S230815 for Epileptic Encephalopathy

(KANDLE Trial)

Recruiting at 12 trial locations

Overseen By Institut de Recherches Internationales Servier

Age: < 18

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Institut de Recherches Internationales Servier

Must not be taking: Quinidine, Antiplatelets, Anticoagulants

Disqualifiers: Other epilepsy genes, Renal insufficiency, Hepatic derangement, Positive hepatitis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

20 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called S230815 (an experimental treatment) to determine its safety and effectiveness for children with a type of epilepsy linked to the KCNT1 gene. The goal is to observe how the body reacts to different doses and understand its long-term effects. Children aged 2 to 12 with genetically confirmed Developmental Epileptic Encephalopathy, who are on stable medication, may qualify to participate. The trial consists of two parts: one to test various doses and another for long-term observation. As a Phase 1 and Phase 2 trial, this research aims to understand how the treatment works in people and measure its effectiveness in an initial, smaller group, offering participants a chance to contribute to groundbreaking research.

Is there any evidence suggesting that S230815 is likely to be safe for humans?

Research has shown that the drug S230815 is being tested in children with KCNT1-related Developmental Epileptic Encephalopathy. This drug is in its early stages, undergoing its first tests in humans. Consequently, information on its tolerability is limited. However, early studies focus on ensuring participant safety by closely monitoring side effects or unexpected reactions.

As this is a new treatment, doctors proceed with caution. They administer the drug in gradually increasing doses to observe its effects closely. This step-by-step approach helps identify potential risks early. Although there is no clear data yet on patient responses, ongoing research aims to ensure safety and understand how the treatment functions in the body. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for epileptic encephalopathy, like anticonvulsant medications, aim to control seizures but often don't address underlying causes. S230815 is unique because it targets specific pathways in the brain that are believed to contribute to the disorder, potentially offering a more direct approach to managing symptoms. Researchers are excited about S230815 because it could pave the way for more effective treatments that not only reduce seizures but also improve cognitive and developmental outcomes, which current options struggle to achieve.

What evidence suggests that S230815 might be an effective treatment for Epileptic Encephalopathy?

Research has shown that S230815 is under investigation for its potential to address KCNT1-related Developmental Epileptic Encephalopathy, a condition that causes severe seizures due to a specific genetic change. S230815 targets the KCNT1 gene, which controls electrical signals in the brain. By focusing on this gene, the treatment aims to reduce the frequency and severity of seizures. Although detailed information on S230815's effectiveness in humans is still being gathered, its targeted approach appears promising based on the drug's mechanism. Early results from similar treatments suggest it might help manage seizures. Participants in this trial will join one of several cohorts to evaluate the effects of S230815. ^{1 3 6 7 8}

Are You a Good Fit for This Trial?

This trial is for children aged 2-12 with a confirmed genetic diagnosis of Developmental Epileptic Encephalopathy (DEE) due to KCNT1 mutations. They must be on stable doses of other medications or seizure interventions like ketogenic diet or vagal nerve stimulation.

Inclusion Criteria

I am a child aged 2-12 with DEE confirmed by genetic testing.

My current medications or seizure treatments have been stable.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment Part 1

Evaluation of multiple ascending doses of S230815

24 weeks

Regular visits for dose escalation and monitoring

Treatment Part 2

Long-term treatment extension for participants who completed Part 1

72 weeks

Regular visits for continued treatment

Follow-up

Participants are monitored for safety and effectiveness after treatment

20 weeks

Periodic visits for monitoring

What Are the Treatments Tested in This Trial?

Interventions

• S230815

Trial Overview

The study tests multiple ascending doses of S230815 in pediatric patients with DEE related to KCNT1. It's an open-label, first-in-human trial assessing safety, tolerability, and how the body processes the drug over two parts: initial dosing and a long-term treatment extension up to 72 weeks.

How Is the Trial Designed?

Group I: Cohort 4 Experimental Treatment 1 Intervention

Group II: Cohort 3 Experimental Treatment 1 Intervention

Group III: Cohort 2 Experimental Treatment 1 Intervention

Group IV: Cohort 1 Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Institut de Recherches Internationales Servier

Lead Sponsor

Trials

91

Recruited

67,100+

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07227857

A First-in-human Study of S230815 in Pediatric Participants ...

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, ...

2.

clinicaltrials.servier.com

clinicaltrials.servier.com/trial/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 ...

This study aims to test a new drug called S230815 in children with a condition called KCNT1-related Developmental and Epileptic Encephalopathy (DEE). KCNT1- ...

3.

clinicaltrials.eu

clinicaltrials.eu/disease/epileptic-encephalopathy/epileptic-encephalopathy-trials-in-disease/

Epileptic encephalopathy – Trials in Disease

Study of intrathecal S230815 to evaluate safety and effectiveness in children with KCNT1-related Developmental and Epileptic Encephalopathy. This study is ...

4.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/39254637/

N-of-1 trials in epilepsy: A systematic review and lessons ...

The studies enrolled individuals with focal seizures, absences or cognitive impairement and electrographic discharges. Treatments included ...

5.

clinicaltrials.eu

clinicaltrials.eu/disease/epileptic-encephalopathy/epileptic-encephalopathy-life-with-disease/

Epileptic encephalopathy – Life with Disease

Study of intrathecal S230815 to evaluate safety and effectiveness in children with KCNT1-related Developmental and Epileptic Encephalopathy. Recruiting.

6.

clinicaltrials.eu

clinicaltrials.eu/trial/study-of-intrathecal-s230815-to-evaluate-safety-and-effectiveness-in-children-with-kcnt1-related-developmental-and-epileptic-encephalopathy/

Study of intrathecal S230815 to evaluate safety and ...

Study of intrathecal S230815 to evaluate safety and effectiveness in children with KCNT1-related Developmental and Epileptic Encephalopathy.

7.

synapse.patsnap.com

synapse.patsnap.com/drug/536a01a279bc2fac57fb2c1388ec47f3

S-230815 - Drug Targets, Indications, Patents

A Phase Ib/II First-in-human, Multicentre, Open-label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, ...

8.

ctis.eu

ctis.eu/search/trial/2024-513332-17-00.html

2024-513332-17-00 - Clinical Trials Search - CTIS.eu

To evaluate the safety and tolerability of S230815 in paediatric participants with KCNT1-Developmental and Epileptic Encephalopathy (KCNT1-DEE). 1.2 Overall ...