Planning Grant for SOD III Multicenter Trial

描述(由申请人提供)： 在美国，每年有超过50万例胆囊切除手术，其中10%-20%的患者在手术后出现持续或复发的疼痛。影像和实验室检查正常的患者通常被怀疑患有III型Oddi括约肌功能障碍(SODIII型)，但很难评估。Oddi括约肌测压(SOM)用于诊断，但不广泛使用，不完全准确，可导致胰腺炎。超氧化物歧化酶III(胆管和/或胰腺括约肌的内窥镜消融术)的常规治疗有可能危及生命的并发症。内窥镜下将肉毒杆菌毒素(Botox)注射到括约肌区域可以降低压力长达6个月，这可能是一种很好的治疗试验。R03计划拨款的目的是为NIH为疑似SOD III患者进行的大型III期随机多中心临床试验准备R01期随机多中心临床试验所需的组织和拨款编写活动提供支持。申请资金用于支持以下活动：敲定方案、病例报告表、数据管理计划和操作手册；招募和认证更多的研究地点；准备培训时间表和材料；开发消化系统疾病疼痛负担问卷(DDPBQ)并最终确定仪器验证的规划；建立委员会成员并确定独立的医疗监督员；以及为SOD III多中心试验编写NIH R01补助金。与超氧化物歧化酶III相关的性质使其独特地适合于这样的研究。第三阶段的SOD随机多中心临床试验将确定：1)如果SOD III确实存在，2)如果可以通过任一临床参数检测到SOD III，那么对括约肌内注射肉毒杆菌、SOM或可能的组合的临时反应，3)哪种检测SOD III的策略最具成本效益，4)新的消化系统疾病疼痛负担问卷(DDPBQ)是否有助于评估这些患者以及他们的治疗反应。这次审判将回答专家小组在1月份举行的NIH ERCP科学状况会议上提出的几个重要问题。

DESCRIPTION (provided by applicant): More than half a million cholecystectomies are performed annually in the United States, and 10-20% of these patients present afterwards with continuing or recurrent pains. Patients with normal imaging and laboratory findings are often suspected of suffering from Sphincter of Oddi dysfunction Type III (SOD III), but are difficult to evaluate. Sphincter of Oddi manometry (SOM) is used for diagnosis, but is not widely available, not completely accurate, and can cause pancreatitis. The usual treatment for SOD III (endoscopic ablation of the biliary and/or pancreatic sphincters) has complications which can be life-threatening. Endoscopic injection of Botulinum toxin (Botox) into the sphincter zone reduces the pressure for up to 6 months, and may be a good therapeutic trial. The objective of this R03 Planning Grant is to provide support to carry out the organization and grant-writing activities needed to prepare an R01 NIH application for a major Phase III Randomized Multicenter Clinical Trial in patients with suspected SOD III. Funding is requested to support the following activities: finalize the protocol, case report forms, Data Management Plan, and Manual of Operations; recruit and certify additional study sites; prepare training schedules and materials; develop the Digestive Disease Pain-Burden Questionnaire (DDPBQ) and finalize the planning for the validation of the instrument; establish membership of committees and identify independent medical monitor; and write the NIH R01 grant for the SOD III Multicenter Trial. The properties associated with SOD III render it uniquely suited for such an investigation. The Phase III SOD Randomized Multicenter Clinical Trial will determine: 1) if SOD III really exists, 2) if SOD III can be detected by either clinical parameters, the temporary response to intra-sphincteric injection of Botox, SOM, or a possible combination, 3) which strategy for detection of SOD III is most cost-effective, and 4) if the new Digestive Disease Pain-Burden Questionnaire (DDPBQ) is useful in evaluating these patients, and their responses to treatment. This trial will answer several of the important questions raised by the expert panel at the NIH State of the Science of ERCP Conference, held in January.