Infantile epileptic spasms syndrome
Conditions
Interventions
Treatment group:GD002 exosomes nasal instillation. The concentration of the exosomes under study is 4×10^9 particles/mL. They are administered via the bilateral nasal instillation route, twice daily,
Sponsors
The First Medical Centre, Chinese PLA General Hospital
Eligibility
Sex/Gender
All
Age
0.5 Years to 8 Years
Inclusion criteria
Inclusion criteria: 1. Meet the diagnostic criteria for IESS in the ILAE:(1) Epileptic seizures: flexor, extensor, or mixed epileptic spasms, usually occurring in clusters;(2)Electroencephalogram (EEG): interictal period shows hypsarrhythmia, multifocal, or focal epileptiform discharges;(3)Age of onset: 1–24 months old; 2. Aged 6 months to 8 years, regardless of gender; 3. Have a history of using ACTH and vigabatrin, but spasms remain uncontrolled; 4. All antiseizure medications (ASMs) used before enrollment have reached the target dose; 5. Have at least one seizure per day; 6. Clinical study participants and/or their guardians can fully understand the content of the informed consent form (ICF) for this study and voluntarily sign the ICF.
Exclusion criteria
Exclusion criteria: 1. IESS caused by a clear and treatable cause (e.g., Glucose Transporter Type 1 Deficiency Syndrome [GLUT1-DS], Methylmalonic Acidemia [MMA], and other metabolic diseases); 2. Epilepsy with structural etiologies (e.g., sequelae of hypoxic-ischemic encephalopathy, sequelae of neonatal hypoglycemic encephalopathy, sequelae of various types of encephalitis, cerebral softening, cortical dysplasia, etc.); 3. Suffering from other uncontrolled diseases that may interfere with the study results, including but not limited to infections, diabetes mellitus, cardiovascular and cerebrovascular diseases, etc.; 4. No known history of immunodeficiency diseases; 5. Previous history of severe allergic reactions or allergies to blood products; 6. Patients with structural abnormalities or lesions of the nasal cavity; 7. Patients with cerebrospinal fluid (CSF) rhinorrhea; 8. Patients whose guardians fail to master the correct nasal drop administration method proficiently despite standardized training; 9. Adjustments to the type or dosage of antiseizure medications (ASMs) made within 2 weeks prior to enrollment; 10. Inability to maintain the frozen state of the nasal drops after they are delivered to the patient’s residence via routine cold chain transportation; 11. Other conditions deemed unsuitable for enrollment by the investigators.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Clinical seizure; | — |
Secondary
| Measure | Time frame |
|---|---|
| Video electroencephalogram and BASED score;Recurrence rate;Vineland Adaptive Behavior Scales;Gesell Developmental Schedules; | — |
Countries
China
Contacts
Public ContactYang Guang
The First Medical Centre, Chinese PLA General Hospital
Outcome results
None listed