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

Cystic Fibrosis Clinical Trial

— CFAbd-Score  

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03052283
• Other study ID #: CFAbd-Score
• Secondary ID: CFAbd-Score.kidC
• Status: Recruiting
• Start date: January 26, 2017
• Est. completion date: December 31, 2027

Study information

• Verified date: March 2023
• Source: Medizinische Hochschule Brandenburg Theodor Fontane
• Contact: Jochen G Mainz, Prof. MD
• Phone: +493381411803
• Email: jochen.mainz[@]mhb-fontane.de
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 2000
• Est. completion date: December 31, 2027
• Est. primary completion date: December 31, 2026
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: N/A and older

Eligibility

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.)

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Diagnostic Test:
• Questionnaire Patient Reported Outcome Measure (PROM)
non-interventional

Locations

Country	Name	City	State
Germany	Klinikum Westbrandenburg	Brandenburg an der Havel
Germany	Medizinische Hochschule Brandenburg (MHB), University	Brandenburg an der Havel

Sponsors (2)

Lead Sponsor	Collaborator
Medizinische Hochschule Brandenburg Theodor Fontane	University of Jena

Country where clinical trial is conducted

Germany, 

References & Publications (6)

Caley LR, Zagoya C, Duckstein F, White H, Shimmin D, Jones AM, Barrett J, Whitehouse JL, Floto RA, Mainz JG, Peckham DG. Diabetes is associated with increased burden of gastrointestinal symptoms in adults with cystic fibrosis. J Cyst Fibros. 2023 Jan 27:S — View Citation

Jaudszus A, Pfeifer E, Lorenz M, Beiersdorf N, Hipler UC, Zagoya C, Mainz JG. Abdominal Symptoms Assessed With the CFAbd-Score are Associated With Intestinal Inflammation in Patients With Cystic Fibrosis. J Pediatr Gastroenterol Nutr. 2022 Mar 1;74(3):355 — View Citation

Jaudszus A, Zeman E, Jans T, Pfeifer E, Tabori H, Arnold C, Michl RK, Lorenz M, Beiersdorf N, Mainz JG. Validity and Reliability of a Novel Multimodal Questionnaire for the Assessment of Abdominal Symptoms in People with Cystic Fibrosis (CFAbd-Score). Pat — View Citation

Mainz JG, Zagoya C, Polte L, Naehrlich L, Sasse L, Eickmeier O, Smaczny C, Barucha A, Bechinger L, Duckstein F, Kurzidim L, Eschenhagen P, Caley L, Peckham D, Schwarz C. Elexacaftor-Tezacaftor-Ivacaftor Treatment Reduces Abdominal Symptoms in Cystic Fibro — View Citation

Tabori H, Arnold C, Jaudszus A, Mentzel HJ, Renz DM, Reinsch S, Lorenz M, Michl R, Gerber A, Lehmann T, Mainz JG. Abdominal symptoms in cystic fibrosis and their relation to genotype, history, clinical and laboratory findings. PLoS One. 2017 May 4;12(5):e — View Citation

Tabori H, Jaudszus A, Arnold C, Mentzel HJ, Lorenz M, Michl RK, Lehmann T, Renz DM, Mainz JG. Relation of Ultrasound Findings and Abdominal Symptoms obtained with the CFAbd-Score in Cystic Fibrosis Patients. Sci Rep. 2017 Dec 12;7(1):17465. doi: 10.1038/s — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Development and evaluation of the CFAbd-Score (initially named JenAbdomen CF-Score), a CF-specific PROM following FDA recommendations	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	7 years
Primary	Development and evaluation of a diary for assessment and quantification of abdominal symptoms, a CF-specific PROM following FDA recommendations		7
Secondary	Abdominal symptoms in cystic fibrosis and their relation to genotype, history, clinical and laboratory findings	relating CFAbd-Scores to history, clinical- and laboratory findings	7 years
Secondary	Assessing the Relation of Ultrasound Findings and Abdominal Symptoms obtained with the CFAbd-Score in Cystic Fibrosis Patients	abdominal ultrasound	3 years
Secondary	Effects of a new therapy with CFTR-modulator on abdominal symtoms, quantified with the CFAbd-Score	Assessing the CFAbd-Score´s sensitivity to identify and quantify changes due to effective therapeutic interventions, following FDA-guidelines for validation of a PROM	7 years

External Links

•   Validity and Reliability of a Novel Multimodal Questionnaire for the Assessment of Abdominal Symptoms in People with Cystic Fibrosis (CFAbd-Score).