Actively Recruiting
A First-in-human Study of S230815 in Pediatric Participants With KCNT1-related Developmental and Epileptic Encephalopathy
Led by Institut de Recherches Internationales Servier · Updated on 2026-05-12
20
Participants Needed
16
Research Sites
124 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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). The study consists of a screening period followed by two consecutive interventional parts. Part 1 will evaluate multiple ascending doses of S230815. Part 2 is a long-term treatment extension for participants who have completed Part 1. Participants will seamlessly roll-over from Part 1 to Part 2, resuming the same cohort as they were assigned in Part 1, and will receive S230815 for a maximum of 72 weeks.
CONDITIONS
Official 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 besides Epilepsy of Infancy with Migrating Focal Seizures or Early-Onset Epileptic Encephalopathy
- Pathogenic or likely pathogenic variants in other genes causing epilepsy identified by prior genetic testing
- Clinically significant medical history or findings unsuitable for study participation, including recent medical conditions, abnormal ECG or lab results such as renal insufficiency or liver problems
- Positive tests for hepatitis B, hepatitis C, or HIV within 6 months prior to screening
- Bone, spine, bleeding disorders, or conditions increasing risk of injury or unsuccessful lumbar puncture
- Contraindications to MRI, lumbar puncture, or intrathecal administration
- History of CNS tumors or malignancies including metastases
- Continuous respiratory support or invasive ventilation including tracheostomy
- Use of quinidine within 30 days prior to screening
- Use or anticipated use of antiplatelet or anticoagulant therapy during the study
- Participation in another interventional clinical trial with investigational therapy within 30 days prior to screening
- Implantable CNS device interfering with lumbar puncture
- Known hypersensitivity to oligonucleotides or other drugs that may prevent study participation
AI-Screening
AI-Powered Screening
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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
Not Yet 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
CONTACT
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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