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
- 105
- 试验地点
- 43
- 主要终点
- Change From Baseline to Week 12 in Mean Sleep Latency From the 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 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 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 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.
详细描述
The drug being tested in this study is called TAK-861. TAK-861 is being tested to evaluate its efficacy and safety in participants with narcolepsy with cataplexy (narcolepsy type 1 [NT1]).
The study will enroll approximately 93 participants. Participants will be randomly assigned (by chance, like flipping a coin) to one of the two treatment groups:
- TAK-861
- 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
- 18 Years 至 70 Years（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 any current unstable psychiatric disorder or current active major depressive episode (MDE) or an active MDE in the past 6 months.
结局指标
主要结局
Change From Baseline to Week 12 in Mean Sleep Latency From the 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 MMRM was used for analysis.
Change From Baseline to Week 12 in ESS Total Score
时间窗: Baseline, Week 12
The ESS provides individuals with 8 different situations of daily life and asks them how likely they are to fall asleep in those situations (scored 0 to 3) and to try to imagine their likelihood of dozing even if they have not actually been in the identical situation; the scores are summed to give an overall score of 0 to 24. Higher scores indicate stronger subjective daytime sleepiness. A negative change from baseline indicates an improvement. The linear mixed effects model for repeated measures (MMRM) was used for analysis.
Change from baseline to Week 12 in ESS total score
Change from baseline to Week 12 in ESS total score
次要结局
- Weekly Cataplexy Rate (WCR) at Week 12(Week 12)
- Change From Baseline to Week 12 in Mean Number of Lapses on the Psychomotor Vigilance Test (PVT)(Baseline, Week 12)
- Proportion of Participants With Improved Patient Global Impression of Change (PGI-C) Score 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)
- Number of Participants With At Least One Treatment-Emergent Adverse Event (TEAE)(Up to 16 weeks)
- Change from baseline to Week 12 in mean sleep latency from the 4 MWT wake trials.
- WCR at Week 12
- Change from baseline to Week 12 in mean number of lapses on the
- PGI-C score at Week 12.
- Change from baseline to Week 12 in NSS-CT total score
- Change from baseline to Week 12 in FINI domain scores for: Tiredness, Cognitive Functioning, Cataplexy, Social Activities, Everyday Activities, and Everyday Responsibilities.
- Change from baseline to Week 12 in SF-36 mental and physical component summary scores
- Occurrence of at least 1 TEAE during the study including the follow-up period, as applicable
研究者
Takeda
Scientific
Takeda Development Center Americas Inc.