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

Phase 2RecruitingNCT07600736

Actio Biosciences, Inc.45 enrolled

Overview

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

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

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

KCNT1-Related Epilepsy

Interventions

ABS-1230DRUG

Once daily

PlaceboDRUG

Once daily

Eligibility

Sex: ALLMin age: 1 MonthMax age: 21 Years

Medical Language ↔ Plain English

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.

Locations (1)

Northeast Regional Epilepsy Group

Hackensack, New Jersey, United States

RECRUITING

Outcomes

Primary Outcomes

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

Time frame: 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

Time frame: 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

Time frame: Measured from Day 1 of Part 3 to End of Study or Early Termination (up to 1 year)

Secondary Outcomes

Maximum Plasma Concentration [Cmax] of ABS-1230

Pharmacokinetics of ABS-1230

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

Total Exposure [AUCtau] ABS-1230

Pharmacokinetics of ABS-1230

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

Central Contacts

Medical Director Actio Biosciences, Inc.

CONTACT

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

Data from ClinicalTrials.gov

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