Continuation of the NIDDK Gastroparesis Consortium

NIDDK 胃轻瘫联盟的延续

• 批准号: 8730618
• 负责人: Richard Warwick McCallum
• 金额: $ 46.63万
• 依托单位: TEXAS TECH UNIVERSITY HEALTH SCIS CENTER
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-09-30 至 2016-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8730618
• 关键词: Abdominal Pain; Address; Adverse effects; Affect; Antiemetics; Asses; Chronic; Clinical; Clinical Data; Clinical Research; Diagnosis; Disease; Domperidone; Dopamine Antagonists; Dose; Double-Blind Method; Etiology; FDA approved; Failure; Gastric Emptying; Gastroparesis; Goals; Knowledge; Lead; Malnutrition; Methods; Metoclopramide; Multicenter Studies; National Institute of Diabetes and Digestive and Kidney Diseases; Nausea and Vomiting; Nutritional Support; Outcome; Pathogenesis; Patients; Phase; Physicians; Property; Protocols documentation; Public Health; Quality of life; Registries; Research; Risk; Safety; Satiation; Series; Stomach; Therapy Clinical Trials; Time; Treatment Efficacy; clinical care; diabetic; follow-up; improved; meetings; novel therapeutics; placebo controlled study; randomized trial; response; young woman

DESCRIPTION (provided by applicant): Gastroparesis (GP) is a disorder of gastric motilityfunction characterized by a delay in gastric emptying, frequently associated with chronic nausea and vomiting, early satiety, postprandial fullness, abdominal pain, and malnutrition that may require nutritional support. GP has a devastating impact on quality of life and predominantly affects younger women. In the past 5 years, the Gastroparesis Clinical Research Consortium (GpCRC) has made a series of important contributions to improve our understanding of this disorder and these observations have advenced the goals of helping our patients and their physicians as well as meeting the goals to improve the management of gastroparesis. We therefore propose the following specific aims for this application: # 1: To complete the ongoing and approved studies initiated by the GpCRC; # 2: To maintain, expand, and refine the GP Registry, and to address important questions related to the pathogenesis, complications and clinical outcome in GP patients by further in-depth analysis of GpR clinical data and biosamples; # 3: To conduct new multicenter studies of gastroparesis investigating the etiology, pathogenesis, diagnosis, and treatment options. TTUHSC is proposing a double-blind, "time- to- failure" placebo controlled trial (phase 1) of 4 months duration with "a best clinical care" follow up (phase 2) of 8 months duration study to evaluate the efficacy and safety of domperidone in gastroparesis. The principal objectives of this multicenter, randomized trial are to:1) Document the efficacy of domperidone in GP patients; 2) Compare domperidone's efficacy in diabetics vs. idiopathic etiologies;3) Investigate the optimal dose or doses; 4) Explore the demographic and clinical factors which predict the best clinical response; 5) Asses safety. Unlike the FDA approved prokinetic agent, metoclopramide, which is also a dopamine antagonist, with antiemetic and prokinetic properties, domperidone has an exellent safety profile for long- term administration. Due to the very restricted accessibility of domperidone in US (through the FDA protocol) many questions related to its efficacy, therapeutic dosing and side effects still remain unanswered. Therefore, we have proposed this study to resolve these questions.

描述（由申请人提供）：胃轻瘫（GP）是一种胃运动功能障碍，其特征是胃排空延迟，经常与慢性恶心和呕吐，早期饱腹感，餐后饱满，腹痛，腹痛和营养不良，可能需要营养支持。 GP对生活质量有毁灭性的影响，主要影响年轻女性。在过去的5年中，胃轻瘫临床研究联盟（GPCRC）为改善我们对这种疾病的理解做出了一系列重要贡献，这些观察结果提高了帮助我们的患者及其医生的目标，并实现了改善胃手术管理的目标。因此，我们提出了以下针对此应用的特定目标：＃1：完成GPCRC发起的正在进行的和批准的研究； ＃2：通过对GPR临床数据和生物样本进行进一步分析，维持，扩展和完善GP注册表，并解决与GP患者的发病机理，并发症和临床结果有关的重要问题； ＃3：进行新的多中心研究，研究病因，发病机理，诊断和治疗方案。 TTUHSC提出了一项双盲，“时间 - 失败”安慰剂对照试验（第1阶段），为4个月的持续时间，“最佳临床护理”随访（第2阶段）持续时间研究，以评估多内替酮在胃轻瘫中的疗效和安全性。该多中心随机试验的主要目标是：1）记录Domperidone在GP患者中的功效； 2）比较多内替酮在糖尿病患者中的功效与特发性病因； 3）研究最佳剂量或剂量； 4）探索预测最佳临床反应的人口统计学和临床​​因素； 5）安全。与FDA批准的原始剂Metocopramide（也是多巴胺拮抗剂）具有抗抑制剂和原始特性不同，多佩酮具有长期施用的明显安全性。由于Domperidone在美国（通过FDA协议）的可及性非常有限，许多与其功效有关的问题，治疗给药和副作用仍然没有得到解答。因此，我们提出了这项研究来解决这些问题。