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

Part of paid clinical trials in Orange, California.

Sponsor
Institut de Recherches Internationales Servier
Study ID
NCT07227857
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Epileptic Encephalopathy

Eligibility Criteria

Sex
ALL
Age
2 Years - 12 Years
Healthy Volunteers
Not accepted

Interventions

  • S230815- Starting dose A — DRUG
    Solution for injection
  • S230815- Dose B — DRUG
    Solution for injection
  • S230815- Dose C — DRUG
    Solution for injection
  • S230815- Dose D — DRUG
    Solution for injection

Study Details

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.

Key Dates

Start date
Nov 24, 2025
Status verified
May 2026
Primary completion
Apr 15, 2028
Completion
Apr 15, 2028

Study Design

Enrollment
20 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1
  • Experimental: Cohort 2
  • Experimental: Cohort 3
  • Experimental: Cohort 4

Primary Outcome Measure

Incidence and severity of Adverse Events (AE)'s. [ Time Frame: Through End of study visit (A maximum of 116 weeks) ]

Central Contacts

Locations (6)

FacilityCityStateZIPSite coordinators
Children's Hospital of Orange CountyOrangeCalifornia92868-
Boston Children's HospitalBostonMassachusetts02115
Christelle Achkar
[email protected]
University of Rochester Medical CenterRochesterNew York14642-
Nationwide Children's HospitalColumbusOhio43205-
The Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-
Children's Health DallasDallasTexas75235-

Find similar trials in Orange, CA

By research site
Children's Hospital of Orange County· Orange, CABoston Children's Hospital· Boston, MAUniversity of Rochester Medical Center· Rochester, NYNationwide Children's Hospital· Columbus, OHThe Children's Hospital of Philadelphia· Philadelphia, PAChildren's Health Dallas· Dallas, TX

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