A First-in-human Study of S230815 in Pediatric Participants With KCNT1-related Developmental and Epileptic Encephalopathy
Public ClinicalTrials.gov record NCT07227857. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
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 Pediatric Participants With KCNT1-related Developmental and Epileptic Encephalopathy
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT07227857
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 20 participants
Conditions and interventions
Conditions
Interventions
- S230815- Starting dose A Drug
- S230815- Dose B Drug
- S230815- Dose C Drug
- S230815- Dose D Drug
Drug
Eligibility (public fields only)
- Age range
- 2 Years to 12 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Nov 23, 2025
- Primary completion
- Apr 14, 2028
- Completion
- Apr 14, 2028
- Last update posted
- Aug 10, 2026
2025 – 2028
United States locations
- U.S. sites
- 5
- U.S. states
- 5
- U.S. cities
- 5
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Children's Hospital of Orange County | Orange | California | 92868 | Not yet recruiting |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | Recruiting |
| University of Rochester Medical Center | Rochester | New York | 14642 | Not yet recruiting |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | Not yet recruiting |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | Not yet recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 10 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT07227857, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 10, 2026 · Synced Sep 6, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT07227857 live on ClinicalTrials.gov.