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

NCT07600736 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: ABS1230-1101
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

KCNT1-Related Epilepsy

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (2)

• Maximum Plasma Concentration [Cmax] of ABS-1230

  Measured from Day 1 to End of Study or Early Termination (up to 12 weeks)

• Total Exposure [AUCtau] ABS-1230

  Measured from Day 1 to End of Study or Early Termination (up to 12 weeks)

Study Arms (4)

ABS-1230 (Part 1)

EXPERIMENTAL

Drug: ABS-1230

ABS-1230 (Part 2)

EXPERIMENTAL

Drug: ABS-1230

Placebo (Part 2)

EXPERIMENTAL

Drug: Placebo

ABS-1230 (Part 3)

EXPERIMENTAL

Drug: ABS-1230

Interventions

ABS-1230 DRUG

Once daily

ABS-1230 (Part 1); ABS-1230 (Part 2); ABS-1230 (Part 3)

Placebo DRUG

Once daily

Placebo (Part 2)

Eligibility Criteria

• Age: 1 Month - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Actio Biosciences, Inc.: lead

Study Sites (1)

Northeast Regional Epilepsy Group

Hackensack, New Jersey, 07601, United States

RECRUITING

Central Study Contacts

Medical Director Actio Biosciences, Inc.

CONTACT

+1 (858) 682-4042; info@actiobiosciences.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: Part 1 will be open-label. Part 2 of the study will be conducted in a randomized double-blind manner. Part 3 will be an open-label extension.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 14, 2026
• First Posted: May 22, 2026
• Study Start: May 18, 2026
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): January 1, 2028
• Last Updated: June 17, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)