NCT07227857

A First-in-human Study of S230815 in Pediatric Participants With KCNT1-related Developmental and Epileptic Encephalopathy

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Institut de Recherches Internationales Servier · Academic / Other
• Sex: All
• Age: 2 Years – 12 Years
• Healthy volunteers: Not accepted

Summary

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

• Epileptic Encephalopathy

Interventions

Type	Name	Description
DRUG	S230815- Starting dose A	Solution for injection
DRUG	S230815- Dose B	Solution for injection
DRUG	S230815- Dose C	Solution for injection
DRUG	S230815- Dose D	Solution for injection

Timeline

• Start date: 2025-11-24
• Primary completion: 2028-04-15
• Completion: 2028-04-15
• First posted: 2025-11-13
• Last updated: 2026-02-11

Locations

16 sites across 5 countries: United States, France, Italy, Japan, Spain

Regulatory

• FDA-regulated drug study