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

项目摘要 PROMIS小儿克罗恩病简表-疲劳将用作疲劳的临床指标， 在临床试验中检查用于治疗克罗恩病的药物的益处。 该指标拟用作使用药理学方法的研究中的主要或协同主要终点。 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CD中症状负担的常见临床标志物， 人口拟定的目标人群是诊断为克罗恩病（CD）的儿童和青少年。 PROMIS小儿克罗恩病简表-疲劳将用于评估疲劳变化 （与对照相比）由药物治疗引起的。该仪器可用于儿童8岁至 17岁，任何性别、人种或种族以及任何基线症状严重程度，前提是受试者 能够理解并用英语自我报告。 为该项目提议的主要活动是： 1.使用FDA反馈修订并重新提交PROMIS小儿克罗恩病的鉴定计划 疾病简表-疲劳。 2.使用最近完成的一项 PEPR Consortium研究是联合收割机随机对照试验（NCT 02772965）的一部分。 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小儿克罗恩病简表的完整资格文件包 - 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.