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)
Trial Snapshot
- Phase
- Not Applicable
- Status
- Recruiting
- Sponsor
- Enrollment
- 2,000
- Locations
- 2
- Primary Endpoint
- Development and evaluation of a diary for assessment and quantification of abdominal symptoms, a CF-specific PROM following FDA recommendations
Study Overview
Brief Summary
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.
Detailed Description
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
Study Design
- Study Type
- Observational
- Observational Model
- Case Control
- Time Perspective
- Prospective
Eligibility Criteria
- Sex
- All
- Accepts Healthy Volunteers
- Yes
Inclusion Criteria
- •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
Exclusion Criteria
- •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.)
Outcomes
Primary Outcomes
Development and evaluation of a diary for assessment and quantification of abdominal symptoms, a CF-specific PROM following FDA recommendations
Time Frame: 7
Development and evaluation of the CFAbd-Score (initially named JenAbdomen CF-Score), a CF-specific PROM following FDA recommendations
Time Frame: 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
Secondary Outcomes
- 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)
Investigators
Prof. Dr. Jochen G. Mainz
Director of the Cystic Fibrosis-Center
Medizinische Hochschule Brandenburg Theodor Fontane