Nichols Deborah A, Steininger Teresa L, Fuller Douglas S, Kirby M Todd, Barker Emily C, Whalen Marisa, Alexander Jessica K, Akerman Sarah, Plante David T
Jazz Pharmaceuticals, 3170 Porter Drive, Palo Alto, CA, 94304, USA.
Jazz Pharmaceuticals, Philadelphia, PA, USA.
Neurol Ther. 2025 Jun 9. doi: 10.1007/s40120-025-00745-7.
Low-sodium oxybate (LXB; Xywav) is approved to treat idiopathic hypersomnia in adults and excessive daytime sleepiness or cataplexy in individuals aged ≥ 7 years with narcolepsy. The efficacy and safety of LXB have been demonstrated in randomized controlled trials. This study will comprehensively evaluate multiple daytime and nighttime symptoms in participants with idiopathic hypersomnia and participants with narcolepsy treated with LXB.
Jazz DUET (Develop hypersomnia Understanding by Evaluating low-sodium oxybate Treatment; NCT05875974) is a prospective, multi-cohort, multicenter, single-arm, open-label, interventional study. Establishing multiple cohorts across different diseases aligns with more generalizable research designs enabling a broader impact. Total study duration is ~ 10-21 weeks, which includes a 2- to 6-week screening period, an 8-day baseline period, a 2- to 8-week titration period, a 2-week stable-dose period, a 1- to 2-week end-of-treatment assessment period, and a 2-week safety follow-up period. To provide a robust dataset of changes with LXB treatment which will inform healthcare providers and their patients, DUET is administering a wide range of patient- and clinician-reported outcome assessments regarding symptom severity and daytime functioning and includes objective measures of sleep (ad libitum polysomnography protocol) and sleep inertia which have not been previously tested with this type of study design. Additionally, pharmacokinetics data and clinician titration feedback are collected to inform titration/dosing guidance for clinicians. DUET was designed in a patient-centric manner to reflect a real-world approach to conducting clinical trials.
Using a patient-centric design aiming to address participants' burden and improve their study experience, the DUET study will fill critical idiopathic hypersomnia and narcolepsy evidence gaps pertaining to sleep architecture (e.g., disrupted nighttime sleep) and response to LXB treatment, as well as provide data on outcomes that are meaningful to patients. Graphical abstract available for this article.
低钠羟丁酸钠（LXB；Xywav）已被批准用于治疗成人特发性嗜睡症，以及治疗年龄≥7岁发作性睡病患者的日间过度嗜睡或猝倒。LXB的疗效和安全性已在随机对照试验中得到证实。本研究将全面评估接受LXB治疗的特发性嗜睡症患者和发作性睡病患者的多种日间和夜间症状。
Jazz DUET（通过评估低钠羟丁酸钠治疗来加深对嗜睡症的理解；NCT05875974）是一项前瞻性、多队列、多中心、单臂、开放标签的干预性研究。在不同疾病中建立多个队列符合更具普遍性的研究设计，能够产生更广泛的影响。研究总时长约为10至21周，包括2至6周的筛查期、8天的基线期、2至8周的滴定期、2周的稳定剂量期、1至2周的治疗结束评估期以及2周的安全性随访期。为了提供关于LXB治疗变化的强大数据集，以便为医疗服务提供者及其患者提供信息，DUET正在进行一系列广泛的患者和临床医生报告的结局评估，涉及症状严重程度和日间功能，并包括睡眠（自由睡眠多导睡眠图方案）和睡眠惯性的客观测量，这些测量此前尚未在这种研究设计中进行过测试。此外，还收集药代动力学数据和临床医生滴定反馈，以为临床医生提供滴定/给药指导。DUET以患者为中心进行设计，以反映开展临床试验的真实世界方法。
DUET研究采用以患者为中心的设计，旨在减轻参与者的负担并改善他们的研究体验，将填补与睡眠结构（如夜间睡眠中断）和对LXB治疗的反应相关的关键特发性嗜睡症和发作性睡病证据空白，并提供对患者有意义的结局数据。本文提供图形摘要。
