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

Sponsor

Medizinische Hochschule Brandenburg Theodor Fontane (Other)

Overall Status

Recruiting

CT.gov ID

NCT03052283

Collaborator

University of Jena (Other)

2,000

Enrollment

Locations

131.1

Anticipated Duration (Months)

1000

Patients Per Site

7.6

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis	Diagnostic Test: Questionnaire Patient Reported Outcome Measure (PROM)

Detailed Description

Development of a multimodal questionnaire considering the FDA guidelines for development of a PROM

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)

Study Design

Study Type:

Observational

Anticipated Enrollment :

2000 participants

Observational Model:

Case-Control

Time Perspective:

Prospective

Official Title:

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)

Actual Study Start Date :

Jan 26, 2017

Anticipated Primary Completion Date :

Dec 31, 2026

Anticipated Study Completion Date :

Dec 31, 2027

Arms and Interventions

Arm	Intervention/Treatment
Patients with CF >2000 in Germany, France, Italy, Spain, Denmark, Belgium, Portugal, GB, Ireland, USA, Australia, Canada, Brazil, Argentina...	Diagnostic Test: Questionnaire Patient Reported Outcome Measure (PROM) non-interventional Other Names: routine-care assessments
Age-matched healthy controls >100 in Germany >100 in each of the other participating countries	Diagnostic Test: Questionnaire Patient Reported Outcome Measure (PROM) non-interventional Other Names: routine-care assessments

Arm

Intervention/Treatment

Patients with CF

>2000 in Germany, France, Italy, Spain, Denmark, Belgium, Portugal, GB, Ireland, USA, Australia, Canada, Brazil, Argentina...

Diagnostic Test: Questionnaire Patient Reported Outcome Measure (PROM)

non-interventional

Other Names:

routine-care assessments

Age-matched healthy controls

>100 in Germany >100 in each of the other participating countries

Diagnostic Test: Questionnaire Patient Reported Outcome Measure (PROM)

non-interventional

Other Names:

routine-care assessments

Outcome Measures

Primary Outcome Measures

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

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Yes

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 (alpha 1 antitrypsin deficiency, celiac disease, food allergy, gastroenteritis, etc.)

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Klinikum Westbrandenburg	Brandenburg an der Havel		Germany	14770
2	Medizinische Hochschule Brandenburg (MHB), University	Brandenburg an der Havel		Germany	14770