Stiripentol in severe myoclonic epilepsy in infancy: a randomised placebo-controlled syndrome-dedicated trial. STICLO study group

Lancet. 2000 Nov 11;356(9242):1638-42. doi: 10.1016/s0140-6736(00)03157-3.


Background: Stiripentol is an inhibitor of cytochrome P450 that showed antiepileptic efficacy in severe myoclonic epilepsy in infancy (SMEI) in association with clobazam and valproate in an open study. To confirm these results, 41 children with SMEI were included in a randomised, placebo-controlled, add-on trial.

Methods: After a baseline period of 1 month, placebo (n=20) or stiripentol (n=21) was added to valproate and clobazam during a double-blind period of 2 months. Patients then received stiripentol in an open fashion. Responders were defined as having more than 50% reduction in the frequency of clonic (or tonic-clonic) seizures during the second month of the double-blind period compared with baseline.

Findings: 15 (71%) patients were responders on stiripentol (including nine free of clonic or tonic-clonic seizures), whereas there was only one (5%) on placebo (none were seizure free; stiripentol 95% CI 52.1-90.7 vs placebo 0-14.6). The 95% CI of the difference was 42.2-85.7. Percentage of change from baseline was higher on stiripentol (-69%) than on placebo (+7%), p<0.0001. 21 patients on stiripentol had moderate side-effects (drowsiness, loss of appetite) compared with eight on placebo, but side-effects disappeared when the dose of comedication was decreased in 12 of the 21 cases.

Interpretation: This controlled trial shows the antiepileptic efficacy, of add-on stiripentol in children with SMEI. The results also provide good reason to focus studies on a specific epilepsy syndrome-a small sample of patients is sufficient to show the efficacy that might have been missed in a heterogeneous population.

Publication types

  • Clinical Trial
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Anti-Anxiety Agents / administration & dosage
  • Anti-Anxiety Agents / therapeutic use
  • Anticonvulsants / administration & dosage
  • Anticonvulsants / adverse effects
  • Anticonvulsants / therapeutic use*
  • Benzodiazepines*
  • Child, Preschool
  • Clobazam
  • Dioxolanes / administration & dosage
  • Dioxolanes / adverse effects
  • Dioxolanes / therapeutic use*
  • Double-Blind Method
  • Drug Therapy, Combination
  • Epilepsies, Myoclonic / drug therapy*
  • Female
  • Humans
  • Infant
  • Male
  • Syndrome
  • Valproic Acid / administration & dosage
  • Valproic Acid / therapeutic use


  • Anti-Anxiety Agents
  • Anticonvulsants
  • Dioxolanes
  • Benzodiazepines
  • Clobazam
  • Valproic Acid
  • stiripentol