NCT04940624

A Study of Soticlestat as an Add-on Therapy in Children and Young Adults With Dravet Syndrome

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)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 144 (actual)
• Sponsor: Takeda · Industry
• Sex: All
• Age: 2 Years – 21 Years
• Healthy volunteers: Not accepted

Summary

The main aim of the study is to learn if soticlestat, when given as an add-on therapy, reduces the number of convulsive seizures in children and young adults with DS. Participants will receive their standard antiseizure therapy, plus either a tablet of soticlestat or placebo for 16 weeks. A placebo looks just like soticlestat but will not have any medicine in it. Participants may continue treatment in an extension study, based on the extension study's entry criteria. Those that want to stop treatment will have a gradual dose reduction during 1 week and then be followed up for 2 weeks.

Detailed description

The drug being tested in this study is called soticlestat (TAK-935). Soticlestat as an adjunctive therapy will be assessed for efficacy, safety, and tolerability in pediatric and adult participants with DS. The study will enroll approximately 142 pediatric and young adult patients. Participants will be randomized at a 1:1 ratio to receive standard of care (SOC) plus one of the following adjunctive therapies: * Soticlestat or * Placebo The total daily dose of study drug will be calculated based on body weight in the 4 weeks Titration Period. Following the Titration Period, participants will continue to receive the same dose in the 12-weeks Maintenance Period. This multi-center trial will be conducted worldwide. The overall time to participate in the study will be from 22-25 weeks. At the end of the Full Treatment Period, participants have the option to either complete the study and taper off the investigational product or to enter the OLE if they meet eligibility requirements. If participants discontinue, they will be followed-up on phone call approximately 14 days after the last dose of study drug for safety.

Conditions

• Dravet Syndrome (DS)

Interventions

Type	Name	Description
DRUG	Soticlestat	Soticlestat mini-tablets or tablets.
DRUG	Placebo	Soticlestat placebo-matching mini-tablets or tablets.

Timeline

• Start date: 2021-10-28
• Primary completion: 2024-04-11
• Completion: 2024-04-11
• First posted: 2021-06-25
• Last updated: 2025-01-01
• Results posted: 2025-01-01

Locations

66 sites across 18 countries: United States, Australia, Brazil, Canada, China, France, Germany, Greece, Hungary, Italy, Japan, Latvia, Netherlands, Poland, Russia, Serbia, Spain, Ukraine

Regulatory

• FDA-regulated drug study