Drug-refractory focal epilepsy
Conditions
Interventions
Treatment Group:GA002 intracranial drug injection
Sponsors
Sanbo Brain Hospital Capital Medical University
Eligibility
Sex/Gender
All
Age
18 Years to 50 Years
Inclusion criteria
Inclusion criteria: 1. Age 18-50 years with signed consent on an Informed Consent Form (ICF) approved by an Ethical Review Board (IRB)/Independent Ethics Committee (IEC)/Human Clinical Research Ethics Committee (HREC); 2. Definite diagnosis of refractory focal epilepsy, number of seizures: an average of >=4 seizures per month in the past 3 months, and the interval between any two seizures should be < 28 days; 3. Unable to effectively control seizures after 2 or more years of standardized treatment with 2 or more antiepileptic drugs; 4. As judged by the investigator, the patient's condition has not changed significantly in the past 3 months under the condition of unchanged antiepileptic drug regimen; 5. Have been stably taking the current antiepileptic drugs =for 3 months until the baseline period, and are expected to not change the type and dose of the current drugs before the end of the effectiveness observation period of this trial; 6. Subject laboratory test values meet the following criteria: normal or abnormal white blood cell count and neutrophil count are not clinically significant; Hemoglobin (HGB): greater than 120g/L; Coagulation function index: platelet count (PLT): greater than 100×10^9/L; International normalized ratio (INR): 1.0-1.2, fibrinogen (FIB): greater than 2.0 g/L; Liver and kidney function indexes: alanine aminotransferase (ALT): 5-40 U/L; Aspartate aminotransferase (AST): 10-40 U/L ; Blood creatinine (Scr): 0.6-1.2 mg/dL for males, 0.5-1.1mg/dL for females; Urea nitrogen (BUN): 2.9-7.5 mmol/L; Blood adeno-associated virus antibody titer less than 1:1200; 7. Male or female subjects of childbearing potential agree to use an effective method of contraception (oral contraceptives are prohibited) for at least 6 months from the time of signing the informed consent form to the time of stopping clozapine.
Exclusion criteria
Exclusion criteria: 1. Accompanied by degenerative diseases or malignant tumors of the central nervous system; 2. The seizure interval fluctuates too much as judged by the investigator; 3. Patients with secondary epilepsy caused by intracranial malignant tumors, neurodegenerative diseases, traumatic brain injuries, cerebral hemorrhage or cerebral infarction, infectious diseases, cerebrovascular diseases or other organic lesions; 4. Accompanied by severe psychiatric illness; 5. There are contraindications to treatment and surgery as judged by the investigator; 6. Subjects are unable or unsuitable to complete the non-invasive or invasive evaluation examinations related to the trial; 7. History of previous intracranial surgery; 8. Patients who have previous contraindications to the drug during the trial; 9. Subject has received any previous gene or cell therapy; 10. Those who have received any other drug clinical trials within 3 months before the date of signing the informed consent form, except for those who have received non-intervention clinical trials; Subjects who have participated in clinical trials of devices can be assessed by the investigator for inclusion; 11. Patients with severe liver and kidney diseases, severe heart diseases, severe pulmonary dysfunction, hypotension, glaucoma and other serious underlying diseases; 12. Patients with bone marrow aplasia; 13. Patients who are taking drugs that cause agranulocytosis or myelosuppressive effects; 14. Patients with agranulocytosis or severe neutropenia caused by previous use of clozapine; 15. Those who are positive for human immunodeficiency virus antibody or Treponema pallidum antibody; 16. Overweight patients, BMI>=30kg/m^2; 17. Those who have digestive system diseases that affect the absorption, metabolism and excretion of drugs, or major digestive system surgery or dysphagia; 18. The patient has a history of alcohol/drug abuse in the past 2 years; 19. Females with positive pregnancy test and lactation; 20. The investigator believes that the subject has other conditions that may interfere with the efficacy and safety evaluation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| AE/SAE incidence rate and severity; | — |
Secondary
| Measure | Time frame |
|---|---|
| The changes in MEG findings during the observation period compared to the baseline period;The severity of seizures during the efficacy observation period;Seizure frequency;Severity of seizure onset;The percentage change in the number of scalp - electrode EEG epileptiform discharges during the efficacy observation period compared to the baseline period;Indicators of drug metabolism;The titer of neutralizing antibodies in peripheral blood; | — |
Countries
China
Contacts
Public ContactLuan Guoming
Sanbo Brain Hospital Capital Medical University
Outcome results
None listed