NCT06422377

A Study Evaluating Soticlestat in Participants With Dravet Syndrome or Lennox-Gastaut Syndrome Who Have Been Exposed to Fenfluramine

An Open-label, Nonrandomized, Phase 3 Study to Evaluate the Efficacy and Safety of Soticlestat in Participants With Dravet Syndrome or Lennox-Gastaut Syndrome Who Have Been Exposed to Fenfluramine

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Takeda · Industry
• Sex: All
• Age: 2 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to check how soticlestat impacts symptoms of Dravet syndrome \[DS\] and Lennox-Gastaut syndrome \[LGS\] in participants who have been exposed to fenfluramine.

Detailed description

The drug being tested in this study is called soticlestat. Soticlestat is being tested to treat people who have DS or LGS and have been exposed to fenfluramine. This study will assess the efficacy and safety of soticlestat in addition to standard care in the treatment of DS or LGS. The study will enroll approximately 45 patients. This study comprises a screening period of up to 6 weeks, a 4-week titration period, a 48-week maintenance period, a taper period of up to 1 week and a follow-up safety visit. Participants will be enrolled to receive soticlestat along with the standard of care: • Soticlestat 100-300 milligrams (mg) Participants will receive oral administration of soticlestat Dose 1 (days 1 to 7), Dose 2 (days 8 to 14), and Dose 3 (Days 15 to 28) with a minimum dose of 100 mg to a maximum dose of 300 mg depending on participant's body weight in the titration period followed by maintenance period up to end of treatment (up to approximately 52 weeks). Percent change from baseline in convulsive in participants with DS and major motor drop (MMD) in participants with LGS seizure frequency per 28 days during the initial 12 weeks of the maintenance period will be assessed. This multi-center trial will be conducted in the United Kingdom and Europe. The overall time to participate in this study is approximately 60 weeks. Participants will make multiple visits to the clinic and will be followed up for safety by visiting the clinic or by telephone approximately 2 weeks after the last dose of the study drug.

Conditions

• Dravet Syndrome (DS)
• Lennox-Gastaut Syndrome (LGS)

Interventions

Type	Name	Description
DRUG	Soticlestat	Soticlestat tablets or mini-tablets

Timeline

• Start date: 2024-04-25
• Primary completion: 2024-08-14
• Completion: 2024-08-14
• First posted: 2024-05-21
• Last updated: 2025-07-29
• Results posted: 2025-03-06

Locations

1 site across 1 country: Denmark

Regulatory

• FDA-regulated drug study