Development and Validation of a Disease Specific PROM to Assess Abdominal Involvement in Patients With CF (CFAbd-Score)

Recruiting

• Conditions: Cystic Fibrosis

• Registration Number: NCT03052283
• Lead Sponsor: Medizinische Hochschule Brandenburg Theodor Fontane

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 Timeline

• Study Start: 2017-01-26
• Study First Submitted: 2017-02-09
• Study First Submitted QC: 2017-02-13
• Study First Posted: 2017-02-14
• Status Verified: 2023-03
• Last Update Submitted: 2023-03-31
• Last Update Posted: 2023-04-04
• Primary Completion: 2026-12-31
• Study Completion: 2027-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 2000

Inclusion Criteria

CF cohort:

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

CF cohort:

• 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.)

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Development and evaluation of a diary for assessment and quantification of abdominal symptoms, a CF-specific PROM following FDA recommendations	7
Development and evaluation of the CFAbd-Score (initially named JenAbdomen CF-Score), a CF-specific PROM following FDA recommendations	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 Outcome Measures

Name	Time	Method
Abdominal symptoms in cystic fibrosis and their relation to genotype, history, clinical and laboratory findings	7 years	relating CFAbd-Scores to history, clinical- and laboratory findings
Assessing the Relation of Ultrasound Findings and Abdominal Symptoms obtained with the CFAbd-Score in Cystic Fibrosis Patients	3 years	abdominal ultrasound
Effects of a new therapy with CFTR-modulator on abdominal symtoms, quantified with the CFAbd-Score	7 years	Assessing the CFAbd-Score´s sensitivity to identify and quantify changes due to effective therapeutic interventions, following FDA-guidelines for validation of a PROM

Trial Locations

Locations (2)

Klinikum Westbrandenburg; 🇩🇪 Brandenburg an der Havel, Germany

Medizinische Hochschule Brandenburg (MHB), University; 🇩🇪 Brandenburg an der Havel, Germany