Table of contents
- Trial overview
- Who can join the study
- Study design and phase
- What the study is measuring
- Trial status and size
Trial overview
The clinical trial for S230815-2 is a first-in-human study, which means it is the first time the treatment is being tested in people in this setting.[1]
It is a multicentre, open-label, multiple ascending dose study in paediatric participants with KCNT1-related Developmental and Epileptic Encephalopathy.[1]
The study is designed to evaluate the safety and tolerability of S230815-2 in this rare epilepsy-related condition.[1]
Who can join the study
The target population is paediatric participants, meaning children and adolescents.[1]
To be part of the study, participants must have KCNT1-related Developmental and Epileptic Encephalopathy, also called KCNT1-DEE.[1]
This means the trial is focused on a very specific group of young patients with a rare condition that affects development and seizures.[1]
Study design and phase
The trial is in Phase 1/2, which is an early stage of clinical research.[1]
Early-phase studies usually look first at safety, then at early signs of whether a treatment may help.[1]
The study is open-label, so the research team and the participants know what treatment is being given.[1]
It is also a multiple ascending dose study, meaning the study may test increasing amounts of the treatment over time in different groups.[1]
The intervention listed is S230815 Solution for injection 10mg/ml (intrathecal use).[1]
What the study is measuring
The main outcome is the incidence and severity of adverse events.[1]
Adverse events are unwanted medical problems that happen during a study, whether or not they are caused by the treatment.[1]
By tracking how often these events happen and how serious they are, researchers can learn more about the safety profile of S230815-2 in children with KCNT1-DEE.[1]
Trial status and size
The trial status is Authorised.[1]
The planned enrollment is 28 participants, which means the study aims to include 28 people.[1]
Because the study is small and early-phase, its main purpose is to build safety information rather than to give a final answer about effectiveness.[1]
