Study of Soticlestat as Adjunctive Therapy in Pediatric & Young Adult Subjects With Dravet Syndrome
Research type
Research Study
Full title
A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy, Safety, and Tolerability of Soticlestat as Adjunctive Therapy in Pediatric and Young Adult Subjects With Dravet Syndrome (DS)
IRAS ID
1003743
Contact name
Lucy Castro-Manrique
Contact email
Sponsor organisation
Takeda Development Center Americas, Inc.
Eudract number
2021-002480-22
Research summary
Dravet Syndrome (DS) is a rare and often genetic neurological condition that is typically characterised by multiple, frequent & prolonged seizures in children and young adults. The condition starts in infancy with signs & symptoms such as frequent seizures caused by a fever at onset followed by seizures that last longer than 5 minutes. Over time DS may cause behavioural & mental impairment. Currently a significant proportion of patients with DS are not receiving adequate seizure control with current anti-seizure medications & there remains an unmet need for new therapies. The study aims to evaluate the efficacy, safety & tolerability of Soticlestat as an add-on medication to the patient's current standard of care therapies. Soticlestat works by reducing the activity of an enzyme called cholesterol-24-hydroxylase in the brain, it is thought that the product of this enzyme may play a part in disorders such as epilepsy. The study will start with a screening period of 4-6weeks, where eligible participants will be randomised into one of two groups: either receiving soticlestat or placebo. There will be a 50/50 chance of either treatment. The study will last around 25weeks, including 4-6weeks of screening period, 4weeks dose adjustment and 12weeks maintenance followed by 1week tapering and a 2week safety follow-up visit. Certain study visits will be carried out using remote options such as telephone & video calls. Soticlestat dose will be calculated based on the participant’s body weight. Each dose will be given twice daily. All participants will complete the same procedures & questionnaires including patient-reported outcomes, physical & neurological examinations, vital signs, blood & urine collection, eye tests & ECG. Participants will be evaluated on their frequency & reduction of seizures over the full treatment period. The study is sponsored by Takeda Development Center Americas, Inc. Approx 142 participants globally will take part in the study.
REC name
London - Westminster Research Ethics Committee
REC reference
21/LO/0546
Date of REC Opinion
22 Sep 2021
REC opinion
Unfavourable Opinion