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
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental ABS-1230 (Part 1) |
|
|
|
Experimental ABS-1230 (Part 2) |
|
|
|
Experimental Placebo (Part 2) |
|
|
|
Experimental ABS-1230 (Part 3) |
|
Recruiting Locations
Northeast Regional Epilepsy Group
Hackensack, New Jersey 07601
Hackensack, New Jersey 07601
More Details
- Status
- Recruiting
- Sponsor
- Actio Biosciences, Inc.
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).