Orri Miguel, Lipset Craig H, Jacobs Bradly P, Costello Anthony J, Cummings Steven R
Pfizer Ltd, Tadworth, United Kingdom.
Pfizer Inc, New York, NY, USA.
Contemp Clin Trials. 2014 Jul;38(2):190-7. doi: 10.1016/j.cct.2014.04.009. Epub 2014 May 2.
Participatory patient-centered, web-based methods could streamline and improve the convenience of clinical trial participation. We used an entirely web-based approach to conduct a randomized, placebo-controlled, Phase 4 (REMOTE) trial under an Investigational New Drug (IND) application to evaluate tolterodine extended release (ER) 4 mg for overactive bladder.
The trial was designed to replicate previous clinic-based trials of tolterodine ER but was conducted via the web from one clinical site overseen by physicians. Participants were recruited via the web, screened for eligibility using web-based questionnaires, had laboratory testing in their community, and entered a run-in phase requiring bladder e-diaries. Informed consent was obtained using an interactive web-based method with physician countersignature. Study medication was shipped directly to participants.
With a goal of 283 randomized participants, 5157 registered on the trial website. Of 456 who passed initial screening, identification verification, and signed consent, 237 passed additional medical screening and were countersigned by the investigator. After laboratory testing, 118 entered the placebo run-in; only 18 passed e-diary assessments and were randomized to treatment. At week 12, the mean change from the baseline in micturitions/24 hours (primary endpoint) was -2.4 for tolterodine ER versus -0.8 for placebo [treatment difference (95% CI): -1.6 (-3.9, 0.6)].
The REMOTE trial is the first entirely web-based trial conducted under an IND application. The efficacy observed was consistent with results from conventional trials. With simplification of multi-step screening and testing, web-based trials or their component parts should provide a participant-friendly approach to many clinical trials.
以患者为中心的参与式网络方法可以简化并提高临床试验参与的便利性。我们采用完全基于网络的方法，在研究性新药（IND）申请下进行了一项随机、安慰剂对照的4期（远程）试验，以评估托特罗定缓释（ER）4毫克治疗膀胱过度活动症的效果。
该试验旨在复制先前基于诊所的托特罗定ER试验，但通过网络在一个由医生监督的临床站点进行。通过网络招募参与者，使用基于网络的问卷进行资格筛选，在其社区进行实验室检测，并进入需要膀胱电子日记的导入期。使用交互式网络方法并由医生会签获得知情同意。研究药物直接运送给参与者。
目标是招募283名随机参与者，有5157人在试验网站注册。在456名通过初始筛选、身份验证并签署同意书的人中，237人通过了额外的医学筛查并由研究者会签。经过实验室检测后，118人进入安慰剂导入期；只有18人通过电子日记评估并被随机分配接受治疗。在第12周时，托特罗定ER组每24小时排尿次数（主要终点）较基线的平均变化为-2.4，而安慰剂组为-0.8 [治疗差异（95% CI）：-1.6（-3.9，0.6）]。
远程试验是在IND申请下进行的首个完全基于网络的试验。观察到的疗效与传统试验结果一致。通过简化多步骤筛查和检测，基于网络技术的试验或其组成部分应为许多临床试验提供一种方便参与者的方法。
