KCNT1-Related
Epilepsy &
ABS-1230
What is ABS-1230?
ABS-1230 is an investigational medicine taken by mouth or by feeding tube that is being developed for people living with KCNT1-related epilepsy, a rare, severe form of genetic epilepsy. Most of these patients experience frequent and treatment-resistant seizures that typically begin during infancy and are accompanied by developmental delays and neurological impairments.
ABS-1230 is designed to directly target the root cause of the condition by inhibiting the overactive KCNT1 ion channel in the brain, with the goal of reducing seizures and improving quality of life. Importantly, it is designed to be effective regardless of the specific KCNT1 gene mutation a patient carries.
About the Phase 1b/2 KYRON Clinical Trial
Actio Biosciences is studying an investigational medicine called ABS-1230 in a Phase 1b/2 clinical trial, called KYRON, in the United States. This trial will enroll infants, children and young adults – from one month to 21 years old – with KCNT1-related epilepsy. Study enrollment will begin with older children and young adults first, then move to younger children, and then infants as more is known about the safety, activity, and appropriate dosing of ABS-1230 in people with KCNT1-related epilepsy. The goal of the trial is to understand whether ABS-1230 is safe, how the body absorbs and processes it, and whether it may help reduce seizures.
Key Details:
The trial has three parts
Part 1 (12 weeks)
All participants receive ABS-1230. This part is open-label, which means everyone knows they are receiving ABS-1230. This part of the study is intended to confirm the dose that will be given in Part 2.
Part 2 (12 weeks)
A new group of participants is randomly assigned to receive either ABS-1230 or a placebo (a look-alike with no active medicine). This part is double-blinded, so neither the families nor the doctors know which one the participant is receiving.
Part 3
An optional long-term extension where participants from both Parts 1 and 2 can continue to receive ABS-1230, or start to receive ABS-1230 if they received placebo in Part 2.
Other Details
Format: Decentralized – this means that you will have the option to participate in the trial from your home. Some study activities will take place through video visits, while other activities will involve healthcare providers coming to your home.
Location: United States
Duration: At least 20 weeks, including a minimum of 4 weeks for screening, 12 weeks of treatment, and 4 weeks of follow-up or the option to continue treatment in an open-label long-term extension part of the trial.
Visits: Remote via telemedicine and home health visits, including blood tests, ECG, and in-home EEG testing. You will have the option to go to a site in-person if that is preferred.
Eligibility Criteria
Your child may be eligible if:
- They have a confirmed KCNT1-related epilepsy diagnosis with a pathogenic or likely pathogenic KCNT1 mutation
- They have the EIMFS or EOEE phenotype of the disease
- They experience 4 or more motor seizures per week
What the Study Will Measure
The clinical trial will evaluate several aspects of ABS-1230, including:
- Overall safety and tolerability
- Change in the number of seizures
- The impact of seizures, including seizure-free days, rescue medication, and life disruptions due to seizures and side effects of antiseizure medications
- Change in development, quality-of-life, and caregiver burden measures
- Change in sleep quality
To learn more about this trial, including eligibility key criteria, please visit our listing on ClinicalTrials.gov.
KCNT1 Foundation
For more information about KCNT1-related epilepsy, new therapies in development, the latest research information and resources for families and caregivers, please visit the KCNT1 Epilepsy Foundation website. The Foundation provides educational materials and a supportive community for families navigating this rare disease.