A Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Clinical Activity of ABS-1230 in Pediatric and Young Adult Participants With KCNT1-related Epilepsy
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: ABS-1230, Placebo.
- Who it may be relevant to
- Registry conditions: KCNT1-Related Epilepsy. Basic parameters: 1 months — 21 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
A Phase 1b/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Clinical Activity of ABS-1230 Administered to Pediatric and Young Adult Participants With KCNT1-Related Epilepsy
Overview
This trial will evaluate the safety, tolerability, pharmacokinetics, and clinical activity of ABS-1230 compared with placebo in participants with KCNT1-related epilepsy
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).
Interventions
- Drug ABS-1230
Daily - Drug Placebo
Daily
Primary outcome measures
- Part 1: Safety and tolerability of ABS-1230 (incidence and severity of 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 [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) [Time frame: Measured from Day 1 of Part 3 to End of Study or Early Termination (up to 1 year)]
Secondary outcome measures (2)
- Maximum Plasma Concentration [Cmax] 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 [Time frame: Measured from Day 1 to End of Study or Early Termination (up to 12 weeks)]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Triple blind
- Primary purpose
- Treatment
Study locations
United States · 1 center
- Northeast Regional Epilepsy Group — Hackensack
Identifiers
NCT: NCT07600736 · ABS1230-1101