NCT02221869已完成3 期
A Multicenter Study of the Efficacy and Safety of Xyrem With an Open- Label Pharmacokinetic Evaluation and Safety Extension in Pediatric Subjects With Narcolepsy With Cataplexy
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 106
- 试验地点
- 20
- 主要终点
- Change in Weekly Number of Cataplexy Attacks
研究概览
简要总结
The purpose of this trial is to assess the efficacy and safety of Xyrem in pediatrics subjects with narcolepsy that includes 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
- 7 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female subjects aged 7-16 years at Visit 2 for subjects on Xyrem at study entry and at Visit 1.1 for Xyrem-naïve subjects (to ensure subjects are <18 years of age at the end of the study)
- •Have a primary diagnosis of narcolepsy with cataplexy that meets International Classification of Sleep Disorders (ICSD)-2 or ICSD-3 criteria, whichever was in effect at the time of the diagnosis or, with the permission of the Medical Monitor, completes a Multiple Sleep Latency Test (MSLT) during Screening to confirm the diagnosis of Type 1 narcolepsy by ICSD-3 criteria (i.e., the subject meets all other ICSD-3 criteria for Type 1 narcolepsy)
- •Have given documented assent indicating that he/she was aware of the investigational nature of the study and the required procedures and restrictions before participation in any protocol-related activities
- •Have parent(s)/guardian(s) who have given informed consent for his/her/their child's participation in the study
- •Be willing to spend the required number of nights (2 to 3) in a sleep laboratory for PSG evaluations
- •If currently treated with Xyrem, must have been taking unchanged doses (twice nightly dosing no higher than 9 g/night) of Xyrem, and stimulants, if applicable, for the treatment of narcolepsy symptoms for at least 2 months prior to screening
- •In addition to the above inclusion criteria, subjects participating in the PK evaluation must meet the following inclusion criteria:
- •Be willing to spend 2 additional nights in the clinic for PK evaluation
排除标准
- •Inability to understand assent or follow study instructions for any reason, in the opinion of the Investigator
- •Parent(s) or guardian(s) unable to comply with the requirements of the study for any reason, in the opinion of the Investigator
- •Other documented clinically significant condition (including an unstable medical condition, chronic disease other than narcolepsy with cataplexy, or history or presence of another neurological disorder) that might affect the subject's safety and/or interfere with the conduct of the study in the opinion of the Investigator
- •Treatment with benzodiazepines, non-benzodiazepine anxiolytics/ hypnotics/sedatives, neuroleptics, opioids, barbiturates, diclofenac, valproate, phenytoin, ethosuximide within 2 weeks prior to enrollment (discontinuation for the purpose of study enrollment is permitted only if considered safe by the Investigator and approved by the Medical Monitor)
- •Treatment with any other medications that have anticataplectic effect (e.g., serotonin-norepinephrine reuptake inhibitors [SNRIs], selective serotonin reuptake inhibitors [SSRIs], or tricyclic antidepressants [TCAs]) within 1 month before Screening
- •Unsafe for the subject to receive placebo treatment for 2 weeks, in the opinion of the Investigator
- •In addition to the above exclusion criteria, subjects participating in the PK evaluation must not demonstrate the following:
研究组 & 干预措施
Xyrem
Experimental
Active Xyrem at a dose ≤9 g/night
干预措施: Xyrem (Drug)
Xyrem Placebo
Placebo Comparator
Xyrem placebo at a volume and regimen equivalent to the stable dose of Xyrem.
干预措施: Xyrem (Drug)
结局指标
主要结局
Change in Weekly Number of Cataplexy Attacks
时间窗: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)
Double-blind comparison of the change in weekly number of cataplexy attacks from the last 2 weeks of the Stable Dose Period to the 2 weeks of the Double-blind Treatment Period.
次要结局
- Change in the Epworth Sleepiness Scale (ESS) (CHAD) Score(From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks))
- Change in Quality of Life (QoL; SF-10 Physical and Psychosocial Summary Score) From the End of the Stable Dose Period to the End of the Double-blind Treatment Period(From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks))
- Clinical Global Impression of Change (CGIc) for Cataplexy Severity(From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks))
- CGIc for Narcolepsy Overall(From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks))
研究者
研究点 (20)
Loading locations...
相似试验
已完成
3 期
A Safety and Efficacy Study of Xyrem® (Sodium Oxybate) to Treat Fibromyalgia.FibromyalgiaNCT00423813Jazz Pharmaceuticals573
已完成
4 期
A Study of the Cypher SES to Treat Restenotic Native Coronary Artery Lesions.Coronary Artery DiseaseNCT00232856Cordis Corporation162
已完成
3 期
NEXUS Study for the Treatment of de Novo Native Coronary Artery LesionsCoronary Artery DiseaseNCT00231283Cordis Corporation100
已完成
3 期
A Safety and Efficacy Study of Xyrem® in Subjects With FibromyalgiaFibromyalgiaNCT00371137Jazz Pharmaceuticals548
已完成
2 期
Trial Comparing the Effects of Xyrem (Sodium Oxybate) With Placebo for the Treatment of FibromyalgiaFibromyalgiaNCT00087555Jazz Pharmaceuticals195