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

Purpose

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

Condition

  • KCNT1-Related Epilepsy

Eligibility

Eligible Ages
Between 1 Month and 21 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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 Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Triple (Participant, Care Provider, Investigator)
Masking Description
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.

Arm Groups

ArmDescriptionAssigned Intervention
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

Recruiting Locations

Northeast Regional Epilepsy Group
Hackensack, New Jersey 07601
Contact:
Hardik Rana
551-497-5000
HRana@epilepsygroup.com

More Details

Status
Recruiting
Sponsor
Actio Biosciences, Inc.

Study Contact

Medical Director Actio Biosciences, Inc.
+1 (858) 682-4042
info@actiobiosciences.com

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).