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

Ведущий спонсор: Takeda

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.

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 Dravet Syndrome (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 Maintenance Period. The dose will then be down-tapered. This multi-center trial will be conducted worldwide. The overall time to participate in the study will be from 22-25 weeks. Participants may have an option to either enter or to discontinue Open-label Extension (OLE). If participants discontinue, they will be followed-up for safety.

• Dravet Syndrome (DS)

Тип вмешательства: Drug

Название вмешательства: Soticlestat

Описание: Soticlestat mini-tablets or tablets.

Этикетка Arm Group: Soticlestat

Другое имя: TAK-935

Тип вмешательства: Drug

Название вмешательства: Placebo

Описание: Soticlestat placebo-matching mini-tablets or tablets.

Этикетка Arm Group: Placebo

Критерии:

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.

Пол:

All

Минимальный возраст:

2 Years

Максимальный возраст:

21 Years

Здоровые волонтеры:

No

Фамилия: Takeda Contact

Телефон: +1-877-825-3327

Australia

Belgium

Canada

China

France

Greece

Hungary

Italy

Japan

Latvia

Netherlands

Poland

Russian Federation

Serbia

Spain

Ukraine

United States

2021-11-01

Тип: Sponsor

• Drug Therapy

• Epilepsies, Myoclonic
• Syndrome

Метка: Soticlestat

Тип: Experimental

Описание: Participants weighing <45 kg: Soticlestat, mini-tablets, at the dose of 40 mg to 200 mg, orally or via gastrostomy tube (G-tube) or percutaneous endoscopic gastrostomy (PEG) tube, twice daily (BID) based on the body weight up to 4 weeks in Titration Period. Participants will continue to receive the dose that they are on at the end of the Titration Period, for 12 weeks in the Maintenance Period. The total duration of the treatment will be up to 16 weeks (Treatment Period). The dose will be tapered down if participants decide to discontinue the treatment. Participants weighing ≥45 kg: Soticlestat mini-tablets or tablets with a starting dose of 100 mg BID followed by 200 mg BID and, then 300 mg BID, up to 4 weeks in Titration Period. Participants will continue to receive 300 mg BID for 12 weeks in the Maintenance Period. The total duration of the treatment will be up to 16 weeks (Treatment Period). The dose will be tapered down if participants decide to discontinue the treatment.

Метка: Placebo

Тип: Placebo Comparator

Описание: Soticlestat placebo-matching mini-tablets or tablets, orally or via G-tube or PEG tube, BID, up to 4 weeks in the Titration Period. Participants will continue to receive the soticlestat placebo-matching mini-tablets or tablets for 12 weeks in the Maintenance Period. The total duration of the treatment will be up to 16 weeks. Soticlestat matching tapering will be done to maintain the blind if participants decide to discontinue the treatment.

Распределение: Randomized

Модель вмешательства: Parallel Assignment

Первичное назначение: Treatment

Маскировка: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)