KCNT1-Related Epilepsy
Conditions
Brief summary
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
DRUGABS-1230
Daily
DRUGPlacebo
Daily
Sponsors
Actio Biosciences, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, 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.
Eligibility
Sex/Gender
ALL
Age
1 Months to 21 Years
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Part 1: Safety and tolerability of ABS-1230 (incidence and severity of adverse events) | Measured from Day 1 to End of Study or Early Termination (up to 12 weeks) | Safety and tolerability of ABS-1230 by determining the incidence and severity of treatment emergent adverse events |
| Part 2: Efficacy of ABS-1230 | Measured from Day 1 to End of Study or Early Termination (up to 12 weeks) | Percentage change from baseline in countable motor seizures (normalized per 28 days) as recorded in the seizure diary |
| Part 3: Safety and tolerability of ABS-1230 (incidence and severity of adverse events) | Measured from Day 1 of Part 3 to End of Study or Early Termination (up to 1 year) | Safety and tolerability of ABS-1230 by determining the incidence and severity of treatment emergent adverse events |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Maximum Plasma Concentration [Cmax] of ABS-1230 | Measured from Day 1 to End of Study or Early Termination (up to 12 weeks) | Pharmacokinetics of ABS-1230 |
| Total Exposure [AUCtau] ABS-1230 | Measured from Day 1 to End of Study or Early Termination (up to 12 weeks) | Pharmacokinetics of ABS-1230 |
Countries
United States
Contacts
CONTACTMedical Director Actio Biosciences, Inc.
Outcome results
None listed