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

Medizinische Hochschule Brandenburg Theodor Fontane

Status: Enrolling

Conditions: Cystic Fibrosis

Treatments: Diagnostic Test: Questionnaire Patient Reported Outcome Measure (PROM)

Study type: Observational

Funder types: Other

Identifiers

NCT03052283

CFAbd-day2day (Other Identifier)

CFAbd-Score

CFAbd-Score.kid (Other Identifier)

Details and patient eligibility

About

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.

Full 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

Enrollment: 2,000 estimated patients

Sex: All

Volunteers: Accepts Healthy Volunteers

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.)

Trial design

2,000 participants in 2 patient groups

Patients with CF

Description:

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

Treatment:

Diagnostic Test: Questionnaire Patient Reported Outcome Measure (PROM)

Age-matched healthy controls

Description:

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

Treatment:

Diagnostic Test: Questionnaire Patient Reported Outcome Measure (PROM)