Evaluation and Validation of a New Questionnaire to Identify and Quantify Abdominal Symptoms in Patients With Cystic Fibrosis Following FDA Guidelines: the CF Abd-Score (Primarily Named JenAbdomen-CF Score)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 2,000
- 试验地点
- 2
- 主要终点
- Development and evaluation of a diary for assessment and quantification of abdominal symptoms, a CF-specific PROM following FDA recommendations
研究概览
简要总结
Development, validation and evaluation of a new multimodal questionnaire to assess and quantify the abdominal involvement in patients with the inherited life shortening multi-organ disease Cystic fibrosis.
In the stepwhise development of the patient reported outcome measures (PROM) repeadedly, CF-patients, their families as well as professionals working in different fields of CF-care will be included.
详细描述
Development of a multimodal questionnaire considering the FDA guidelines for development of a PROM, the CFAbd-Score
- Evaluation of reliability of the questionnaire by examining internal consistency and construct validity
- Evaluation of reproducibility of the questionnaire by re-testing of patients
- Assessment of cross-generational applicability of the questionnaire by sub-scoring of age groups
- Evaluation of the responsiveness of the score by comparison with age-matched healthy controls
- Assessment of putative relationships of abdominal symptoms with faecal inflammatory markers
- Assessment of applicability of the questionnaire in other CF centers
- Transfer the CFAbd-Score to other countries and languages
Development and validation of a CF-specific tool for daily assessment of abdominal symptoms (patient diary), the CFAbd-day2day
Development and validation of a CF-specific tool for assessment of abdominal symptoms in children < 12 years of age, the CFAbd-kid
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Diagnosis of CF determined by
- •a sweat chloride of >60 mEq/L and/or
- •detection of 2 disease causing CFTR mutations with evidence of organ involvement.
- •Healthy controls:
- •Age-matched
排除标准
- •Lacking ability to cooperate of patients /their families, respectively
- •Healthy controls:
- •Other diseases causing abdominal symptoms (Inflammatory bowel disease (IBD), alpha 1 antitrypsin deficiency, celiac disease, food allergy, gastroenteritis, etc.)
结局指标
主要结局
Development and evaluation of a diary for assessment and quantification of abdominal symptoms, a CF-specific PROM following FDA recommendations
时间窗: 7
Development and evaluation of the CFAbd-Score (initially named JenAbdomen CF-Score), a CF-specific PROM following FDA recommendations
时间窗: 7 years
Evaluation and validation of the CFAbd-Score based on a CF patient-reported outcome measure (PROM) that includes all relevant gastrointestinal symptoms and their impact on subjective quality of life
次要结局
- Abdominal symptoms in cystic fibrosis and their relation to genotype, history, clinical and laboratory findings(7 years)
- Assessing the Relation of Ultrasound Findings and Abdominal Symptoms obtained with the CFAbd-Score in Cystic Fibrosis Patients(3 years)
- Effects of a new therapy with CFTR-modulator on abdominal symtoms, quantified with the CFAbd-Score(7 years)
研究者
Prof. Dr. Jochen G. Mainz
Director of the Cystic Fibrosis-Center
Medizinische Hochschule Brandenburg Theodor Fontane