IT and IV Lentiviral Gene Therapy for X-ALD
Recruiting
N/A
Interventional Study
X-linked Adrenoleukodystrophy
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 1 – 60
- Sex
- Any
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 30 (estimated)
- Sponsor
- Shenzhen Geno-Immune Medical Institute · Other
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About This Trial
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.
Trial Locations
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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 a…
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