A Study of Soticlestat as an Add-on Therapy in Children, Teenagers, and Adults With Lennox-Gastaut Syndrome

Ведущий спонсор: Takeda

The aims of the study are: - to learn if soticlestat, when given as add-on therapy, reduces the number of major motor drop seizures in children, teenagers, and adults with Lennox-Gastaut Syndrome. - to assess the safety profile of soticlestat when given in combination with other therapies. Participants will receive their standard anti-seizure therapy, plus either tablets of soticlestat or placebo. A placebo looks just like soticlestat but will not have any medicine in it. Participants will take soticlestat or placebo for 16 weeks, followed by a gradual dose reduction for 1 week. Then, participants will be followed up for 2 weeks.

The drug being tested in this study is called soticlestat (TAK-935). Soticlestat will be assessed for efficacy, safety, and tolerability in pediatric and adult participants with Lennox-Gastaut syndrome (LGS). The study will enroll approximately 234 patients. Participants will be randomly assigned (by chance, like flipping a coin) in a 1:1 ratio to receive standard of care (SOC) plus one of the following adjunctive therapies which will remain undisclosed to the participant and study doctor during the study (unless there is an urgent medical need): 1. Soticlestat 2. Placebo (dummy inactive pill - this is a tablet/mini-tablet that looks like the study drug but has no active ingredient) Participants will receive soticlestat or matching placebo based on their weight in the 4-week 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 will then have an option to either enter the Open-label Extension (OLE) Study or discontinue. If participants discontinue, they will be followed-up for safety.

• Lennox Gastaut Syndrome (LGS)

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 Lennox-Gastaut Syndrome (LGS). 2. Has ≥8 major motor drop (MMD) seizures each month in the 3 months prior to Screening based on the historical information and has ≥8 MMD 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 anti-seizure medications (ASMs) based on historical information, and is currently on an anti-seizure therapy or other treatment options considered as standard of care (SOC). 5. Currently taking 0 to 3 ASMs at stable doses for at least 4 weeks before the Screening Visit (Visit 1); ASM dosing regimen must remain constant throughout the study. Exclusion Criteria: 1. Admitted to a medical facility and intubated for treatment of status epilepticus 2 or more times in the 3 months immediately before Screening (Visit 1). Status epilepticus is defined as continuous seizure activity lasting longer than 5 minutes or repeated seizures without return to Baseline in between seizures. 2. 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. 3. Considered by the investigator to be at imminent risk of suicide or injury to self, others, or property, or the participant has attempted suicide within 12 months before the Screening Visit (Visit 1). Participants who have positive answers on item numbers 4 or 5 on the Columbia suicide severity rating scale (C-SSRS) before randomization/dosing (Visit 2) are excluded. This scale will only be administered to participants aged ≥6 years.

Пол:

All

Минимальный возраст:

2 Years

Максимальный возраст:

35 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

2021-10-01

Тип: Sponsor

• Drug therapy

• Lennox Gastaut Syndrome
• 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 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. Total duration of the treatment will be up to 16 weeks (Treatment Period). 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. Total duration of the treatment will be up to 16 weeks (Treatment Period). 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 (Treatment Period). 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)