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

• Status: Recruiting
• Trial end date: 2023-03-05
• Target enrollment: 0
• Participant gender: All

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 Dravet Syndrome.

Participants will receive their standard anti-seizure 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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Takeda

Criteria

Inclusion Criteria:

1. Has documented clinical diagnosis of Dravet Syndrome (DS).

2. Has ≥4 convulsive seizures in each 1-month period in the 3 months before screening based on the historical information and has ≥4 convulsive seizures per 28 days during the 4- to 6-week prospective baseline period.

3. Weighs ≥10 kg at the screening visit (Visit 1).

4. Failure to control seizures despite appropriate trials of at least 2 ASMs based on historical information and is currently on an antiseizure therapy or other treatment options considered as SOC.

5. Currently taking 0 to 4 antiseizure medication (ASMs) at stable doses for at least 4 weeks before the screening visit (Visit 1); benzodiazepines used chronically (daily) to treat seizures are considered ASMs. ASM dosing regimen must remain constant throughout the study.

Exclusion Criteria:

1. Unstable, clinically significant neurologic (other than the disease being studied), psychiatric, cardiovascular, ophthalmologic, pulmonary, hepatic, renal, metabolic, gastrointestinal, urologic, immunologic, hematopoietic, endocrine disease, malignancy including progressive tumors, or other abnormality that may impact the ability to participate in the study or that may potentially confound the study results. It is the responsibility of the investigator to assess the clinical significance; however, consultation with the medical monitor may be warranted.