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Study of intrathecal S230815 to evaluate safety and effectiveness in children with KCNT1-related Developmental and Epileptic Encephalopathy

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on children with KCNT1-related Developmental and Epileptic Encephalopathy, a rare genetic condition that causes severe epilepsy and developmental delays in young children. The study will test a new medication called S230815, which is given through an injection into the spinal fluid. This medication is designed to target a specific gene called KCNT1 that is involved in causing this type of epilepsy.

The purpose of this research is to determine if the medication is safe and how well children's bodies process it. The study will involve multiple doses of the medication, with the amount being gradually increased over time. The medication is being tested for the first time in humans, specifically in children between 2 and 12 years of age who have confirmed genetic changes in the KCNT1 gene.

During the study, participants will receive the medication through intrathecal injections (delivered directly into the fluid surrounding the spinal cord). The medication is an antisense oligonucleotide, which is a type of drug that can reduce the activity of specific genes. Throughout the study, doctors will monitor how the children respond to the treatment and check for any side effects.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial medical assessment

    Your participation begins with confirmation of a genetic diagnosis of KCNT1-related Developmental and Epileptic Encephalopathy

    A medical team will verify that you are between 2-12 years old and meet the requirements for the procedure

    Your current seizure frequency will be documented through daily seizure logs

  2. Step 2

    Medication preparation

    The study medication (S230815) will be prepared as an injectable solution with a concentration of 10mg/ml

    Your current medications and treatments (such as ketogenic diet or nerve stimulation) will need to remain at stable doses throughout the study

  3. Step 3

    Treatment administration

    The medication will be administered through an intrathecal injection (an injection into the fluid-filled space around the spinal cord)

    Multiple doses will be given over the course of the study

    The medical team will monitor how your body processes the medication by measuring drug levels in your system

  4. Step 4

    Monitoring period

    Your safety will be closely monitored through regular medical check-ups

    Any side effects or adverse events will be recorded and evaluated

    The medical team will track how well the medication is working

  5. Step 5

    Study duration

    The study is scheduled to run from September 2025 to April 2028

    Your individual participation duration will be determined by the study protocol

Who can join the trial?

10 criteria

  • Children between 2 and 12 years old can participate
  • Both boys and girls can take part in the study
  • Must have a confirmed genetic diagnosis of Developmental Epileptic Encephalopathy (a severe form of epilepsy that affects development) caused by changes in the KCNT1 gene
  • Can have either:
    • Epilepsy of Infancy with Migrating Focal Seizures (seizures that move from one part of the brain to another), or
    • Early-Onset Epileptic Encephalopathy (severe epilepsy that starts early in life)
    • Family must be able to keep daily records of seizures
    • Must be on a stable dose of current medications or treatments, such as:
      • Regular medicines
      • Ketogenic diet (special high-fat, low-carbohydrate diet)
      • Vagal nerve stimulation (device that helps control seizures)
      • Must be suitable for a lumbar puncture procedure (a medical test where a small sample of fluid is taken from the lower back) according to hospital guidelines

Who cannot join the trial?

15 criteria

  • History of severe allergic reactions or anaphylaxis (life-threatening allergic reaction) to any medications
  • Participation in another clinical trial within the past 30 days
  • Major surgery in the last 3 months
  • Significant heart, liver, or kidney problems
  • Uncontrolled high blood pressure
  • Current or recent serious infections
  • History of cancer in the past 5 years
  • Pregnant or breastfeeding women
  • Use of certain medications that might interact with the study drug
  • Unable to follow study procedures or attend scheduled visits
  • Any condition that, in the opinion of the study doctor, would make participation unsafe
  • History of non-compliance with medical treatments
  • Unstable medical conditions that require frequent medication changes
  • Active substance abuse or addiction
  • Inability to provide informed consent (for adults) or lack of parent/guardian consent (for children)
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Investigated drugs

S230815 is an experimental medication that is administered intrathecally (directly into the spinal fluid). It is being studied for the first time in humans, specifically in children who have a rare form of epilepsy called KCNT1-related Developmental and Epileptic Encephalopathy. This medication is designed to treat seizures and developmental issues associated with this specific genetic form of epilepsy. The treatment is being tested to understand how safe it is and how well children's bodies process and respond to it.

What is already known about the treatment

S230815 - An investigational drug administered intrathecally (directly into the spinal fluid) that is currently being studied in clinical trials for the treatment of KCNT1-related Developmental and Epileptic Encephalopathy in pediatric patients. This novel therapeutic agent is undergoing Phase Ib/II trials to evaluate its safety, tolerability, and pharmacological properties in children with this rare genetic form of epilepsy. While the exact molecular mechanism of action is still being investigated through these first-in-human studies, the drug is specifically designed to target the neurological pathways affected by KCNT1 gene mutations that cause severe seizures and developmental delays.

Investigated diseases

KCNT1-related Developmental and Epileptic Encephalopathy - A rare genetic neurological disorder that affects brain development and function. The condition typically begins in early childhood and is characterized by frequent seizures and developmental delays. It is caused by mutations in the KCNT1 gene, which affects how brain cells communicate with each other. The disorder impacts various aspects of development, including motor skills, speech, and cognitive abilities. Children with this condition often experience different types of seizures that can occur multiple times per day.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase I/IITrial ID2024-513332-17-00Protocol codeCL1-230815-001Estimated enrolment28 patientsSponsorInstitut De Recherches Internationales Servier IRIS

sourced from the EU Clinical Trials Register and site verification

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