Development of the Full Qualification Package for the Pediatric PROMIS Fatigue Instrument for Children with Crohn's Disease

项目总结 普罗米斯儿科克罗恩病简写--疲劳--将作为临床测量疲劳的一种方法。 对克罗恩病患儿进行临床试验，检查用于治疗克罗恩病的药物的益处。 在使用药理学的研究中，该方法被建议用作主要或共同主要终点 改善与CD相关的疲劳的干预措施，并作为药物试验的次要终点 CD的总体症状负担，因为疲劳是本病症状负担的常见临床标志 人口。拟议的目标人群是被诊断为克罗恩病(CD)的儿童和青少年。 将使用Promis儿科克罗恩病简写-疲劳来评估疲劳的变化 (与对照相比)由医疗引起的。该仪器可供8岁以下儿童使用 17岁，任何性别、种族或民族和任何基线症状严重程度，只要参与者是 能够理解并能用英语进行自我报告。 该项目拟开展的主要活动包括： 1.使用FDA的反馈修改并重新提交PROMIS儿科克罗恩病的资格计划 疾病简称--疲劳。 2.使用最近完成的COA期间收集的数据评估建议COA的响应性 作为联合随机对照试验(NCT02772965)的一部分的PEPR联盟研究。 3.起草一份用户手册，其中包括易于使用的评分说明和评分解释指南。 4.起草并提交PROMIS儿科克罗恩病的完整资格包 -疲劳。

PROJECT SUMMARY The PROMIS Pediatric Crohn's Disease Short Form - Fatigue will be used as a clinical measure of fatigue in children with Crohn's disease in clinical trials to examine the benefit of drugs used to treat Crohn's disease. The measure is proposed to be used as a primary or co-primary endpoint in studies that use pharmacological interventions to improve the fatigue associated with CD and as a secondary endpoint in drug trials that reduce the overall symptom burden of CD, as fatigue is a common clinical marker of symptom burden in this population. The proposed target population is children and adolescents diagnosed with Crohn's Disease (CD). The PROMIS Pediatric Crohn's Disease Short Form – Fatigue will be used to assess the change in fatigue (compared to a control) induced by a medical treatment. The instrument may be used with children 8 years to 17 years old of any gender, race, or ethnicity and any baseline symptom severity, provided the participant is able to understand and self-report in English. The primary activities proposed for this project are: 1. Use FDA feedback to revise and resubmit the Qualification Plan for the PROMIS Pediatric Crohn's Disease Short Form - Fatigue. 2. Evaluate the responsiveness of the proposed COA using data collected during a recently completed PEPR Consortium study as part of the COMBINE randomized controlled trial (NCT02772965). 3. Draft a user manual that includes easy-to-use scoring instructions and score interpretation guidance. 4. Draft and submit the Full Qualification Package for the PROMIS Pediatric Crohn's Disease Short Form - Fatigue.