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A Phase Ib/II first-in-human, multicentre, open-label, multiple ascending dose study of S230815 in paediatric participants with KCNT1-related Developmental and Epileptic Encephalopathy
EU CTIS ID: 2024-513332-17-00
What this study is testing
To evaluate the safety and tolerability of S230815 in paediatric participants with KCNT1-Developmental and Epileptic Encephalopathy (KCNT1-DEE).
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male or female paediatric participants aged 2-12 years old at screening, with a genetically confirmed diagnosis of Developmental Epileptic Encephalopathy (DEE) (Epilepsy of Infancy with Migrating Focal Seizures (EIMFS) or non-EIFMS Early-Onset Epileptic Encephalopathy (EOEE) phenotypes) due to a pathogenic or likely pathogenic variant in KCNT1 confirmed by central genetic testing.[CCI]
- [CCI] Seizure count data will be captured by daily family seizure logs.
- [CCI] stable dose of other regular medications and/or stable antiseizure interventions (such as ketogenic diet and vagal nerve stimulation).[CCI]
- Must meet age-appropriate institutional guidelines for LP procedure.
You likely can't join if
- Other clinical phenotypes associated with pathogenic or likely pathogenic variants in KCNT1 other than EIMFS or EOEE (e.g., Sleep-related Hypermotor Epilepsy SHE).
- Contraindications to undergoing Magnetic Resonance Imaging (MRI), lumbar puncture (LP) procedure and intrathecal (IT) 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.
- History of hydrocephalus requiring a ventriculoperitoneal shunt
See the full eligibility criteria
Who can join
- Male or female paediatric participants aged 2-12 years old at screening, with a genetically confirmed diagnosis of Developmental Epileptic Encephalopathy (DEE) (Epilepsy of Infancy with Migrating Focal Seizures (EIMFS) or non-EIFMS Early-Onset Epileptic Encephalopathy (EOEE) phenotypes) due to a pathogenic or likely pathogenic variant in KCNT1 confirmed by central genetic testing.[CCI]
- [CCI] Seizure count data will be captured by daily family seizure logs.
- [CCI] stable dose of other regular medications and/or stable antiseizure interventions (such as ketogenic diet and vagal nerve stimulation).[CCI]
- Must meet age-appropriate institutional guidelines for LP procedure.
What rules you out
- Other clinical phenotypes associated with pathogenic or likely pathogenic variants in KCNT1 other than EIMFS or EOEE (e.g., Sleep-related Hypermotor Epilepsy SHE).
- Contraindications to undergoing Magnetic Resonance Imaging (MRI), lumbar puncture (LP) procedure and intrathecal (IT) 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.
- History of hydrocephalus requiring a ventriculoperitoneal shunt
- 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 LP.
- 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: 1) Clinically significant prior or ongoing medical conditions within 30 days of the screening visit, as per investigator judgement. 2) Clinically significant abnormality on electrocardiogram (ECG) at the screening visit, as per investigator judgement. 3) Clinically significant abnormality on laboratory testing at screening, including, but not limited to: a) Renal insufficiency, which is defined as creatinine clearance < 40 mL/min assessed as estimated glomerular filtration rate (eGFR) using Schwartz formula, b) 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 LP (e.g., haemophilia, Von Willebrand’s disease, liver disease).
The study team makes the final eligibility decision.
Where it's taking place
- Japan
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Japan; United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.