Use of the antiepileptic drug stiripentol for the treatment of severe myoclonic epilepsy in infancy (SMEI)

JRCT ID: jRCT1091220014

Registered date:06/11/2007

Basic Information

• Recruitment status: COMPLETED
• Health condition(s) or Problem(s) studied: Dravet syndrome (severe myoclonic epilepsy in infancy: SMEI)
• Date of first enrollment: 01/10/2007
• Target sample size: 10
• Countries of recruitment: Japan
• Study type: Interventional

Intervention(s)

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 - 100
50mg/kg/day
Dosing frequency / Frequency of use:BID
2 or 3 times per day
Planned duration of intervention:
Intended dose regimen:Stiripentol will be started at 50 mg/kg (or 1000 mg for patients weighing 20 kg or more) and will be titrated up to 100 mg/kg (maximum of 4.0 g).
detailes of teratment arms:
Comparative intervention name:
Dose form / Japanese Medical Device Nomenclature:
Route of administration / Site of application:
Dose per administration:
Dosing frequency / Frequency of use:
Planned duration of intervention:
Intended dose regimen:

Outcome(s)

• Primary Outcome: 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 Outcome

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.

Key inclusion & exclusion criteria

• Age minimum: >= 1age old
• Age maximum: Not applicable
• Gender: Both

Include 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.

Exclude 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.

Related Information

Primary Sponsor

Yushi Inoue
National Epilepsy Center, Shizuoka Institute of Epilepsy and Neurological Disorders

Secondary Sponsor

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 Organization Nagasaki Medical Center

Katsuyuki Fukushima
National Hospital Organization Yakumo Hospital

• Source(s) of Monetary Support: Ministry of Health, Labour and Welfare
• Secondary ID(s): Nill Known,JMA-IIA00014