Planning Grant for SOD III Multicenter Trial

点击翻译按钮获取中文摘要

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.