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Recruiting NCT03052283

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

— Observational Cystic Fibrosis

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What is being studied
The protocol lists: Questionnaire Patient Reported Outcome Measure (PROM).
Who it may be relevant to
Registry conditions: Cystic Fibrosis. Basic parameters: No limits · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Germany
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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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)

Overview

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

Interventions

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

Primary outcome measures

  • Development and evaluation of the CFAbd-Score (initially named JenAbdomen CF-Score), a CF-specific PROM following FDA recommendations [Time frame: 7 years]
  • Development and evaluation of a diary for assessment and quantification of abdominal symptoms, a CF-specific PROM following FDA recommendations [Time frame: 7]
Secondary outcome measures (3)
  • Abdominal symptoms in cystic fibrosis and their relation to genotype, history, clinical and laboratory findings [Time frame: 7 years]
  • Assessing the Relation of Ultrasound Findings and Abdominal Symptoms obtained with the CFAbd-Score in Cystic Fibrosis Patients [Time frame: 3 years]
  • Effects of a new therapy with CFTR-modulator on abdominal symtoms, quantified with the CFAbd-Score [Time frame: 7 years]

Eligibility criteria

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.)

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: Yes

Study design

Observational model
Case-control

Study locations

Germany · 2 centers
  • Klinikum Westbrandenburg — Brandenburg an der Havel
  • Medizinische Hochschule Brandenburg (MHB), University — Brandenburg an der Havel

Publications

  • 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):e0174463. doi: 10.1371/journal.pone.0174463. eCollection 2017. PMID 28472055
  • 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/s41598-017-17302-4. PMID 29234058
  • 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). Patient. 2019 Aug;12(4):419-428. doi: 10.1007/s40271-019-00361-2. PMID 30887269
  • 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):355-360. doi: 10.1097/MPG.0000000000003357. PMID 34789668
  • 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 Fibrosis-Early results Obtained With the CF-Specific CFAbd-Score. Front Pharmacol. 2022 Jun 3;13:877118. doi: 10.3389/fphar.2022.877118. eCollection 2 PMID 35721187
  • 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 Mar;22(2):275-281. doi: 10.1016/j.jcf.2023.01.010. Epub 2023 Jan 27. PMID 36710099
  • Sadrieh P, Bechinger L, Duckstein F, Barucha A, Nahrlich L, Eickmeier O, van Dullemen S, Kemp D, Muhl B, Eschenhagen P, Schwarz C, Graepler-Mainka U, Zagoya C, Mainz JG. Validation of the CFAbd-Score.kidⒸ, a Novel gastrointestinal patient reported outcome measure, specific for children with cystic fibrosis. J Cyst Fibros. 2025 Sep;24(5):886-893. doi: 10.1016/j.jcf.2025.07.010. Epub 2025 Jul 23. PMID 40701924

Identifiers

NCT: NCT03052283 · CFAbd-Score · CFAbd-Score.kid · CFAbd-day2day

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗