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drugset / Trial / NCT04940624

A Study of Soticlestat as an Add-on Therapy in Children and Young Adults With Dravet Syndrome

NCT04940624 ↗

Phase 3 Completed 144 enrolled Takeda
RandomizedParallel-groupQuadruple-blindTreatment

Summary

The main aim of the study is to learn if soticlestat, when given as an add-on therapy, reduces the number of convulsive seizures in children and young adults with DS. Participants will receive their standard antiseizure therapy, plus either a tablet of soticlestat or placebo for 16 weeks. A placebo looks just like soticlestat but will not have any medicine in it. Participants may continue treatment in an extension study, based on the extension study's entry criteria. Those that want to stop treatment will have a gradual dose reduction during 1 week and then be followed up for 2 weeks.

Timeline

Start
2021-10-28
Primary completion
2024-04-11
Completion
2024-04-11

Outcome

Missed primary endpoint

registry analysis (superiority test); Placebo vs Soticlestat; p =0.061 NCT04940624 ↗

registry analysis (superiority test); Placebo vs Soticlestat; p =0.089 NCT04940624 ↗

release “SKYLINE Study in Dravet Syndrome Narrowly Missed its Primary Endpoint of Reduction in Convulsive Seizure Frequency” takeda.com ↗

Drugs

EvaluationDrugModalityDoseRoute
Subject Soticlestat Small molecule 40 mg —
Subject Soticlestat Small molecule 200 mg —
Subject Soticlestat Small molecule 300 mg —

Indications

Dravet syndrome