IT and IV Lentiviral Gene Therapy for X-ALD

Plain-language summary

This Phase 1/2 trial uses a high-safety, self-inactivating lentiviral vector, TYF-ABCD1, to functionally correct the defective gene in X-linked adrenoleukodystrophy (X-ALD). The vector is delivered both intrathecally and intravenously, and the safety and efficacy of this protocol are being evaluated. Eligible participants are patients aged 1 year and older with an ALD diagnosis supported by plasma VLCFA levels and brain MRI, and a neurological function score (NFS) of 1 or higher.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Not Applicable

Status

Recruiting

Sponsor

Shenzhen Geno-Immune Medical Institute

Intervention

Intrathecal and intravenous LV gene therapy

Start date

8/31/2025

Estimated completion

12/31/2028

Countries

China

Locations

• Shenzhen, China

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

1. X-ALD patients ≥ 1 year of age
2. ALD diagnosis of the brain: evaluation of the VLCFA value in plasma
3. Central imaging of the MRI to examine the damage on the CNS.
4. Neurological function score (NFS) ≥ 1
5. Parent / guardian / patient signing informed consent
6. 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:

1. HIV positive patients
2. Stablized condition after statins, Lorenzo's oil, or diet to reduce VLCFA levels
3. Patients who are experiencing severe viral, bacterial or fungal infections, malignant tumors, heart abnormalities, liver dysfunction, or renal insufficiency
4. Cannot perform an MRI
5. Infection or dermatosis at pre-injection site