Stiripentol in childhood partial epilepsy: randomized placebo-controlled trial with enrichment and withdrawal design.

Abstract

Stiripentol, a new antiepileptic drug inhibiting cytochrome P450-enzymes, suggested some efficacy when combined with carbamazepine in an open trial in refractory partial epilepsy of childhood. Our objective was to test these results in a placebo-controlled trial. To limit the number of patients included, we used an enrichment and withdrawal design. Among the 67 children entered in a 4-month open add-on stiripentol study following a 1-month single-blind placebo baseline, the 32 responders were randomized for 2 months either to continue stiripentol (n = 17) or to withdraw to placebo (n = 15). If seizures increased by at least 50% after randomization compared with baseline, the patients dropped out (primary end point): there were six patients on stiripentol and eight patients on placebo (not significant). However, a decrease in seizure frequency compared with baseline (secondary end point) was greater on stiripentol (-75%) than on placebo (-22%) (P < .025). Twelve patients experienced at least one adverse event on stiripentol (71%) compared with four patients on placebo (27%); none were reported as severe. The combination of stiripentol and carbamazepine proved to reduce seizure frequency in children with refractory partial epilepsy, although it failed to show a significant impact according to the escape criteria selected as the primary end point in the present study, for ethical reasons.

• Authors: Catherine Chiron, Sylvie Tonnelier, Elisabeth Rey, Marie-Lucie Brunet, Agnes Tran, Philippe d'Athis, Jean Vincent, Olivier Dulac, Gerard Pons
• Journal: Journal of child neurology (J Child Neurol) Vol. 21 Issue 6 Pg. 496-502 (Jun 2006) ISSN: 0883-0738 [Print] United States
• PMID: 16948934 (Publication Type: Journal Article, Randomized Controlled Trial)

Chemical References

• Anticonvulsants
• Dioxolanes
• Carbamazepine
• stiripentol

Topics

• Adolescent
• Anticonvulsants (administration & dosage)
• Carbamazepine (administration & dosage)
• Child
• Child, Preschool
• Dioxolanes (administration & dosage)
• Dose-Response Relationship, Drug
• Double-Blind Method
• Drug Administration Schedule
• Drug Therapy, Combination
• Epilepsies, Partial (drug therapy)
• Female
• Humans
• Male
• Treatment Outcome
• Withholding Treatment