X-linked adrenoleukodystrophy
Conditions
Interventions
experimental group:lentiviral vectors carrying modified genes
Sponsors
Senior department of Pediatrics, Chinese PLA General Hospital; Department of Pediatrics, the First Medical Center of Chinese PLA General Hospital
Eligibility
Sex/Gender
Male
Age
6 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. ALD was diagnosed by evaluating VLCFA and brain MRI imaging examination; 2. Neurological function score (NFS) =0, =10; 3. Diagnosis of ALD by ABCD1 genetic diagnosis, abnormal MRI images, abnormally high levels of VLCFA and adrenocorticotropic hormone (ACTH); 4. Age >= 6 years; 5. Informed consent signed by the patient, guardian or power of attorney.
Exclusion criteria
Exclusion criteria: 1. The disease progresses rapidly; 2. The patient is undergoing other clinical trials or has previously used other gene therapy products; 3. Abnormal blood routine and/or liver and kidney function; 4. Left ventricular ejection fraction < 40%, impaired heart function; 5. Clinically significant active bacterial, viral, fungal, parasitic or prion-related infections; 6. Any conditions (including allergies to anesthetics or contrast media) that preclude MRI studies; 7. Any clinically significant cardiovascular or pulmonary disease, or any other disease or condition contraindicated by any other study procedure.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Neurological Function Scale score;Loes score;Gene copy number;Very long chain fatty acids; | — |
Countries
China
Contacts
Public ContactLiping Zou
Senior department of Pediatrics, Chinese PLA General Hospital; Department of Pediatrics, the First Medical Center of Chinese PLA General Hospital?
Outcome results
None listed