Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy
Conditions
Interventions
Experimental Group:ALT001 will be administered via intravenous (IV) infusion at a dose of 130 µg per day. The lyophilized powder will be reconstituted in 100 ml of 0.9% sodium chloride injection.
(1)
Sponsors
Beijing Tiantan Hospital, Capital Medical University
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. 18 years old=1 recent subcortical infarct >=1 cavity >=1 microhemorrhagic foci in the basal ganglia or brainstem An enlarged perivascular space is visible in the center of the basal ganglia or semi-ovale Brain atrophy 4. Presence of cognitive dysfunction (MoCA score: illiteracy <=13 points, years of education of 1~6 years<=19 points, years of education of 7 years and above<=24 points); 5. Independence in daily life (mRS < 2 points); 6. The patient or his legal representative clearly understands, voluntarily participates in the study and signs the informed consent form; 7. Subjects (including male subjects) are willing to have no birth plan and voluntarily take effective contraceptive measures and no sperm donation or egg donation plan during the entire study period and for 3 months after the end of the study. Note: The total score of Fazekas is 6 points, which is the sum of the Fazekas score of subcortical and periventricular white matter lesions.
Exclusion criteria
Exclusion criteria: 1. Combined with other active and major neurological diseases (such as intracranial infection, multiple sclerosis, intracranial tumors, brain trauma, etc.); 2. History of infectious diseases (positive or positive test for HIV, positive or positive test for hepatitis C virus antibody, positive for hepatitis B surface antigen and/or positive serum HBV DNA or serum HBV DNA>2×10^8 IU/ml); 3. Combined with severe hepatic insufficiency, renal insufficiency or severe cardiac insufficiency (of which severe hepatic insufficiency refers to ALT value =2.0 times the upper limit of normal value or AST value =2.0 times the upper limit of normal value; severe renal insufficiency refers to serum creatinine (CRE) =1.5 times the upper limit of normal value or glomerular filtration rate eGFR<40mL/min/1.73m^2); severe cardiac insufficiency refers to NYHA score grade 3-4); 4. History of coagulation disorders or systemic bleeding, history of thrombocytopenia or neutropenia, history of drug-induced hematology or abnormal liver function, low white blood cells (<2×10^9/L) or platelet count (<100×10^9/L) 5. Suffering from serious organic diseases, such as malignant tumors, etc., with an expected survival time of < 1 year; 6. History of alcohol and drug abuse; 7. Other mental illnesses diagnosed according to DSM-V diagnostic criteria, or obvious suicidal intent; 8. Patients who are pregnant, lactating, or have the possibility of pregnancy and plan to become pregnant; 9. Patients who participate in other clinical trials or apply other biologics, drugs or devices under investigation; 10. Known history of allergy to biological agents such as proteins and cell products; 11. Patients who have received any vaccination within 28 days; 12. MRI examination is contraindicated (such as claustrophobia); 13. Unable to cooperate with the completion of follow-up for other reasons.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of adverse events (AEs) and serious adverse events (SAEs);Vital signs and physical examination;Laboratory tests and auxiliary examinations data collection;Assessment of gait function;Assessment of affective status;Assessment of sleep status;Assessment of migraine;Incidence of new-onset stroke events;Brain structural and functional imaging;Blood biomarkers of neuronal injury;Quality of life; | — |
Secondary
| Measure | Time frame |
|---|---|
| Assessment of cognitive function; | — |
Countries
China
Contacts
Public ContactYilong Wang
Beijing Tiantan Hospital, Capital Medical University
Outcome results
None listed