In short
Clinical trials are investigating S230815-2 in children and adolescents with KCNT1-related Developmental and Epileptic Encephalopathy. The study is looking mainly at safety and tolerability, and it is an early-phase trial. It is designed for paediatric participants with this rare epilepsy-related condition.
Key points
- S230815-2 is being studied in an early-phase clinical trial for paediatric participants with KCNT1-related Developmental and Epileptic Encephalopathy. The trial is Phase 1/2 and is open-label, meaning everyone in the study knows what treatment is being used. Researchers are mainly checking safety and tolerability. The main endpoint is the incidence and severity of adverse events, which are unwanted medical problems during the study. The trial is authorised and plans to enroll 28 participants. The study uses an intrathecal solution for injection.
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.
It is a multicentre, open-label, multiple ascending dose study in paediatric participants with KCNT1-related Developmental and Epileptic Encephalopathy.
The study is designed to evaluate the safety and tolerability of S230815-2 in this rare epilepsy-related condition.
Who can join the study
The target population is paediatric participants, meaning children and adolescents.
To be part of the study, participants must have KCNT1-related Developmental and Epileptic Encephalopathy, also called KCNT1-DEE.
This means the trial is focused on a very specific group of young patients with a rare condition that affects development and seizures.
Study design and phase
The trial is in Phase 1/2, which is an early stage of clinical research.
Early-phase studies usually look first at safety, then at early signs of whether a treatment may help.
The study is open-label, so the research team and the participants know what treatment is being given.
It is also a multiple ascending dose study, meaning the study may test increasing amounts of the treatment over time in different groups.
The intervention listed is S230815 Solution for injection 10mg/ml (intrathecal use).
What the study is measuring
The main outcome is the incidence and severity of adverse events.
Adverse events are unwanted medical problems that happen during a study, whether or not they are caused by the treatment.
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.
Trial status and size
The trial status is Authorised.
The planned enrollment is 28 participants, which means the study aims to include 28 people.
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.