AAN 2025 | A modern trial design to improve safety for patients with epilepsy

Traditional epilepsy studies are placebo-controlled, double-blind studies. And they do an observation phase and subjects are in the observation phase for a while, for a couple of months, a few months, and see how many seizures they’re having per month. And then they go into the double-blind phase, and again, it’s also a few months, and we see how many seizures they have over that period of time and basically are comparing them. The problem is there was a study that was out just a few years ago that showed that subjects who were in anti-seizure medication trials, and this was over 100 trials they looked at, subjects who continued to have, who were in a placebo or in a treatment that wasn’t working, had a much higher, I think it was about six times higher rate of SUDEP, sudden unexpected death in epilepsy in those trials. And we know that the type of seizure most associated with it, the highest risk is the generalized tonic-clonic seizures. So to have someone in a study like that with a typical observation period and then a typical double-blind period, they’re at a much higher risk over that period of time if they’re on a placebo or treatment that’s not working. So the design of this trial is kind of a new idea in which the observation period was really retrospective, and so we’re not observing them prospectively, and they’re not having more seizures. It’s just a retrospective observation phase. And then the double-blind phase is actually the time to event. And so after their second seizure, after a month, then they are able to go straight into open-label. So as opposed to having to stay in the double-blind period for 12 weeks or however many weeks it is, if they’re still having seizures after a month, they go straight to open-label. So less seizures during the observation phase, less seizures during the double-blind phase. And then we basically compare the subjects who end up being on placebo versus subjects who end up on treatment and see who had a longer time period between when they started treatment and their second seizure. And so we feel like this is a much safer study. It really decreases the risk of SUDEP. And I really think it’s something that probably studies like this are probably going to be run in the future too, just because of the safety aspects.

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