Status:
RECRUITING
IT and IV Lentiviral Gene Therapy for X-ALD
Lead Sponsor:
Shenzhen Geno-Immune Medical Institute
Conditions:
X-linked Adrenoleukodystrophy
Eligibility:
All Genders
1-60 years
Phase:
NA
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 ...
Detailed Description
X-linked adrenoleukodystrophy (X-ALD) is a devastating neurological disorder caused by mutations in the ABCD1 gene that encodes a peroxisomal ATP-binding cassette transporter (ABCD1). ABCD1 is respons...
Eligibility Criteria
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:
- HIV positive patients
- Stablized condition after statins, Lorenzo's oil, or diet to reduce VLCFA levels
- Patients who are experiencing severe viral, bacterial or fungal infections, malignant tumors, heart abnormalities, liver dysfunction, or renal insufficiency
- Cannot perform an MRI
- Infection or dermatosis at pre-injection site
Key Trial Info
Start Date :
August 31 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 31 2028
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT03727555
Start Date
August 31 2025
End Date
December 31 2028
Last Update
September 9 2025
Active Locations (1)
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1
Shenzhen Geno-immune Medical Institute
Shenzhen, Guangdong, China, 518000