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

Not Applicable

Completed

• Conditions: Dravet syndrome (severe myoclonic epilepsy in infancy: SMEI)

• Registration Number: JPRN-jRCT1091220014
• Lead Sponsor: Yushi InoueNational Epilepsy Center, Shizuoka Institute of Epilepsy and Neurological Disorders

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2007-11-06
• Study Completion: 2012-11-30
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 10

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

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.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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 Measures

Name	Time	Method
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.<br>Safety: (i) incidence and outcomes of adverse events; (ii) laboratory data (hematology and biochemistry); and (iii) Cytochrome P450 genotyping test.