X-linked adrenal leukodystrophy
Conditions
Interventions
Test group:GC311 treatment
Sponsors
The Seventh Medical Center of the PLA General Hospital
Eligibility
Sex/Gender
All
Age
3 Years to 8 Years
Inclusion criteria
Inclusion criteria: Participants must meet all of the following conditions in order to participate in this study: (1) 3 years old <= age <= 8 years old, male; (2) Clearly diagnose CALD through clinical phenotype and genetic testing; (3) Neurological functional score (NFS) <= 1 point; (4) Head MRI Loes score ranging from 0.50 to 9.50 (inclusive); (5) The patient or the patient's guardian is able to understand and willing to comply with the requirements and procedures of the research protocol, voluntarily participate and sign an informed consent form.
Exclusion criteria
Exclusion criteria: (1) The condition progresses rapidly and may even be in the late stages of disease development; (2) Has undergone hematopoietic stem cell transplantation, is currently undergoing other clinical trials, or has previously used other gene therapy products; (3) The titer of neutralizing antibodies against AAV9 in the screening period serum is >= 1:100; (4) Abnormal blood system, manifested as: absolute neutrophil count (ANC) in peripheral blood= 3 times the upper limit of normal (ULN); Total bilirubin (TBil) >= 1.5 times the upper limit of normal (ULN), etc; (6) Left ventricular ejection fraction<40%, impaired cardiac function; (7) Renal dysfunction (measured or calculated creatinine clearance rate<50 mL/min); (8) Infections associated with clinically significant active bacteria, viruses, fungi, parasites, or prions; (9) Any clinically significant cardiovascular or pulmonary disease, or any other disease or condition that is contraindicated by any other research procedure; (10) Those who have contraindications to the use of glucocorticoid, such as severe hypertension, diabetes, systemic fungal infection, glaucoma, osteoporosis, gastric ulcer, tuberculosis, etc; (11) Any conditions that prevent it from undergoing enhanced MRI studies (including allergies to anesthetics or contrast agents); (12) Individuals with contraindications for lateral ventricular injection therapy; (13) Evaluated patients with medical diseases that affect the interpretation of the research results or the safety of the subjects, including but not limited to organ dysfunction, acute infectious diseases, primary/acquired immunodeficiency disease, serious cardiovascular and cerebrovascular diseases, digestive tract diseases, diabetes, known epilepsy, meningitis, convulsions or convulsions, or family history of psychosis; Patients with cerebrospinal fluid circulation disorders, etc; (14) Human immunodeficiency virus (HIV) antibody positive, or hepatitis B surface antigen positive, or hepatitis C antibody positive, or treponema pallidum antibody positive; (15) Those who receive the vaccine with an interval of less than 2 weeks between administration; Individuals who are deemed unsuitable by other researchers to participate in this study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Adverse event incidence rate; | — |
Secondary
| Measure | Time frame |
|---|---|
| Survival without major functional impairment;NFS and Loes ratings;Copy number of target gene;Serum levels of anti-AAV9 and anti-ABCD1 protein antibodies; | — |
Countries
China
Contacts
Public ContactZhichun Feng
The Seventh Medical Center of the PLA General Hospital
Outcome results
None listed