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New treatment option for Epileptic Encephalopathy
Official title A First-in-human Study of S230815 in Pediatric Participants With KCNT1-related Developmental and Epileptic Encephalopathy
ClinicalTrials.gov ID: NCT07227857
What this study is testing
What is S230815- Starting dose A?
S230815- Starting dose A is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for epileptic encephalopathy.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Study CL1-230815-001 (KANDLE) is a Phase Ib/II, First In Human, multicentre, open-label, multiple ascending dose study to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamic (PD) effect of S230815 in pediatric participants with KCNT1-related Developmental Epileptic Encephalopathy. To participate in the study, participants must have a diagnosis of Developmental Epileptic Encephalopathy due to a documented pathogenic or likely pathogenic variant in KCNT1 (to be confirmed by central genetic testing at the screening visit).
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 12
You may be able to join if
- Male or female pediatric participants aged 2-12 years old at screening, with a genetically confirmed diagnosis of Developmental Epileptic...
- Stable dose of other regular medications and/or stable antiseizure interventions (such as ketogenic diet and vagal nerve stimulation).
You likely can't join if
- Other clinical phenotypes associated with pathogenic or likely pathogenic variants in KCNT1 other than Epilepsy of Infancy with Migrating Focal...
- Documented pathogenic or likely pathogenic variants in any other gene known to cause epilepsy identified through prior genetic testing. Variants of...
- Clinically significant medical history or clinical findings on physical examination, other than DEE, that in the judgment of the investigator, make...
- 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:
See the full eligibility criteria
- 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...
- Stable dose of other regular medications and/or stable antiseizure interventions (such as ketogenic diet and vagal nerve stimulation).
- 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...
- 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...
- 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...
- 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...
- 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 treatment 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...
The study team makes the final eligibility decision.
Where it's taking place
- Orange, California, United States
- Boston, Massachusetts, United States
- Rochester, New York, United States
- Columbus, Ohio, United States
- Philadelphia, Pennsylvania, United States
- Dallas, Texas, United States
- Marseille, France
- Paris, France
- Florence, Italy
- Roma, Italy
- Nagano, Japan
- Osaka, Japan
- Shizuoka, Japan
- Esplugues de Llobregat, Spain
- Madrid, Spain
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, 2 years to 12 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Orange, California, United States; Boston, Massachusetts, United States; Rochester, New York, United States; Columbus, Ohio, United States; Philadelphia, Pennsylvania, United States; Dallas, Texas, United States and 9 more location(s). 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 ClinicalTrials.gov; the study team decides eligibility.