A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of TAK-861 for the Treatment of Narcolepsy With Cataplexy (Narcolepsy Type 1)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 168
- 试验地点
- 105
- 主要终点
- Change From Baseline to Week 12 in Mean Sleep Latency From the 4 Maintenance of Wakefulness Test (MWT) Wake Trials
研究概览
简要总结
The main aim of this study is to learn how effective TAK-861 (oveporexton) is in improving excessive sleepiness during the day (called excessive daytime sleepiness or EDS) after 3 months of treatment. Other aims are to learn how effective TAK-861 (oveporexton) is in lowering the number of sudden, unexpected attacks of muscle weakness while staying conscious (cataplexy) in a week; to learn the effect TAK-861 (oveporexton) has on participants' ability to maintain attention, participant's overall quality of life, the spectrum of narcolepsy symptoms, and daily life functions; and to learn about the safety of TAK-861 (oveporexton).
详细描述
The drug being tested in this study is called TAK-861 (oveporexton). TAK-861 (oveporexton) is being tested to evaluate its efficacy and safety in people with narcolepsy type 1 (NT1).
The study will enroll approximately 152 patients. Participants will be randomly assigned (by chance, like flipping a coin) to one of the three treatment groups:
- TAK-861 Dose 1
- TAK-861 Dose 2
- Placebo
The study drug will be administered for 12 weeks. This multi-center trial will be conducted globally.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 16 Years 至 70 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The participant has a body mass index (BMI) within the range 18 to 40 kilograms per meter square (kg/m^2).
- •The participant has an International Classification of Sleep Disorders, Third Edition (ICSD-3) or International Classification of Sleep Disorders, Third Edition, Text Revision (ICSD-3-TR) diagnosis of NT
- •The participant has greater than or equal to (≥)4 partial or complete episodes of cataplexy/week (WCR).
- •The participant is positive for the human leukocyte antigen (HLA) genotype HLA-DQB1*06:02 or results from radioimmunoassay indicate the participant's cerebrospinal fluid (CSF) orexin (OX)/hypocretin-1 concentration is less than or equal to (≤)110 picograms per milliliter (pg/mL) [or less than one-third of the mean values obtained in normal participants within the same standardized assay].
排除标准
- •The participant has a current medical disorder, other than narcolepsy with cataplexy, associated with EDS.
- •The participant: (a) has a history of myocardial infarction; (b) has a history of clinically significant hepatic disease, thyroid disease, coronary artery disease, cardiac rhythm abnormality or heart failure; or (c) has any medical condition (such as unstable cardiovascular, pulmonary, renal or gastrointestinal disease).
- •The participant has current or recent (within 6 months) gastrointestinal disease that is expected to influence the absorption of drugs.
- •The participant has a history of cancer in the past 5 years.
- •The participant has a clinically significant history of head injury or head trauma.
- •The participant has a history of epilepsy, seizure, or convulsion.
- •The participant has a history of cerebral ischemia, transient ischemic attack (<5 years from screening), intracranial aneurysm, or arteriovenous malformation.
研究组 & 干预措施
Placebo
Participants received oveporexton (OVE)-matching placebo tablets, orally, for 12 weeks.
干预措施: Placebo (Drug)
OVE 1 mg BID
Participants received OVE tablets, 1 milligram (mg), orally, for 12 weeks.
干预措施: Oveporexton (Drug)
OVE 2 mg BID
Participants received OVE tablets, 2 mg, orally, for 12 weeks.
干预措施: Oveporexton (Drug)
结局指标
主要结局
Change From Baseline to Week 12 in Mean Sleep Latency From the 4 Maintenance of Wakefulness Test (MWT) Wake Trials
时间窗: Baseline, Week 12
The MWT evaluates a person's ability to remain awake under soporific conditions for a defined period of time. Because there is no biological measure of wakefulness, wakefulness is measured indirectly by the inability or delayed tendency to fall asleep. This tendency to fall asleep is measured via electroencephalography-derived sleep latency in the MWT. The MWT consists of four 40-minute sessions (trials) done 2 hours apart. Sleep latency in each session was recorded. Participants were required to stay awake in between the four sessions. A positive change from baseline indicates an improvement. The linear mixed effects model for repeated measures (MMRM) was used for analysis.
次要结局
- Change From Baseline to Week 12 in ESS Total Score(Baseline, Week 12)
- Change From Baseline to Week 12 in Mean Number of Lapses on the 3 Psychomotor Vigilance Test (PVT)(Baseline, Week 12)
- Patient Global Impression of Change (PGI-C) Score at Week 12(Week 12)
- Number of Participants with At Least one Treatment-Emergent Adverse Event (TEAE)(Up to 16 weeks)
- Weekly Cataplexy Rate (WCR) at Week 12(Week 12)
- Change From Baseline to Week 12 in Narcolepsy Severity Scale for Clinical Trials (NSS-CT) Total Score(Baseline, Week 12)
- Change From Baseline to Week 12 in Functional Impacts of Narcolepsy Instrument (FINI) Domain Scores(Baseline, Week 12)
- Change From Baseline to Week 12 in Short Form-36 Survey (SF-36) Mental and Physical Component Scores(Baseline, Week 12)
- Change From Baseline to Week 12 in Epworth Sleepiness Scale (ESS) Total Score(Baseline, Week 12)
- Change From Baseline to Week 12 in Mean Number of Lapses on the Psychomotor Vigilance Test (PVT)(Baseline, Week 12)
- Responder Rate on Patient Global Impression of Change (PGI-C) Score at Week 12(Week 12)
- Number of Participants With At Least One Treatment-Emergent Adverse Event (TEAE)(Up to 16 weeks)