A Study Evaluating Soticlestat in Participants Who Have Been Exposed to Fenfluramine

• Research type

  Research Study

• Full title

  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.

• IRAS ID

  1008170

• Contact name

  Yasir Khan

• Contact email

  yasir.khan@takeda.com

• Sponsor organisation

  Takeda Development Centre Americas Inc.

• Research summary

  Epilepsy is a condition in the brain that causes seizures. Dravet Syndrome (DS) and Lennox-Gastaut Syndrome
  (LGS) are rare epileptic conditions that start at an early age. In addition to ongoing seizures, children with DS
  and LGS develop learning and behavioural difficulties over time. The seizures continue in adulthood and are
  difficult to control. Soticlestat is a potential new treatment to control seizures in children and adults with DS and LGS.

  This study, sponsored by Takeda Development Centre Americas, is to find out about soticlestat as an additional therapy when given to children and adults with DS and LGS who are taking or have taken fenfluramine. Fenfluramine is a medicine, recently approved for treatment of DS in the UK, that is eventually expected to be used as standard of care to control seizures in people with both DS and LGS.

  This is an open-label study which means that participants know they are being receiving soticlestat. Patients aged 2 and over can take part in this study; 45 worldwide with about 14 at 6 hospitals in the UK.

  All participants will receive soticlestat twice daily, either by mouth or by tube feeding for about a year. Participants will start on a low dose of soticlestat which will slowly increase during the first 4 weeks of treatment. Participants will stay on their last dose of soticlestat for the next 48 weeks.

  During the study, participants will attend the study site a few times and blood samples will be taken. They
  will have physical and neurological examinations at most visits, and complete several questionnaires about their well-being, behaviour, quality of life, daily life and symptoms, and risk of suicide. They will have vital signs recorded at clinic visits. They will need to keep a paper diary about their seizures throughout their time in the study.

• REC name

  Wales REC 5

• REC reference

  23/WA/0232

• Date of REC Opinion

  30 Nov 2023

• REC opinion

  Further Information Favourable Opinion