NCT03727555 IT and IV Lentiviral Gene Therapy for X-ALD
| NCT ID | NCT03727555 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Shenzhen Geno-Immune Medical Institute |
| Condition | X-linked Adrenoleukodystrophy |
| Study Type | INTERVENTIONAL |
| Enrollment | 30 participants |
| Start Date | 2025-08-31 |
| Primary Completion | 2027-12-31 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
This trial targets 30 participants in total. It began in 2025-08-31 with a primary completion date of 2027-12-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 Criteria
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
Contact & Investigator
Frequently Asked Questions
Who can join the NCT03727555 clinical trial?
This trial is open to participants of all sexes, aged 1 Year or older, up to 60 Years, studying X-linked Adrenoleukodystrophy. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT03727555 currently recruiting?
Yes, NCT03727555 is actively recruiting participants. Contact the research team at c@szgimi.org for enrollment information.
Where is the NCT03727555 trial being conducted?
This trial is being conducted at Shenzhen, China.
Who is sponsoring the NCT03727555 clinical trial?
NCT03727555 is sponsored by Shenzhen Geno-Immune Medical Institute. The trial plans to enroll 30 participants.