Actively Recruiting
A Phase Ib/II First-in-human, Multicentre, Open-label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effect of Intrathecal S230815 in Pediatric Participants With KCNT1-related Developmental and Epileptic Encephalopathy
Led by Institut de Recherches Internationales Servier · Updated on 2026-05-26
20
Participants Needed
16
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting a Phase Ib/II, first-in-human, multicenter, open-label study to assess the safety, tolerability, and how the body processes and responds to the drug S230815 in children aged 2 to 12 years with Developmental Epileptic Encephalopathy caused by a genetic variant in the KCNT1 gene. To join, participants must have a confirmed diagnosis through genetic testing. The study is sponsored by Institut de Recherches Internationales Servier and focuses on this rare pediatric epilepsy condition. The study includes a screening period followed by two parts. Part 1 tests multiple increasing doses of S230815 given as a solution for injection. Part 2 is a long-term treatment extension where participants continue receiving the same dose for up to 72 weeks after completing Part 1. Participants will move seamlessly from Part 1 to Part 2, staying in their assigned dose group. Participants will be closely monitored throughout, with assessments including daily seizure logs and periodic 24-hour video EEGs to record seizure frequency. Researchers will also evaluate drug levels in the cerebrospinal fluid and blood plasma. Safety is carefully tracked by recording any adverse events through the study, which can last up to 116 weeks. The study aims to understand how safe and tolerable S230815 is and how it affects seizures in this patient group.
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 2-12 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 linked to KCNT1 variants except 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 abnormalities on physical exam or lab tests that may affect study participation
- Renal insufficiency with creatinine clearance less than 40 mL/min
- Liver enzyme abnormalities exceeding 3 times the normal limit or high bilirubin
- Positive tests for hepatitis B, hepatitis C, or HIV within 6 months prior to screening
- Disorders increasing risk for lumbar puncture injury such as bleeding disorders
- Contraindications to MRI, lumbar puncture, or intrathecal drug administration
- History of central nervous system tumors or malignancies
- Continuous respiratory support or invasive ventilation
- Use of quinidine within 30 days prior to screening
- Current or planned use of antiplatelet or anticoagulant therapy
- Participation in other interventional studies within 30 days prior to screening
- Implantable CNS devices interfering with lumbar puncture
- Known hypersensitivity to oligonucleotides or any drugs that preclude study participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 116 weeks
Participants receive intrathecal doses of S230815 in multiple ascending dose cohorts to assess safety, tolerability, pharmacokinetics, and pharmacodynamics.
Multiple visits for dosing and assessments throughout treatment
Trial Site Locations
Total: 16 locations
1
Children's Hospital of Orange County
Orange, California, United States, 92868
Not Yet Recruiting
2
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
3
University of Rochester Medical Center
Rochester, New York, United States, 14642
Not Yet Recruiting
4
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Not Yet Recruiting
5
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Not Yet Recruiting
6
Children's Health Dallas
Dallas, Texas, United States, 75235
Not Yet Recruiting
7
Institut Des Neurosciences De La Timone
Marseille, France, 13005
Actively Recruiting
8
Hopital Necker Enfants Malades
Paris, France, 75015
Actively Recruiting
9
Robert Debre University Hospital
Paris, France, 75019
Actively Recruiting
10
Azienda Ospedaliera Universitaria Meyer IRCCS
Florence, Italy, 50139
Not Yet Recruiting
11
Ospedale Pediatrico Bambino Gesu
Roma, Italy, 00165
Not Yet Recruiting
12
Shinshu University Hospital
Nagano, Japan
Actively Recruiting
13
Osaka City General Hospital
Osaka, Japan
Actively Recruiting
14
Shizuoka Institute of Epilepsy and Neurological Disorders
Shizuoka, Japan
Actively Recruiting
15
Hospital Sant Joan De Deu Barcelona
Esplugues de Llobregat, Spain, 08950
Actively Recruiting
16
Hospital Ruber Internacional
Madrid, Spain, 28035
Actively Recruiting
Research Team
I
Institut de Recherches Internationales Servier
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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