCT IDNCT03727555

SponsorShenzhen Geno-Immune Medical Institute

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2027-12-31

Contact for this trial

Lung-Ji Chang, Ph.D

86-13671121909 c@szgimi.org

View on ClinicalTrials.gov More X-linked Adrenoleukodystrophy trials