Actively Recruiting
Phase IbII Study of Intrathecal S230815 in Children Aged 2 to 12 With KCNT1-related Developmental and Epileptic Encephalopathy
Led by Institut de Recherches Internationales Servier · Updated on 2026-08-11
20
Participants Needed
15
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying pediatric participants aged 2 to 12 years with Developmental Epileptic Encephalopathy caused by a genetic variant in the KCNT1 gene. This Phase IbII, first-in-human, multicenter, open-label study aims to assess the safety, tolerability, and how the investigational drug S230815 behaves in the body and affects the disease. The trial is sponsored by Institut de Recherches Internationales Servier and includes a screening period followed by two treatment parts. Participants will receive multiple ascending doses of S230815 administered intrathecally during Part 1 of the study. Those who complete Part 1 may continue into Part 2, a long-term treatment extension lasting up to 72 weeks, continuing with the same dose cohort as in Part 1. The treatment is given as a solution for injection, and dosing is designed to evaluate safety and drug effects over time. Throughout the study, participants will be closely monitored for adverse events and drug levels in cerebrospinal fluid and plasma. Seizure frequency will be tracked using daily logs and periodic 24-hour video EEG assessments. Researchers will also record the use of rescue medications. The total participation may last up to 116 weeks, including the screening, treatment, and follow-up visits, ensuring thorough evaluation of the drugs impact and safety profile.
CONDITIONS
Brief Title
A First-in-human Study of S230815 in Pediatric Participants With KCNT1-related Developmental and Epileptic Encephalopathy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female pediatric participants aged 2 to 12 years at screening
- Genetically confirmed diagnosis of Developmental Epileptic Encephalopathy due to a pathogenic or likely pathogenic KCNT1 variant confirmed by central genetic testing
- Stable dose of other regular medications and/or stable antiseizure interventions such as ketogenic diet and vagal nerve stimulation
You will not qualify if you...
- Other clinical phenotypes associated with KCNT1 variants besides Epilepsy of Infancy with Migrating Focal Seizures or Early-Onset Epileptic Encephalopathy
- Pathogenic or likely pathogenic variants in other epilepsy-causing genes identified by prior genetic testing
- Clinically significant medical history or findings that make participation unsuitable, including recent medical conditions within 30 days
- Significant abnormality on ECG or laboratory tests at screening, including renal insufficiency (creatinine clearance < 40 mL/min) and hepatic derangement (transaminases > 3x ULN or bilirubin > 1.5x ULN)
- Positive tests for hepatitis B, hepatitis C, or HIV within 6 months or at screening
- Bone, spine, bleeding disorders, or other conditions increasing risk with lumbar puncture
- Contraindications to MRI, lumbar puncture, or intrathecal administration
- History of CNS tumors or malignancies
- Continuous respiratory support or invasive ventilation
- Use of quinidine within 30 days prior to screening
- Current or anticipated use of antiplatelet or anticoagulant therapy
- Participation in another interventional clinical study with investigational therapy within 30 days prior to screening
- Implantable CNS devices interfering with intrathecal drug administration
- Known hypersensitivity to oligonucleotides or other drugs precluding participation
- History of hydrocephalus requiring a ventriculoperitoneal shunt
Research Team
I
Institut de Recherches Internationales Servier
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