Studyof Soticlestat asAdjunctive Therapy in Pediatric and Adult SubjectsWith Lennox-Gastaut 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 Adult Subjects With Lennox-Gastaut Syndrome (LGS)
IRAS ID
1003882
Contact name
Lucy Castro-Manrique
Contact email
Sponsor organisation
Takeda Development Center Americas, Inc.
Eudract number
2021-002481-40
Research summary
Lennox-Gastaut syndrome (LGS) is a rare epilepsy that causes frequent & multiple types of seizures.
The syndrome usually starts at an early age of 1 & 8 years but occasionally it occurs in children who are older than 8 years or even into adulthood.It causes seizures which may cause uncontrollable movements of body parts; a person may suddenly lose muscle tone or muscle strength so the head or body may become unsteady. The seizures may also be linked with loss of consciousness & violent muscle shaking. Currently a significant proportion of patients with LGS are not receiving adequate seizure control with current anti-seizure medications & there remains an unmet medical 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 medication. Soticlestat works by reducing the activity of an enzyme called cholesterol-24-hydroxylase in the brain.
The study starts with a screening period of 4-6 weeks, eligible participants are randomised into one of two groups: either receiving soticlestat or placebo. There is a 50/50 chance of either treatment. The study will last around 25weeks, including 4-6 weeks of screening, 4weeks-dose adjustment & 12weeks maintenance followed by 1week tapering & a 2week safety follow-up visit. Certain study visits will also be performed using remote options such as telephone & video calls. Soticlestat dose is calculated based on the participant's body weight. Each dose will be given twice daily. All participants will complete the same procedures and 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 sponsor is Takeda Development Center Americas, Inc. Approximately 234 participants globally will participate in this study.REC name
London - Westminster Research Ethics Committee
REC reference
21/LO/0547
Date of REC Opinion
24 Sep 2021
REC opinion
Unfavourable Opinion