SCN1A-related epilepsy
Conditions
Interventions
Experimental group:iPSC derived exosomes
Experimental group :iPSC derived exosomes
control:none
Sponsors
Senior department of Pediatrics, Chinese PLA General Hospital; Department of Pediatrics, the First Medical Center of Chinese PLA General Hospital
Eligibility
Sex/Gender
All
Age
2 Years to 18 Years
Inclusion criteria
Inclusion criteria: Part 1 Inclusion Criteria: The following inclusion criteria must be met to be included in the study 1. Both male and female, age 2-18 years old; 2. A clear diagnosis of Dravet syndrome, and current antiepileptic drugs cannot completely control the disease; 3. clearly contain a pathogenic mutation of the SCN1A gene; 4. During the 4-week baseline observation period, the patient must experience at least 4 seizures; 5. The patient must be taking one or more antiepileptic drugs whose doses have been stable for at least 4 weeks; 6. All drugs or interventions related to epilepsy are stable during the screening period. In order to avoid the impact of adjustment, no adjustment of antiepileptic drugs or interventions will be made during the evaluation period; 7. The legal guardian of the patient must be willing and able to cooperate with the test to complete relevant inspections and tests; 8. The legal guardian of the patient agrees to sign a written informed consent. Part II Inclusion Criteria: The following inclusion criteria must be met to be included in the study 1. Both male and female, age 2-18 years old; 2. clearly contain a pathogenic mutation of the SCN1A gene; 3. During the 4-week baseline observation period, the patient must experience at least 4 seizures; 4. One or more antiepileptic drugs must be taken, and the dose has been stabilized for at least 4 weeks; 5. All drugs or interventions related to epilepsy have stabilized during the screening period. In order to avoid the impact of adjustment, no adjustment of antiepileptic drugs or interventions will be made during the evaluation period; 6. The legal guardian of the patient must be willing and able to cooperate with the test to complete relevant inspections and tests; 7. The legal guardian of the patient agrees to sign a written informed consent.
Exclusion criteria
Exclusion criteria: 1. The patient suffers from a clinically significantly unstable disease other than epilepsy; 2. Patients developed clinically significant diseases other than epilepsy within 4 weeks before screening or randomization; 3. The patient's cooperation or compliance is poor, and it cannot be guaranteed that the program will be carried out according to the requirements of the program; 4. Currently participating in other clinical studies or receiving other drug treatment; 5. Those who are allergic to drug ingredients; 6. According to the evaluation of the investigator, patients who are considered unsuitable for enrollment due to various other reasons; 7. There are plans to travel to other countries during the research period; 8. Women who are pregnant, breastfeeding or planning to become pregnant during the study or within the next three months.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Seizure frequency;adverse event; | — |
Secondary
| Measure | Time frame |
|---|---|
| EEG;blood routine examination;cytokine; | — |
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