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Study of S230815 for KCNT1-related Epileptic Encephalopathy in Children

This study is testing a new medication called S230815 for children aged 2 to 12 years old who have a specific type of epilepsy called KCNT1-related Developmental and Epileptic Encephalopathy (DEE). This is the first time S230815 is being given to humans. The main goal is to see how safe S230815 is and what side effects it might cause. Researchers will also look at how the body handles the medication and if it has any effects on the condition. To join, your child must have a confirmed diagnosis of KCNT1-related DEE. The study is currently unclear about its recruitment status.

Study design
This is a Phase Ib/II, open-label study, meaning everyone knows what treatment is being given. It plans to enroll 20 participants and will test different doses of S230815.
What's involved
The study involves a screening period, followed by two parts: an initial treatment phase with ascending doses of S230815, and a long-term treatment extension if Part 1 is completed.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for up to 116 weeks to monitor for any adverse events (side effects).

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07227857

A First-in-human Study of S230815 in Pediatric Participants With KCNT1-related Developmental and Epileptic Encephalopathy

Recruiting
PHASE1Ages 2–12InterventionalTreatment
Institut de Recherches Internationales Servier
~20 participants
Updated 2026-08-11 on ClinicalTrials.gov
What's tested:S230815- Starting dose AS230815- Dose BS230815- Dose CS230815- Dose D

At a glance

Recruiting sites
9 of 15 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence and severity of Adverse Events (AE)'s.
Measured over Through End of study visit (A maximum of 116 weeks)

Conditions

Epileptic Encephalopathy

Explore related trials

Epileptic encephalopathyKCNT1 Gene

NCT07227857

Where you'd take part

This study runs at 15 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Azienda Ospedaliera Universitaria Meyer IRCCS

    Florence, Italystudy coordinator listed

    Not yet recruiting

  • Boston Children's Hospital

    Boston, Massachusettsstudy coordinator listed

    Recruiting

  • Hopital Necker Enfants Malades

    Paris, Francestudy coordinator listed

    Recruiting

  • Hospital Ruber Internacional

    Madrid, Spainstudy coordinator listed

    Recruiting

  • Hospital Sant Joan De Deu Barcelona

    Esplugues de Llobregat, Spainstudy coordinator listed

    Recruiting

  • Institut Des Neurosciences De La Timone

    Marseille, Francestudy coordinator listed

    Recruiting

  • Ospedale Pediatrico Bambino Gesu

    Roma, Italystudy coordinator listed

    Not yet recruiting

  • Robert Debre University Hospital

    Paris, Francestudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Who to contact

Institut de Recherches Internationales Servier
[email protected]
+33 1 55 72 60-00
Email the study team

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Eligibility criteria

Inclusion

Male or female pediatric participants aged 2-12 years old at screening, with a genetically confirmed diagnosis of Developmental Epileptic Encephalopathy (DEE) due to a pathogenic or likely pathogenic variant in KCNT1 confirmed by central genetic testing.
Stable dose of other regular medications and/or stable antiseizure interventions (such as ketogenic diet and vagal nerve stimulation).

Exclusion

Other clinical phenotypes associated with pathogenic or likely pathogenic variants in KCNT1 other than Epilepsy of Infancy with Migrating Focal Seizures or Early-Onset Epileptic Encephalopathy
Documented pathogenic or likely pathogenic variants in any other gene known to cause epilepsy identified through prior genetic testing. Variants of uncertain significance in other genes known to cause epilepsy may be considered on discussion with the sponsor.
Clinically significant medical history or clinical findings on physical examination, other than DEE, that in the judgment of the investigator, make the participant unsuitable for participation in the study and/or completion of the trial procedures, including, but not limited to:
Clinically significant prior or ongoing medical conditions within 30 days of the screening visit, as per investigator judgement.
Clinically significant abnormality on Electrocardiogram (ECG) at the screening visit, as per investigator judgement.
Clinically significant abnormality on laboratory testing at screening, including, but not limited to:
Renal insufficiency, which is defined as creatinine clearance \< 40 mL/min assessed as estimated glomerular filtration rate (eGFR) using Modification of Diet in Renal Disease (MDRD) formula
Hepatic derangement defined as transaminase values more than 3 times the Upper Limit of Normal (ULN) range, or total bilirubin values more than 1.5 times the ULN.
Positive hepatitis B surface antigen test, positive hepatitis C antibody test, positive for human immunodeficiency virus (HIV), as reported by a laboratory test within 6 months prior to the screening visit, or on screening bloods.
Bone, spine, bleeding disorders, or other disorder that exposes the participant to risk of injury or unsuccessful Lumbar puncture (e.g., haemophilia, Von Willebrand's disease, liver disease).
Contraindications to undergoing Magnetic Resonance Imaging (MRI), Lumbar puncture procedure and Intrathecal administration.
History of Central Nervous System (CNS) tumors or malignancies, including CNS metastatic disease.
Continuous respiratory support, defined as oxygen supplementation or non-invasive ventilation (e.g.: continuous positive airway pressure, bi-level intermittent positive airway pressure), required during waking hours. This does not include suctioning; cough assist devices or other devices that may be used regularly to clear airways.
Invasive ventilation including the presence of a tracheostomy.
Use of quinidine within 30 days prior to the screening visit.
Current use or anticipated use of antiplatelet or anticoagulant therapy during the study.
Current or past enrolment in an interventional clinical study in which an investigational therapy is/was administered within 30 days (or 5 half-lives of study agent, whichever is longer) prior to the screening visit.
Implantable CNS device that may interfere with the ability to administer the study drug via Lumbar puncture.
Known hypersensitivity to any oligonucleotide, as demonstrated by a systemic allergic reaction (e.g., changes in pulse, blood pressure, breathing function, etc.), or any other drug that in the opinion of the investigator may preclude study participation.
History of hydrocephalus requiring a ventriculoperitoneal shunt.

What this trial measures

  • Incidence and severity of Adverse Events (AE)'s.Through End of study visit (A maximum of 116 weeks)