Fecal Microbiome Transplant National Registry

粪便微生物组移植国家登记处

• 批准号: 9751162
• 负责人: Colleen Renee Kelly
• 金额: $ 60.12万
• 依托单位: AMERICAN GASTROENTEROLOGICAL ASSN/INST
• 依托单位国家: 美国
• 财政年份: 2016
• 资助国家: 美国
• 起止时间: 2016-08-04 至 2021-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9751162
• 关键词: Adoption; Adverse event; American; Animal Model; Archives; Biometry; Bypass; Centers for Disease Control and Prevention (U.S.); Characteristics; Chronic Disease; Clinical; Clinical Data; Clinical effectiveness; Clostridium difficile; Collection; Communities; Conflict of Interest; Consensus; Data; Data Element; Development; Disease; Donor person; Education; Educational workshop; Effectiveness; Effectiveness of Interventions; Electronic Mail; Elements; Enrollment; Ethics; Feces; Foundations; Generations; Goals; Health; Health Promotion; Human; Human Microbiome; Incidence; Infection; Information Distribution; Infrastructure; Institutional Review Boards; Intervention; Investigation; Knowledge; Laboratories; Leadership; Link; Metadata; Methodology; Modality; Monitor; Observational Study; Outcome; Participant; Pathogenesis; Pathway interactions; Patient Education; Patients; Physicians; Prospective cohort; Randomized Controlled Trials; Recurrence; Registries; Reporting; Research; Resources; Risk; Role; Safety; Sampling; Scientific Advances and Accomplishments; Signal Detection Analysis; Site; Source; Standardization; Statistical Data Interpretation; Techniques; Therapeutic; Therapeutic Intervention; Transplant Recipients; United States; United States National Institutes of Health; Work; adverse outcome; base; biobank; clinical application; clinical practice; comparative effectiveness; data registry; discrete data; effective therapy; experience; fecal transplantation; follow-up; gut microbiome; gut microbiota; interest; microbiome research; novel strategies; outreach program; participant safety; patient outreach; prospective; screening; tool; transplant registry

Project Summary Rapidly accumulating evidence suggests that modifying gut microbiota may promote health or treat disease, with fecal microbiota transplantation (FMT) for C. difficile infection (CDI) being the first successful clinical application of this concept. Given the rising incidence of CDI and the lack of effective therapies for recurrent CDI, the use of FMT is growing rapidly, despite a paucity of clinical data and despite the theoretical risk that transfer of gut microbiota may induce chronic diseases. The early adoption and expansion of FMT in clinical practice has bypassed the standard investigatory pathway and preceded large randomized controlled trials that typically evaluate efficacy and contribute important short- and long-term safety data. In order to overcome this deficit in knowledge and experience, we propose to develop a national FMT registry. The goal of this proposal is to collect clinical data from both FMT donors and recipients for the following purposes: 1) To assess short- and long-term safety; 2) to gather information on practice in the U.S. and assess effectiveness of the intervention; 3) to promote scientific investigation; and 4) to aid practitioners and trial sponsors in satisfying regulatory requirements. This registry will prospectively enroll patients who undergo FMT at sites throughout the U.S. Information on FMT methodology, FMT indication, as well as baseline data on donors and recipients will be collected from each site. Patients will be followed at regular intervals for up to 10 years after FMT for pre-defined and spontaneously reported adverse events and for pre-defined outcomes of effectiveness. The registry will facilitate statistical analyses for signal detection, generation of descriptive data, and the conduct of comparative effectiveness studies. A prospective cohort of 4,000 FMT patients will provide precise estimates of the incidence of safety and effectiveness endpoints in real-world practice and rule out uncommon adverse events with high confidence. This registry will link to an independent biobank that will collect, archive, and analyze approximately 3,000 fecal samples from the donors as well as recipients, providing a rich source for studying manipulation of the gut microbiota in humans. The leadership of the American Gastroenterological Association (AGA) and its partner organizations have worked together to develop the plans for this registry, which will be an extremely important tool to assess short-term safety, identify potential long-term consequences, determine effectiveness in real-world practice, help standardize best FMT practice, and gauge the potential consequences of manipulating the gut microbiome in human health and disease.

项目摘要 快速积累的证据表明，改变肠道微生物群可以促进健康或治疗疾病， 粪便微生物群移植（FMT）治疗C.艰难梭菌感染（CDI）是第一个成功的临床应用 这个概念。鉴于CDI的发病率不断上升，并且缺乏有效的治疗复发性CDI的方法， FMT正在迅速发展，尽管缺乏临床数据，尽管理论上存在肠道转移的风险， 微生物群可能诱发慢性疾病。FMT在临床实践中的早期采用和扩展 绕过了标准的治疗途径，在大型随机对照试验之前， 评估疗效并提供重要的短期和长期安全性数据。为了克服这一缺陷， 根据我们的知识和经验，我们建议建立一个国家裂变材料条约登记册。本提案的目的是 收集FMT供者和受者的临床数据，用于以下目的：1）评估短期和长期FMT供者和受者的临床数据。 长期安全性; 2）收集有关美国实践的信息，并评估 干预; 3）促进科学调查; 4）帮助从业人员和试验申办者满足 监管要求本登记研究将前瞻性入组在整个研究中心接受FMT的患者。 美国关于FMT方法学、FMT适应症以及供体和受体的基线数据的信息将在 从每个网站收集。在FMT后，将定期对患者进行随访，最长10年， 和自发报告的不良事件以及预定义的有效性结局。书记官处将 促进信号检测的统计分析，生成描述性数据，并进行比较 有效性研究。4,000例FMT患者的前瞻性队列将提供发病率的精确估计， 安全性和有效性终点，并排除不常见的不良事件， 信心该注册表将链接到一个独立的生物库，该生物库将收集，存档和分析大约 来自捐赠者和接受者的3，000份粪便样本，为研究操纵提供了丰富的来源。 人体肠道菌群。美国胃肠病学协会（阿加）及其 合作伙伴组织共同制定了该登记册的计划，这将是一个极其重要的 评估短期安全性、识别潜在长期后果、确定有效性的重要工具 在现实世界的实践中，帮助标准化最佳FMT实践，并衡量 操纵人类健康和疾病中的肠道微生物组。