Epilepsy affects about 3.4 million people in the U.S., causing recurrent seizures that vary widely in type and severity. About 70% of people gain seizure control with medications, but a third continue having seizures despite trying multiple drugs. Treatment options now include more than 30 anti-seizure medications, brain surgery, nerve stimulation devices, and dietary therapies.
What's actually going on in research
Trials are testing new anti-seizure medications with different mechanisms, including drugs that modulate potassium channels and GABA receptors. Researchers are studying gene therapies for genetic epilepsies like Dravet syndrome and CDKL5 deficiency, brain-responsive neurostimulation devices that detect and stop seizures before they spread, and precision approaches based on specific genetic mutations or seizure origins.
Gene therapy
Several trials are testing gene therapies for rare genetic epilepsies where a single gene defect drives seizures. Early results in Dravet syndrome and other conditions show promise in reducing seizure frequency.
Responsive neurostimulation
Devices implanted in the brain can now detect seizure activity and deliver targeted electrical pulses to stop seizures before they start. Newer systems are being tested for more seizure types and locations.
Precision dosing
Researchers are studying how genetic variants affect how people metabolize anti-seizure drugs. This could help doctors choose the right drug and dose faster, avoiding years of trial and error.
What to know before you search
Eligibility typically depends on seizure frequency, number of medications tried, seizure type, and sometimes genetic testing results or MRI findings showing where seizures start.
What types of trials are currently open
- Drug trials — Testing new anti-seizure medications, often as add-on therapy for people whose seizures aren't controlled. These trials typically track seizure frequency over several months.
- Device trials — Studies of neurostimulation devices, including responsive systems that detect and interrupt seizures, and vagus nerve stimulators that deliver regular pulses to prevent seizures.
- Gene therapy trials — Testing one-time treatments that deliver a working copy of a gene or modify gene expression in people with genetic epilepsies.
- Surgical trials — Comparing different surgical approaches for drug-resistant epilepsy, including laser ablation and traditional resection.
- Biomarker studies — Following people with epilepsy to identify patterns in EEG, imaging, or blood tests that predict treatment response or seizure risk.
Recently added Epilepsy trials
Study Designed to Evaluate the Safety, Tolerability, and PK of RAP-219 Formulated as an Long Acting Injectable.
This is a Phase 1, double-blind, placebo-controlled, 5-cohort study evaluating the pharmacokinetics (PK), safety, and tolerability of RAP-219-LAI in healthy participants aged 18 to 65 years. Assessment of up to four single-dose cohorts will characterize the PK profile of up to four ascending dose levels and evaluate dose proportionality. One multiple-dose cohort will characterize the accumulation profile of RAP-219-LAI. The study includes a Screening Period of up to 28 days (Day -28 to Day -1) for participants in all cohorts. Cohorts 1, 2, 3, and 4 will include admission on Day -1, a single dosing day (Day 1), a 28-day inpatient and outpatient Treatment Period (Day 1 to Day 28), and a 28-day Follow-up Period (Day 29 to Day 56). Cohort 5 will include admission on Day -1 and Day 28, two dosing days (Day 1 and Day 29), a 56-day inpatient and outpatient Treatment Period (Day 1 to Day 56), and a 28-day Follow-up Period (Day 57 to Day 84). Up to 60 participants are planned to be enrolled in this study, with 12 participants per cohort randomized in a 3:1 ratio to receive RAP-219-LAI or placebo.
Identification of Biomarkers for Vagus Nerve Stimulation in Patients With Refractory Epilepsy
A VNS longitudinal study focused on pre and post-VNS implantation in order to predict the VNS response and to understand better mechanisms of VNS using some connectivity and network measures.
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