A Phase 2/3 Multicenter, Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy, Safety and Tolerability of BHV-7000 in Subjects with Refractory Focal Onset Epilepsy (RISE-3)

Research summary

This is a Phase 3, multi-center, randomized, double-blind, placebo controlled 3-arm study designed to assess the efficacy and safety of BHV7000 in participants with refractory focal epilepsy who are stable on at least one ASM. Participants must be considered uncontrolled on their current ASM regimen, defined as the occurrence of a minimum of an average of 6 seizures during an 8-week OP and no more than 21 consecutive days without a focal seizure in the OP. The study is planned to consist of a prospective Screening and OP of 8 weeks. The DBP of 8 weeks/12 weeks includes study visits at Baseline, Week 2, Week 4, Week 8, (and Week 12). All subjects who complete the study will have the option to enroll in a long-term, open-label safety study. Subjects who do not opt in to the open-label safety study should enter a follow-up phase of this study which includes a study visit 2 weeks after last dose of study drug. To assess eligibility, all potential subjects will have a Screening visit and participate in the 8-week OP. During the OP subjects will continue taking their previously prescribed ASMs and report each occurrence and the characteristics of seizures in an electronic diary. Subjects will be assessed for study eligibility at the Baseline visit. Approximately 453 eligible subjects will be randomized in a 1:1:1 ratio and assigned BHV-7000 high dose mg, BHV-7000 medium dose mg, or matching placebo at the Baseline visit. Subjects will take blinded study drug every day. Subjects who complete the DBP and who tolerated study medication, may be eligible to enroll in a separate open-label study and receive the same dose level assigned in the DBP in the open-label study for one year. Subjects who were assigned placebo in the DBP phase will receive BHV-7000 high mg in the open-label study. Subjects who do not continue in the open-label study will enter the post-dose follow-up phase for 2 weeks, with a study visit at follow-up week 2. All subjects will participate in sparse pharmacokinetic (PK) sampling at all study visits; a sub-group of approximately 8-12 subjects in each study arm between 12 and < 18 years, may participate in serial PK sampling at the Week 2 visit.

Principal Investigator

Arjune Sen

Contact us

Email: dendron@ouh.nhs.uk

IRAS number

1009549