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

Completed · Phase 3 · Has a placebo group

Conditions studied: Dravet Syndrome (DS)

In brief

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.

Key facts

Study ID

NCT04940624

Run by

Takeda

People needed

144

Starts

2021-10-28

Expected to finish

2024-04-11

Last updated by the study team

2025-01-01

Who can join

Age: 2 and older, up to 21. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Has documented clinical diagnosis of DS.
• Had ≥12 convulsive seizures over 12 weeks before screening based on the historical information and has had ≥4 convulsive seizures per 28 days during the 4- to 6-week prospective baseline period.
• Weighs ≥10 kg at the screening visit (Visit 1).
• Failure to control seizures despite appropriate trials of at least 1 ASM based on historical information and is currently on an antiseizure therapy or other treatment options considered as SOC.
• Artisanal cannabidiols are allowed at a stable dose for at least 4 weeks before the screening visit (Visit 1); the dosing regimen and manufacturer should remain constant throughout the study (Artisanal cannabidiols will not be counted as ASMs.).
• Currently taking 0 to 4 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. Fenfluramine and cannabidiol (Epidiolex) are allowed where available and should be counted as an ASM. ASM dosing regimen must remain constant throughout the study.

You may not qualify if…

• 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.

Where it is running

• David Geffen School of Medicine at UCLA — Los Angeles, California, United States
• University of California Benioff Children's Hospital — San Francisco, California, United States
• Clinical Integrative Research Center of Atlanta — Atlanta, Georgia, United States
• University of Iowa Hospitals & Clinics - (CRS) — Iowa City, Iowa, United States
• NYU Comprehensive Epilepsy Center — New York, New York, United States
• University of Toledo — Toledo, Ohio, United States
• Medical University of South Carolina — Charleston, South Carolina, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• Multicare Health System - Mary Bridge Pediatrics — Tacoma, Washington, United States
• Queensland Childrens Hospital — South Brisbane, Queensland, Australia
• Instituto de Neurologia de Curitiba (INC) — Curitiba, Paraná, Brazil
• Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS) — Porto Alegre, Rio Grande do Sul, Brazil
• Universidade de Sao Paulo — São Paulo, Brazil
• Alberta Childrens Hospital — Calgary, Alberta, Canada
• Child and Family Research Institute — Vancouver, British Columbia, Canada
• Hospital For Sick Children — Toronto, Ontario, Canada
• Peking University First Hospital — Beijing, Beijing Municipality, China
• Beijing Children's Hospital,Capital Medical University — Beijing, Beijing Municipality, China
• Children's Hospital of Chongqing Medical University — Chongqing, Chongqing Municipality, China
• The Second Affiliated Hospital of Guangzhou Medical University — Guangzhou, Guangdong, China
• Shenzhen Children's Hospital — Shenzhen, Guangdong, China
• Wuhan Childrens hospital — Wuhan, Hubei, China
• Xiangya Hospital Central South University — Changsha, Hunan, China
• The First Hospital of Jilin University — Changchun, Jilin, China
• Phoenix Childrens Hospital — Phoenix, Arizona, United States

Full record on ClinicalTrials.gov

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