Clinical trial NCT03052283: Development and Validation of a Disease Specific PROM to Assess Abdominal Involvement in Patients With CF (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)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Cystic Fibrosis
Keywords: Cystic fibrosis
Abdomen
Pancreas
Liver
Gut
Symptom Score
Patient reported outcome measure (PROM)
diabetes
CFTR-modulator
Tricafta
Kaftrio
Lead sponsor: Medizinische Hochschule Brandenburg Theodor Fontane
Sponsor class: OTHER
Collaborators: name: University of Jena
First posted: 2017-02-14
Enrollment: 2000
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: CASE_CONTROL
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with CF of all ages

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

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.)

Primary outcomes:
measure: Development and evaluation of the CFAbd-Score (initially named JenAbdomen CF-Score), a CF-specific PROM following FDA recommendations
timeFrame: 7 years
measure: Development and evaluation of a diary for assessment and quantification of abdominal symptoms, a CF-specific PROM following FDA recommendations
timeFrame: 7
Secondary outcomes:
measure: Abdominal symptoms in cystic fibrosis and their relation to genotype, history, clinical and laboratory findings
timeFrame: 7 years
measure: Assessing the Relation of Ultrasound Findings and Abdominal Symptoms obtained with the CFAbd-Score in Cystic Fibrosis Patients
timeFrame: 3 years
measure: Effects of a new therapy with CFTR-modulator on abdominal symtoms, quantified with the CFAbd-Score
timeFrame: 7 years
Other outcomes:
Locations:
city: Brandenburg an der Havel
country: Germany
facility: Klinikum Westbrandenburg
city: Brandenburg an der Havel
country: Germany
facility: Medizinische Hochschule Brandenburg (MHB), University
Study officials:
affiliation: Medizinische Hochschule Brandenburg (MHB), University
name: Jochen G Mainz, Prof. MD
role: PRINCIPAL_INVESTIGATOR
References:
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):e0174463. doi: 10.1371/journal.pone.0174463. eCollection 2017.
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/s41598-017-17302-4.
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). Patient. 2019 Aug;12(4):419-428. doi: 10.1007/s40271-019-00361-2.
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):355-360. doi: 10.1097/MPG.0000000000003357.
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 Fibrosis-Early results Obtained With the CF-Specific CFAbd-Score. Front Pharmacol. 2022 Jun 3;13:877118. doi: 10.3389/fphar.2022.877118. eCollection 2022.
citation: 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.