adrenoleukodystrophy
Conditions
Interventions
experimental group:Direct injection lentiviral vectors with modified genes into the brain
Sponsors
The First Medical Center of PLA General Hospital
Eligibility
Sex/Gender
Male
Age
6 Years to No maximum
Inclusion criteria
Inclusion criteria: (1) Diagnosing ALD by evaluating the value of VLCFA and head MRI imaging; (2) Neurological function score (NFS) >= 0, = 6 years; (5) The patient, guardian or authorized power of attorney signs the informed consent form.
Exclusion criteria
Exclusion criteria: (1) The disease progresses rapidly; (2) Are undergoing other clinical trial studies, or have previously used other gene therapy products; (3) Abnormal blood and/or liver and kidney function; (4) Left ventricular ejection fraction < 40%, impaired cardiac function; (5) Clinically significant active bacterial, viral, fungal, parasitic or prion-associated infections; (6) Any conditions that make it impossible to perform an MRI study (including allergies to anesthetics or contrast agents); (7) Any clinically significant cardiovascular or pulmonary disease, or any other disease or condition that is contraindicated by any other research procedure.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Neurological function score (NFS);MRI score; | — |
Secondary
| Measure | Time frame |
|---|---|
| VLFCA; | — |
Countries
China
Contacts
Public ContactLiping Zou
The First Medical Center of PLA General Hospital
Outcome results
None listed