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

Recruiting

• Conditions: Cystic Fibrosis
• Interventions: Diagnostic Test: Questionnaire Patient Reported Outcome Measure (PROM)

• 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

Arm && Interventions

Group	Intervention	Description
Patients with CF	Questionnaire Patient Reported Outcome Measure (PROM)	\>2000 in Germany, France, Italy, Spain, Denmark, Belgium, Portugal, GB, Ireland, USA, Australia, Canada, Brazil, Argentina...
Age-matched healthy controls	Questionnaire Patient Reported Outcome Measure (PROM)	\>100 in Germany \>100 in each of the other participating countries

Primary Outcome Measures

Name	Time	Method
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
Development and evaluation of a diary for assessment and quantification of abdominal symptoms, a CF-specific PROM following FDA recommendations	7

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