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A Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Clinical Activity of ABS-1230 in Pediatric and Young Adult Participants With KCNT1-related Epilepsy
What is epilepsy?
A seizure is a sudden surge of abnormal electrical activity in the brain. Epilepsy is the condition of having repeated, unprovoked seizures, and doctors usually diagnose it after two seizures, or after one when tests show a high chance of more. Seizures come in many forms. Focal seizures start in one area of the brain and may cause a strange feeling, a blank stare, or jerking in one limb. Generalized seizures involve both sides at once and include the convulsive tonic clonic type most people picture, as well as brief absence seizures that look like daydreaming. About 3.4 million Americans live with active epilepsy, including around 470,000 children, and roughly 50 million people worldwide. It can start at any age, but new cases peak in early childhood and after 60. Epilepsy is not contagious, not a mental illness, and in most cases not inherited in a simple way.
Medicines control seizures in about two thirds of people, often completely. But about 1 in 3 keep having seizures even after trying two or more drugs, which doctors call drug resistant epilepsy. That group carries most of the risk: people with uncontrolled seizures have higher rates of injury, depression, and a rare but real risk called SUDEP, sudden unexpected death in epilepsy, which affects about 1 in 100 people with drug resistant epilepsy each year. For them, options beyond pills include surgery to remove the seizure focus, implanted stimulators, and a strict ketogenic diet. The most promising new directions, gene therapy and cell therapy that target the seizure source directly, are in trials right now.
Types of epilepsy clinical trials
Drug trials for drug resistant epilepsy. Most new medicine trials enroll people whose seizures continue despite two or more drugs. The study medicine is added on top of what you already take, and success is measured by how much monthly seizure frequency drops, usually over 12 weeks after a baseline period where you log your seizures.
Gene and cell therapy trials. A new frontier. Instead of a daily pill that affects the whole brain, these deliver a treatment directly to the seizure focus, for example the temporal lobe. Early trials on our platform test a one time gene therapy and an injection of lab grown inhibitory neurons.
Device and neuromodulation trials. Implanted stimulators (vagus nerve, deep brain, and responsive neurostimulation) are approved options, and trials test how to tune them better. Newer studies test non invasive approaches like low intensity focused ultrasound, and wearables that forecast seizures from brain and body signals.
Diet and pediatric trials. The ketogenic diet is one of the oldest epilepsy treatments and one of the most actively studied, including in new onset childhood epilepsy and infant spasms. Children with epilepsy are underrepresented in adult drug trials, so pediatric studies matter more than their numbers suggest.
Imaging, genetics, and registry studies. These map where seizures start with advanced MRI, look for genetic causes in infants, or follow rare epilepsy syndromes over years. No treatment is promised, but this is where surgery gets more precise and where tomorrow's targeted drugs come from.
Who can participate in epilepsy clinical trials?
Epilepsy studies almost always start with your seizure type, focal or generalized, and your seizure frequency, confirmed with a seizure diary during a screening period of several weeks. Second, your treatment history: most drug trials require that you have tried at least two seizure medicines without full control, and that you stay on your current ones during the study. Third, for surgery, gene, and cell therapy trials, whether tests have pinpointed where your seizures start, since those treatments target one location. Some trials are built for specific groups: infants with genetic epilepsies, people with an implanted stimulator, or people with a first seizure. Eligibility always varies by study.
The types of epilepsy and seizures, and why they matter for trials
Epilepsy has no stage 1 to 4. Doctors classify it by where seizures start and how they spread:
- Focal seizures. Start in one area of the brain. Awareness may stay intact or fade. The most common type in adults, and the type most new drug trials enroll.
- Generalized seizures. Involve both sides of the brain from the start. Includes tonic clonic (convulsive) seizures, absence seizures, and myoclonic jerks.
- Drug resistant epilepsy. Seizures that continue after two well chosen medicines. About 1 in 3 people. This label opens the door to most surgery, device, gene, and cell therapy trials.
- Epilepsy syndromes. Specific patterns like Dravet, Lennox Gastaut, or infantile spasms, often genetic and often starting in childhood, with their own dedicated trials.
Study titles say "focal onset seizures," "drug resistant," or name a syndrome right up front. Your neurologist's classification and your monthly seizure count tell you at a glance which studies fit.
Do epilepsy trial participants get paid?
Some studies offer compensation for time and travel, and study related care is typically provided at no cost to participants. Compensation varies by trial and is always described during the informed consent process before you agree to anything.
Curious whether clinical trials pay participants? Here's how compensation actually works.
How to enroll in an epilepsy clinical trial
Enrolling in an epilepsy study through our platform takes about 5 minutes. Here is how it works:
Identify your trial. Use the search bar or filters to find epilepsy studies that match your situation. Titles usually name the seizure type ("focal onset seizures") and often "drug resistant," so you can tell quickly which ones fit.
Select your preferred location. Enter your city or state in the filter on the left. Gene therapy, cell therapy, and device trials run at specialized epilepsy centers, so those may mean travel, while diary based drug trials and wearable studies are often closer to home.
Explore study details. Click "Learn More" on any study for its goals, procedures, and eligibility, including how many prior medicines it requires, how long the seizure diary period is, and whether EEG monitoring is involved.
Complete the health profile. Click "Get started" to begin the 5-step application. Have three things ready: your seizure type as your neurologist described it, your average seizures per month, and the list of seizure medicines you have tried. Those three answers decide eligibility for most epilepsy studies. If you have had a driving restriction or use a seizure diary app, that history helps too.
Submit the application. A clinical trial coordinator reviews it and contacts you about whether you may qualify. Nothing is decided until you have gone through informed consent, and participation is voluntary at every step. Because seizures can affect memory, many people bring a family member to the consent conversation, and study teams welcome that.