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

Actio Biosciences, Inc.

Status and phase: Enrolling; Phase 2

Conditions: KCNT1-Related Epilepsy

Treatments: Drug: ABS-1230; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT07600736

ABS1230-1101

Details and patient eligibility

About

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

Full 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).

Enrollment: 55 estimated patients

Sex: All

Ages: 1 month to 21 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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55 participants in 4 patient groups

ABS-1230 (Part 1)

Experimental group

Treatment:

Drug: ABS-1230

ABS-1230 (Part 2)

Experimental group

Treatment:

Drug: ABS-1230

Placebo (Part 2)

Experimental group

Treatment:

Drug: Placebo

ABS-1230 (Part 3)

Experimental group

Treatment:

Drug: ABS-1230