A Multicenter, Open-label, Pilot Study of TAK-935 (OV935) in Patients With 15Q Duplication Syndrome or CDKL5 Deficiency Disorder (ARCADE Study)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 20
- 试验地点
- 16
- 主要终点
- Percent Change From Baseline in Motor Seizure Frequency Per 28 Days During the Maintenance Period
研究概览
简要总结
The purpose of this study is to investigate the effect of soticlestat on the frequency of motor seizures for participants with Dup15q or CDD during the Maintenance Period.
详细描述
The drug being tested in this study is called soticlestat. Soticlestat is being tested to treat people with Dup 15q or CDD. This study will assess the effects of TAK-935 on seizure frequency, safety.
The study will enroll approximately 30 participants. Participants will be enrolled into 2 groups based on their diagnosis as: Dup 15q or CDD.
All participants will be asked to take soticlestat tablets twice daily with or without food.
The study comprises of 2 periods: Screening/Baseline Period and Treatment Period (Dose Optimization and Maintenance). The overall time to participate in this study is approximately 30 weeks, including 4 to 6 weeks Screening/Baseline Period, 20 weeks Treatment Period, 2 weeks Taper, and 2 weeks safety follow up period. Participants completing this study will have an option to enroll in the open-label extension (OLE) study, under a separate protocol.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 55 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of Dup 15q or CDKL5 deficiency disorder.
- •Currently taking 1 to 6 antiepileptic drugs (AEDs) at a stable dose.
排除标准
- •Two or more episodes of convulsive status epilepticus per 3 months requiring hospitalization and intubation.
- •Currently receiving a study drug or participated in a clinical study involving another investigational product in the previous month.
研究组 & 干预措施
Soticlestat Dup 15q
Soticlestat tablets twice daily (BID) orally or via gastrostomy tube (G-tube)/ percutaneous endoscopic gastrostomy (PEG) tube, BID. Participants with Dup 15q weighing <60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
干预措施: Soticlestat (Drug)
Soticlestat CDD
Soticlestat tablets BID orally or via G-tube/ PEG tube, BID. Participants with CDD weighing <60 kg at Baseline received total daily dose of study drug calculated based on body weight. Participants weighing ≥60 kg at Baseline, were administered with 200 mg/day followed by 400 mg/day, then 600 mg/day, up to Week 20.
干预措施: Soticlestat (Drug)
结局指标
主要结局
Percent Change From Baseline in Motor Seizure Frequency Per 28 Days During the Maintenance Period
时间窗: Maintenance Period: Weeks 9 to 20
Seizure frequency per 28 days is defined as total number of Seizures reported during the period divided by number of days with no missing seizure count during the period multiplied by 28. Percent Change from Baseline is defined as (frequency of seizures per 28 days during maintenance period - frequency of seizures per 28 days at Baseline) divided by frequency of seizures per 28 days at Baseline multiplied by 100. Positive percent change from Baseline indicates seizure increase and negative percent change from Baseline indicates seizure decrease.
次要结局
- Percent Change From Baseline in Motor Seizure Frequency Per 28 Days During the Treatment Period(Treatment Period: Weeks 0 to 20)
- Percentage of Participants Considered as Treatment Responders During the Maintenance Period(Maintenance Period: Weeks 9 to 20)
- Percent Change From Baseline in Frequency of Motor Seizures Longer Than 5 Minutes in Participants With CDD(Treatment Period: Weeks 0 to 20)
- Proportion of Motor Seizure-free Days in Participants During the Maintenance Period(Maintenance Period: Weeks 9 to 20)
- Change From Baseline in Clinician's Global Impression of Severity (CGI-S) Responses of Investigator(Baseline to Week 20)
- Percentage of Participants With Clinical Global Impression of Change (CGI-C) Responses as Per the Investigator Reported Impression(Week 20)
- Percentage of Participants With Care Clinical Global Impression of Change (CGI-C) Responses of Parent/Family(Week 20)
- Change From Baseline of Plasma 24S-hydroxycholesterol (24HC) Levels(Baseline to Week 20)
- Change From Baseline in Seizure Frequency in Participants Treated With TAK-935 as an Adjunctive Therapy(Baseline to Week 20)