NCT02611687已完成3 期
Double Blind, Multicentre, Randomized, Placebo-controlled Trial to Evaluate Safety and Efficacy of Pitolisant in Children From 6 to Less Than 18 Years With Narcolepsy With/Without Cataplexy, Followed by a Prolonged Open-label Period
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Bioprojet
- 入组人数
- 110
- 试验地点
- 11
- 主要终点
- To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale Score.
研究概览
简要总结
The purpose of this multicenter double blind study is to assess efficacy and safety of Pitolisant versus placebo in paediatric Narcoleptic patients with or without cataplexy.
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
- 6 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female children from 6 to less than 18 years of age suffering from narcolepsy with or without cataplexy - ICSD-3 criteria (narcolepsy type 1 and 2).
- •Patients should be free of non-authorized medication, in particular psychostimulant treatments as from the screening visit onwards.
- •Parents - and patients old enough to understand who have expressed a willingness to participate in the study, who have signed and dated the informed consent form prior to beginning protocol required procedures.
- •In the opinion of the investigator, the patient must have adequate support to comply with the entire study requirements as described in the protocol (e.g., transportation to and from trial site, self rating scales and diaries completion, drug compliance, scheduled visits, tests).
排除标准
- •Any other conditions that can be considered the primary causes of EDS.
- •Cataplectic patients treated by anticataplectics which are not under a stable treatment at the time of inclusion.
- •Patients treated for cataplexy or any other pathology, by tricyclic antidepressants.
- •Any significant abnormality of the electrocardiogram and particularly Fridericia's QTc interval.
- •Patients with significant abnormality or clinical laboratory results.
- •Psychiatric and neurological disorders in the investigator's opinion, would preclude the patient's participation and completion of this trial or comprise reliable representation of subjective symptoms.
- •Active clinically significant illness, including unstable cardiovascular, endocrine, neoplastic, gastrointestinal, haematological, hepatic, immunologic, metabolic, neurological (other than narcolepsy/cataplexy), pulmonary, and/or renal disease which could interfere with the study conduct or counter-indicate the study treatments or place the patient at risk during the trial or compromise the study objectives.
研究组 & 干预措施
pitolisant
Experimental
tablet, oral, once a day.
干预措施: pitolisant (Drug)
placebo
Placebo Comparator
tablet, oral, once a day.
干预措施: Placebo (Drug)
结局指标
主要结局
To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale Score.
时间窗: 8 weeks
Changes in EDS measured by the Ullanlinna Narcolepsy Scale Score (UNS) from baseline (mean of two pre-treatment measures) to the end of double-blind period (mean of the last two measures). The UNS is an 11-item scale used to measure the intensity and frequency of symptoms of narcolepsy (EDS and cataplexy). The total score varies from 0 to 44, with higher scores denoting greater narcoleptic tendencies.
次要结局
- To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Pediatric Daytime Sleepiness Scale.(8 weeks)
- To Evaluate the Efficacy of Pitolisant in Reducing Residual Excessive Daytime Sleepiness (EDS), Ullanlinna Narcolepsy Scale-Cataplexy Subscore .(8 weeks)
研究者
研究点 (11)
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