IT and IV Lentiviral Gene Therapy for X-ALD
ConditionX-linked Adrenoleukodystrophy
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age1-60
SponsorShenzhen Geno-Immune Medical Institute
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.
Eligibility criteria
Qualifiers
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
Disqualifiers
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
Trial design
Treatments tested in this trial
- Intrathecal and intravenous LV gene therapy
Treatment groups
30 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov