Dravet syndrome (severe myoclonic epilepsy in infancy: SMEI)
Conditions
Interventions
Intervention type:DRUG
Name of intervention:Stiripentol
Dose form / Japanese Medical Device Nomenclature:FINE GRANULES
Route of administration / Site of application:ORAL
Dose per administration:
50 -
Sponsors
Yushi Inoue
National Epilepsy Center, Shizuoka Institute of Epilepsy and Neurological Disorders
Shunya Ikeda
Department of Pharmaceutical Sciences, International University of Health and Welfare
Yoko Ohtsuka
Okayama University, Graduate School of Medicine, Dentistry and Pharmaceutical Sciences
Hirokazu Oguni
Department of Pediatrics, Tokyo Women's Medical University
Yukitoshi Takahashi
National Epilepsy Center, Shizuoka Institute of Epilepsy and Neurological Disorders
Jun Tohyama
National Hospital Organization Nishi-Niigata Chuo National Hospital
Hiroshi Baba
National Hospital Organiz
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Clinically and electroencephalographically confirmed diagnosis of SMEI including borderline SMEI according to the classification of the International League against Epilepsy (ILAE, 1989). 2. Aged 1 year or older (at the time of consent). 3. Inpatients or outpatients 4. Male or female 5. Seizure frequency: - the parents/guardians of the patient are able to provide an accurate count of seizures (generalized or unilateral, tonic or clonic) together with their approximate duration. - having 4 or more times of seizures of any type described above during 4 weeks prior to stiripentol treatment. 6. Currently treated with AED(s) (up to 3 AEDs, excluding rescue drugs). 7. Are on stable AED regimens during 4 weeks prior to stiripentol treatment. 8. A written consent from the parents/guardians of the patient. 9. With or without gene mutations (including SCN1A), but prior testing is recommended.
Exclusion criteria
Exclusion criteria: 1. Liver disease or GOT/GPT of more than 3 times the upper normal limits. 2. Kidney disease or a creatinine level of more than 1.5 mg/dL. 3. WBC count < 2500 or platelet count < 10.0 x 10^4 /mcL, unless considered medically acceptable. 4. History of drug hypersensitivity. 5. Use of prohibited concomitant medications. 6. Pregnant women. 7. Use of another study drug or unapproved drug. 8. For any reason is judged by the investigator to be inappropriate for study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Improvement in seizure control, including percent reduction in seizure frequency and duration, during the beginning phase of stiripentol treatment (evaluation period 1) compared with baseline. | — |
Secondary
| Measure | Time frame |
|---|---|
| Efficacy: Improvement in seizure control, including percent reduction in seizure frequency and duration, during the second fixed dose treatment period (evaluation period 2) compared with baseline. Safety: (i) incidence and outcomes of adverse events; (ii) laboratory data (hematology and biochemistry); and (iii) Cytochrome P450 genotyping test. | — |
Countries
Japan
Contacts
Public ContactYushi Inoue / Yukitoshi Takahashi
National Epilepsy Center, Shizuoka Institute of Epilepsy and Neurological Disorders
Outcome results
None listed