Consortium of Eosinophilic Gastrointestinal Disease Researchers

嗜酸性粒细胞胃肠病研究人员协会

• 批准号: 9803035
• 负责人: Marc E. Rothenberg
• 金额: $ 177.07万
• 依托单位: CINCINNATI CHILDRENS HOSP MED CTR
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-08-15 至 2024-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9803035
• 关键词: Address; Adult; Allergic; American; Arkansas; Biological Markers; California; Childhood; Clinical; Clinical Data; Clinical Research; Clinical Trials; Collaborations; Colorado; Common Data Element; Consensus; Controlled Clinical Trials; Data; Diagnosis; Diagnostic; Disease; Eating; Education; Enrollment; Ensure; Eosinophilic Colitis; Eosinophilic Esophagitis; Eosinophilic Gastritis; Faculty; Feasibility Studies; Focus Groups; Food; Fostering; Foundations; Future; Gastroenterology; Gastrointestinal Diseases; General Population; Geographic Locations; Geography; Goals; Grant; Health Personnel; Hypersensitivity; Illinois; Immunity; Immunology; Individual; Inflammatory; Institution; Interleukin-13; International; Leadership; Maryland; Massachusetts; Mediating; Mentors; Mentorship; Minnesota; Mission; Multicenter Trials; Nature; New York; North Carolina; Ohio; Outcome; Pathogenesis; Pathology; Patient Care; Patient-Focused Outcomes; Patients; Pennsylvania; Personal Satisfaction; Phenotype; Positioning Attribute; Precision therapeutics; Productivity; Professional Organizations; Rare Diseases; Readiness; Research; Research Design; Research Personnel; Research Training; Resource Sharing; Resources; Role; Scientific Advances and Accomplishments; Scientist; Site; Specimen; Standardization; Technology; Tennessee; Testing; Texas; Therapeutic; Tissues; Translational Research; Treatment outcome; United States National Institutes of Health; Utah; Work; base; biobank; career; career development; clinically relevant; cohort; disease natural history; eosinophil; eosinophilic gastroenteritis; experience; gastrointestinal; genotyped patients; improved; innovation; longitudinal analysis; medical specialties; next generation; novel; novel strategies; nutrition; outreach; patient advocacy group; patient population; patient stratification; personalized medicine; pre-doctoral; predictive marker; success; tool; treatment optimization; web site; working group

Project Summary (Overall) This renewal application aims to continue research on rare eosinophilic gastrointestinal diseases with a focus on eosinophilic esophagitis (EoE), eosinophilic gastritis (EG), eosinophilic gastroenteritis (EGE), and eosinophilic colitis (EC) through the Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR). CEGIR will promote highly collaborative, multi-site, patient-centric, translational, and clinical research with the intent of addressing unmet clinical trial readiness needs. CEGIR comprises clinical centers in Ohio (coordinating center), Colorado, Arkansas, California, Illinois, Maryland, Massachusetts, Minnesota, New York, North Carolina, Pennsylvania, Tennessee, Texas, and Utah. The CEGIR sites have been carefully chosen on the basis of their pre-existing record of collaboration and success with each other before and during the first cycle of CEGIR; diverse expertise in relevant clinical specialties including Gastroenterology, Allergy, Immunology, Nutrition, and Pathology; the ability to integrate pediatric and adult patients into a common consortium; well-established record of excellence in clinical and translational research involving this patient population; and excellent mentorship experience and capacity to impact early stage trainees’ career development. CEGIR sites have proven access to an adequate number of patients with EoE, EG, EGE, and EC, as evidenced from completion of several controlled clinical trials involving these patients. Proactive patient advocacy groups (PAGs), including the American Partnership for Eosinophilic Disorders and the Campaign Urging Research for Eosinophilic Disease, partner synergistically in the context of CEGIR. Though still in a relatively early stage, CEGIR is demonstrating success via its collaborative interactions between investigators (at all stages of career development including newly established promising faculty who represent the next generation of thought leaders), highly engaged PAGs, and partnering institutions and has emerged into the leading research group focused on rare EoE, EG, EGE, and EC. Current and proposed CEGIR initiatives are developing clinical trial readiness for this set of rare diseases, such as the establishment of deeply phenotyped and genotyped patients who are ready to participate in research and emerging tools that quantify clinical states, endoscopic abnormalities, tissue pathology, and patient well-being. In this renewal application, investigators plan to study an already developed observational cohort of patients, providing a valuable opportunity for longitudinal analyses. Data emerging from this cohort has already substantiated the association of type 2 allergic immunity (e.g. IL-13) in the pathogenesis of EG, leading to a proposal, described herein, to examine the impact of a promising new anti-type 2 immunity therapeutic (dupilumab, anti-IL-4Ra). CEGIR includes transformative Pilot-Feasibility and Career Enhancement Cores based on collaboration across and outside of the RDCRN and which will collectively test novel hypotheses, particularly focused on enhancing clinical trial readiness and attracting early stage investigators for careers in rare disease research.

项目摘要（总体） 这种更新的应用旨在继续研究罕见的嗜酸性胃肠道疾病，重点 在嗜酸性静脉炎（EOE），嗜酸性胃炎（EG），嗜酸性粒细胞胃炎（EGE）和嗜酸性粒细胞上 结肠炎（EC）通过嗜酸性胃肠道疾病研究人员（CEGIR）的联盟（EC）。 Cegir Will 促进高度协作，多站点，以患者为中心，翻译和临床研究，目的 解决未满足的临床试验准备需求。 Cegir包括俄亥俄州（协调中心）的临床中心， 科罗拉多州，阿肯色州，加利福尼亚，伊利诺伊州，马里兰州，马萨诸塞州，明尼苏达州，纽约，北卡罗来纳州， 宾夕法尼亚州，田纳西州，德克萨斯州和犹他州。 Cegir站点是根据其基础仔细选择的 在Cegir的第一个周期之前和期间，彼此之间的协作和成功的记录； 潜水员在相关临床专业方面的专业知识，包括胃肠病学，过敏，免疫学，营养和 病理;将小儿和成年患者整合到一个共同财团中的能力；成熟的记录 涉及该患者人群的临床和翻译研究卓越；和出色的心态 经验和影响早期学员职业发展的能力。 Cegir网站已证明访问 对于EOE，EG，EGE和EC的足够数量 对照临床试验涉及这些患者。主动患者倡导组（PAG），包括 美国嗜酸性疾病的伙伴关系和运动敦促研究嗜酸性疾病， 合作伙伴在Cegir的背景下协同作用。尽管仍然处于相对较早的阶段，但Cegir正在证明 通过调查人员之间的协作互动取得成功（在职业发展的各个阶段，包括 代表下一代思想领袖的新成立的应许教师）高度参与 PAG和合作机构，并已进入领先的研究小组，重点是稀有EOE，例如 EGE和EC。当前和拟议的Cegir计划正在为这组罕见的临床试验准备 疾病，例如准备参与的深层表型和基因型患者 在量化临床状态，内窥镜异常，组织病理学和的研究和新兴工具中 病人的幸福。在此续签应用中，调查人员计划研究已经开发的观察性 一群患者，为纵向分析提供了宝贵的机会。该队列中出现的数据 已经证实了2型过敏性免疫（例如IL-13）在EG发病机理中的关联， 在本文中描述的提案，以检查承诺新的反型2免疫培养基的影响 治疗性（Dupilumab，抗IL-4RA）。 Cegir包括变革性的飞行员可行性和职业增强 基于RDCRN之间和外部协作的核心，该核心将集体测试小说 假设，尤其专注于增强临床试验准备并吸引早期研究人员 用于稀有疾病研究的职业。