Home/Rare Diseases/KCNT1-Related Epilepsy

Neurological & Neuromuscular

KCNT1-Related Epilepsy (EIMFS) Clinical Trials

Also called KCNT1 epilepsy, KCNT1+ epilepsy, EIMFS, Epilepsy of Infancy with Migrating Focal Seizures, Malignant Migrating Partial Seizures of Infancy, MMPSI, ADNFLE, Autosomal Dominant Nocturnal Frontal Lobe Epilepsy, Sleep-Related Hypermotor Epilepsy, SHE

KCNT1 is a gene on chromosome 9 that carries the instructions for a sodium-activated potassium channel called KNa1.1 (also written Slack).

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About KCNT1-Related Epilepsy

KCNT1 is a gene on chromosome 9 that carries the instructions for a sodium-activated potassium channel called KNa1.1 (also written Slack). Potassium channels act as brakes on electrical activity in neurons. The disease-causing changes in KCNT1 are gain-of-function variants, meaning the channel opens too readily and passes too much current. Counterintuitively, a channel that should quiet neurons produces severe epilepsy when overactive, most likely because the effect falls hardest on inhibitory interneurons whose job is to restrain the rest of the network.

The severe end of the spectrum is epilepsy of infancy with migrating focal seizures (EIMFS), first linked to KCNT1 in 2012. Seizures usually begin within the first weeks to months of life. On EEG they migrate, starting in one cortical region and shifting to another and across hemispheres, which is the finding that gives the syndrome its name. Autonomic features such as apnea, perioral cyanosis, and flushing are common and can be frightening for parents. Seizures typically escalate to become nearly continuous by six to nine months. Development arrests once seizures begin: many children never sit, walk, or speak. Mortality in early childhood is substantial.

The milder end is sleep-related hypermotor epilepsy (SHE), historically called autosomal dominant nocturnal frontal lobe epilepsy. Here, seizures come in clusters during sleep and involve sudden vigorous movements that can be mistaken for night terrors or other parasomnias. Seizure burden is lower, but cognitive and psychiatric difficulties are common and can be more disabling than the seizures themselves. Between these two poles sit other focal epilepsies and developmental and epileptic encephalopathies that do not fit either label cleanly, and reports have described features outside the nervous system as well.

Diagnosis is made by genetic testing, usually an epilepsy gene panel, exome sequencing, or genome sequencing. Because the specific variant carries implications for both prognosis and emerging targeted treatment, confirming the genetic diagnosis matters even when the clinical picture already looks like EIMFS.

Common Symptoms of KCNT1-Related Epilepsy

Recognizing the signs of KCNT1-Related Epilepsy early can lead to faster diagnosis and better outcomes. Symptoms may vary in severity from person to person. If you or a loved one are experiencing any of the following, consider speaking with a specialist.

  • Focal seizures beginning in the first six months of life, often within the first weeks
  • Seizures that migrate from one brain region to another and from one side to the other, visible on EEG
  • Seizures that become nearly continuous by six to nine months of age in the infantile form
  • Autonomic signs during seizures including apnea, perioral blueness (cyanosis), and flushing
  • Developmental plateau or regression once seizures begin, with loss or non-attainment of milestones such as sitting, walking, and speaking
  • Clusters of brief hypermotor seizures during sleep in the later-onset form, sometimes mistaken for parasomnias or night terrors
  • Poor head growth, low muscle tone, and feeding difficulties
  • Seizures that do not respond to multiple antiseizure medications (pharmacoresistance)
  • Cognitive and psychiatric difficulties in the later-onset form, sometimes out of proportion to seizure burden

Who KCNT1-Related Epilepsy Affects

KCNT1-related epilepsy is inherited in an autosomal dominant manner, but most affected children are the only person in their family with the condition. Their variant arose new (de novo) at conception rather than being passed down. Families with the later-onset sleep-related form more often show the variant across multiple generations. Both sexes are affected.

The age at onset largely sorts the two presentations. The infantile developmental and epileptic encephalopathy form begins in the first six months of life, with roughly 80% of cases starting in infancy. The sleep-related hypermotor form typically begins in later childhood, adolescence, or adulthood. Genotype tends to track with phenotype: variants in the first regulator of conductance of potassium (RCK1) domain are more often associated with the severe infantile form, while variants in the RCK2 domain are more often associated with the sleep-related form.

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Genetic Testing

Genetic testing can confirm a diagnosis, guide treatment decisions, and identify family members who may be at risk.

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Trusted KCNT1-Related Epilepsy Resources

Reputable organizations and medical references for learning more about KCNT1-Related Epilepsy, including disease registries, foundation resources, and clinical guidelines.

Active Clinical Trials for KCNT1-Related Epilepsy

Use this KCNT1-Related Epilepsy clinical trial finder to see the 3 studies recruiting patients in the United States and worldwide, with eligibility criteria in plain English. These studies play a critical role in advancing care for neurological & neuromuscular conditions and may offer access to treatments not yet widely available. Each trial below is sourced directly from ClinicalTrials.gov, with eligibility criteria translated into plain English to help patients and caregivers evaluate whether a study may be a fit.

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Note: Trial recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator before making plans.

3 active trials worldwide
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RECRUITINGPHASE1, PHASE2Recently updatedNCT07227857

A First-in-human Study of S230815 in Pediatric Participants With KCNT1-related Developmental and Epileptic Encephalopathy

Intervention: S230815- Starting dose A, S230815- Dose B, S230815- Dose C, S230815- Dose D

Sponsor: Institut de Recherches Internationales Servier

Study CL1-230815-001 (KANDLE) is a Phase Ib/II, First In Human, multicentre, open-label, multiple ascending dose study to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamic (PD) effect of S230815 in pediatric participants with KCNT1-related Developmental E...

Ages 2 Years – 12 Years15 locations
Started Nov 2025Updated 1 month agoEst. Apr 2028 (~1y 6m)
RECRUITINGPHASE2Recently updatedNCT07600736

A Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Clinical Activity of ABS-1230 in Pediatric and Young Adult Participants With KCNT1-related Epilepsy

Intervention: ABS-1230, Placebo

Sponsor: Actio Biosciences, Inc.

This trial will evaluate the safety, tolerability, pharmacokinetics, and clinical activity of ABS-1230 compared with placebo in participants with KCNT1-related epilepsy

Ages 1 Month – 21 Years1 location
Started May 2026Updated 1 month agoEst. Jan 2027 (~4 months)
RECRUITINGPHASE1Recently updatedNCT07156201

A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ABS-1230 Given Orally Compared With Placebo in Healthy Participants Aged 18 to 55 Years

Intervention: ABS-1230, Placebo, Omeprazole, Voriconazole

Sponsor: Actio Biosciences, Inc.

This first in human trial will evaluate the safety, tolerability, and pharmacokinetics of single ascending doses, multiple ascending doses, and fed and fasted doses of ABS-1230 given orally compared with placebo in adult healthy participants.

Ages 18 Years – 55 Years1 location
Started Aug 2025Updated 1 month agoEst. Dec 2026 (~3 months)
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Active trial locations6 cities in the US

Trial Pipeline

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Data from ClinicalTrials.gov, U.S. National Library of Medicine.
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Related Neurological & Neuromuscular Conditions

Other rare diseases in the neurological & neuromuscular category. Patients with KCNT1-Related Epilepsy may find relevant research, shared treatment pathways, or overlapping clinical trials among these related conditions.

Companies Developing KCNT1-Related Epilepsy Treatments

3 pharmaceutical companies have KCNT1-Related Epilepsy in their rare disease portfolio

Frequently Asked Questions About KCNT1-Related Epilepsy