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

A Phase 1b/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Clinical Activity of ABS-1230 Administered to Pediatric Participants With KCNT1-Related Epilepsy

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

Study Overview

• Status: Recruiting
• Conditions: KCNT1-Related Epilepsy
• Intervention / Treatment: Drug: Placebo; Drug: ABS-1230

Detailed Description

This is a Phase 1b/2 study that consists of 3 parts.

In part 1, participants will receive ABS-1230 for 12 weeks, with a follow-up period of 2 weeks.

In part 2, participants will receive ABS-1230 or placebo for 12 weeks, with a follow-up period of 2 weeks.

All participants who complete part 1 or part 2 will have the option to continue receiving ABS-1230 in an open-label extension study (part 3).

• Study Type: Interventional
• Enrollment (Estimated): 45
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Medical Diretor Actio Biosciences, Inc.; Phone Number: +1 (858) 682-4042; Email: info@actiobiosciences.com

Study Locations

• United States
  • New Jersey
    • Hackensack, New Jersey, United States, 07601
      • Recruiting
      • Northeast Regional Epilepsy Group
      • Principal Investigator: Eric Segal, MD
      • Contact: Hardik Rana; Phone Number: 551-497-5000; Email: research@epilepsygroup.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Aged 1 month to <22 years
• Clinician-confirmed diagnosis of KCNT1-related epilepsy
• Has an average of at least 4 countable motor seizures per week
• Is taking no more than 6 antiseizure medications (ASM) and is able to keep stable doses of ASMs for the duration of Part 1 or Part 2

Exclusion Criteria:

• Is currently taking phenytoin, carbamazepine, stiripentol, or quinidine
• Has a medical condition that, in the opinion of the investigator, would limit the participant's ability to participate in the study or might compromise participant safety or interfere with evaluation of the study drug.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ABS-1230 (Part 1)	Drug: ABS-1230; Once daily
Experimental: ABS-1230 (Part 2)	Drug: ABS-1230; Once daily
Experimental: Placebo (Part 2)	Drug: Placebo; Once daily
Experimental: ABS-1230 (Part 3)	Drug: ABS-1230; Once daily

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Part 1: Safety and tolerability of ABS-1230 (incidence and severity of adverse events)	Safety and tolerability of ABS-1230 by determining the incidence and severity of treatment emergent adverse events	Measured from Day 1 to End of Study or Early Termination (up to 12 weeks)
Part 2: Efficacy of ABS-1230	Percentage change from baseline in countable motor seizures (normalized per 28 days) as recorded in the seizure diary	Measured from Day 1 to End of Study or Early Termination (up to 12 weeks)
Part 3: Safety and tolerability of ABS-1230 (incidence and severity of adverse events)	Safety and tolerability of ABS-1230 by determining the incidence and severity of treatment emergent adverse events	Measured from Day 1 of Part 3 to End of Study or Early Termination (up to 1 year)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Maximum Plasma Concentration [Cmax] of ABS-1230	Pharmacokinetics of ABS-1230	Measured from Day 1 to End of Study or Early Termination (up to 12 weeks)
Total Exposure [AUCtau] ABS-1230	Pharmacokinetics of ABS-1230	Measured from Day 1 to End of Study or Early Termination (up to 12 weeks)

Collaborators and Investigators

• Sponsor: Actio Biosciences, Inc.

Study record dates

Study Major Dates

• Study Start (Estimated): May 1, 2026
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): January 1, 2028

Study Registration Dates

• First Submitted: May 14, 2026
• First Submitted That Met QC Criteria: May 14, 2026
• First Posted (Actual): May 22, 2026

Study Record Updates

• Last Update Posted (Actual): May 22, 2026
• Last Update Submitted That Met QC Criteria: May 14, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Other Study ID Numbers: ABS1230-1101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No