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Home » Find Clinical Trials » 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

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

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Servier Protocol Code: CL1-230815-001 EU Trial (CTIS) Number: 2024-513332-17-00

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How to participate in this study
If you think you are eligible for this study (see
eligibility criteria
below), you can identify the location closest to you and contact them directly. If you can’t find a location close to you, please contact Institut de Recherches Internationales Servier (I.R.I.S.)
Name: Institut De Recherches Internationales Servier IRIS
Phone number: +33155726000
The study has 7 locations

Study description

This study aims to test a new drug called S230815 in children with a condition called KCNT1-related Developmental and Epileptic Encephalopathy (DEE). KCNT1-related DEE is a group of severe epilepsies that cause frequent seizures and slow down a child’s development. It is caused by changes in the KCNT1 gene, which helps control brain activity. The changes in the KCNT1 gene cause too much electrical activity in the brain, resulting in seizures. Studies in animals found that S230815, the new study drug, acts by blocking KCNT1 activity. Researchers believe that S230815 may help reduce seizures in children with KCNT1-related DEE by targeting KCNT1. This study is important because it could lead to a treatment option for children with this condition.

Official title: 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 paediatric participants with KCNT1-related Developmental and Epileptic Encephalopathy
Protocol
Conditions
KCNT1-related Developmental and Epileptic Encephalopathy DEE
Interventions / Treatments
  • S230815
Other study id numbers
  • CL1-230815-001

Eligibility Criteria

Eligible age for the study

(Child)

Sex

Male/Female

Accepts Healthy Volunteers

No

To take part in the study, participants have to:

  • Be a child aged 2 to 12 years old.
  • Have confirmed KCNT1-related DEE.
  • Have frequent seizures.
  • Be on a stable dose of their current anti-seizure medication and/or any other treatment before starting the study.
  • Be able to undergo several lumbar puncture procedures, which involve inserting a needle into the lower back to access the spinal fluid.

Participants will not be able to join the study if:

  • They have another type of epilepsy related to the KCNT1 gene.
  • They have changes in other genes that cause epilepsy.
  • They have an implantable device in their brain or spinal cord that may affect the ability to perform study procedures, such as lumbar puncture or brain scans. Implantable devices are a treatment option for people with epilepsy.

How is the study designed?

Allocation
N/A
Interventional study model
Sequential
Participant Group / Arm
Experimental: S230815 (Dose Finding Phase)

The dose finding phase is split into two periods: the treatment period and the follow-up period.

Intervention / Treatment
Drug: S230815

During the treatment period, a given dose of S230815 will be tested in a small group of participants.

Participant Group / Arm
Experimental: S230815 (Long-term Treatment Phase)
Intervention / Treatment
Drug: S230815

Participants who complete Part 1 will continue to receive S230815 until the end of the study.