Beginning enrollment in 2022, the NAUTILUS pivotal study will be the first in the United States to evaluate brain-responsive neuromodulation in patients with idiopathic generalized epilepsy.
According to a recent announcement, the FDA has granted an investigational device exemption (IDE) to NeuroPace for its responsive neurostimulation (RNS) system to be evaluated in patients with drug-resistant idiopathic generalized epilepsy (IGE).1
The news comes just a few months after the company announced it had been granted breakthrough device designation status from the FDA for the treatment of IGE. Dubbed NAUTILUS, this new, prospective, single-blind, multicenter, randomized pivotal study will be the first to evaluate the effects of brain-responsive neuromodulation in this patient population. Enrollment is expected to begin in 2022. NeuroPace has yet to announce further details of the study, including primary and secondary end points.
"We are pleased with FDA’s decision to grant IDE approval for the pivotal study, allowing us to evaluate the safety and effectiveness of the RNS System in patients who are living with drug-resistant, idiopathic generalized epilepsy," said Martha Morrell, MD, chief medical officer, NeuroPace, said in a statement.1 "We look forward to working closely with the study investigators to evaluate a new treatment option that could potentially improve quality of life for these individuals in a meaningful way."
The RNS System was originally approved in 2013 for the treatment of medical refractory epilepsy and remains the only such of its kind approved for this patient population. It acts on closed-looped technology that monitors and responds to a patient’s unique brain patterns to deliver therapy in real time prior to symptom onset.
Recently, the company was awarded a grant from the National Institutes of Health to fund an IDE study to evaluate the system in patients with Lennox-Gastaut syndrome (LGS), another rare form of epilepsy.2 This 5-year, $9.3 million grant will not only explore the neuromodulation technology as a new therapy for those with LGS, but individualized therapy may be optimized as a result of continual monitoring and recording of brain activity through the RNS System.
Since its approval, there evidence has been collected on its long-term impact. One of which was a 9-year, multicenter study that included 230 patients across 34 epilepsy centers with nearly 1900 patient-implant years of follow-up, considered the largest prospective neuromodulation trial in the field of epilepsy. At 9 years, the median percent reduction in seizure frequency was 75% (P <.0001; Wilcoxon Signed Rank), responder rate was 73%, and 35% of patients had a ≥90% reduction in seizure frequency.3
Furthermore, 18.4% (47 of 256) of patients experienced ≥1 year of seizure freedom with 62% (29 of 47) seizure-free at last follow-up and an average seizure-free period of 3.2 years (range, 1.04-9.6 years). The research, conducted by Morrell and colleagues, showed that enduring improvements were seen in overall QoL and cognitive function (P <.05).
A few months ago, the company also announced their nSight Platform, which works alongside the RNS System to track seizure-related outcomes for those with drug-resistant epilepsy. Listen below for commentary from Morrell, as she explained the impact the platform has and how it proves an additional source of information to eventually improve patient outcomes.