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RecruitingNCT03052283CFAbd-ScoreUpdated Apr 4, 2023

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

An observational study in Cystic Fibrosis, sponsored by Medizinische Hochschule Brandenburg Theodor Fontane. Recruiting at 2 sites in Germany. Per ClinicalTrials.gov, last updated 2023-04-04.

Sponsored by Medizinische Hochschule Brandenburg Theodor Fontane · Observational

From the registry’s dates

  • Started Jan 2017; still recruiting 9 years 8 months later.
Study type
Observational
Model
Case-control
Time perspective
Prospective
Enrollment
2,000
Sex
All
01

Study 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.

Read the 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

02

Conditions studied

  • Cystic Fibrosis

Keywords

  • Cystic fibrosis
  • Abdomen
  • Pancreas
  • Liver
  • Gut
  • Symptom Score
  • Patient reported outcome measure (PROM)
  • diabetes
  • CFTR-modulator
  • Tricafta
  • Kaftrio
03

In context

Cystic Fibrosis

1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.

This study's planned enrollment of 2,000 is above the median of 85 across 482 observational studies indexed under Cystic Fibrosis.

Browse Cystic Fibrosis studies →

Lead sponsor

Medizinische Hochschule Brandenburg Theodor Fontane is the lead sponsor of 17 studies on the registry; 2 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
Yes
Sampling method
Non-probability sample

Study population

Patients with CF of all ages

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

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.)
05

Study design

Observational model
Case-control
Time perspective
Prospective
Enrollment
2,000 participants (estimated)
Patient registry
No
Biospecimen retention
Samples without dna

Groups and cohorts

  • 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)

  • Age-matched healthy controls

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

    Diagnostic Test: Questionnaire Patient Reported Outcome Measure (PROM)

Interventions

  • Diagnostic testQuestionnaire Patient Reported Outcome Measure (PROM)

    non-interventional

    Also known as: routine-care assessments

06

What researchers measure

Primary outcomes

  1. 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

    Time frame: 7 years

  2. Development and evaluation of a diary for assessment and quantification of abdominal symptoms, a CF-specific PROM following FDA recommendations

    Time frame: 7

Secondary outcomes

  1. Abdominal symptoms in cystic fibrosis and their relation to genotype, history, clinical and laboratory findings

    relating CFAbd-Scores to history, clinical- and laboratory findings

    Time frame: 7 years

  2. Assessing the Relation of Ultrasound Findings and Abdominal Symptoms obtained with the CFAbd-Score in Cystic Fibrosis Patients

    abdominal ultrasound

    Time frame: 3 years

  3. 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

    Time frame: 7 years

07

Study locations

2 of 2 sites recruiting
08

References and documents

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. PubMed 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. PubMed 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. PubMed 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. PubMed 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 2022. Erratum In: Front Pharmacol. 2023 May 03;14:1207356. doi: 10.3389/fphar.2023.1207356. PubMed 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. PubMed 36710099 ↗

Individual participant data

Plan to share: No

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Apr 4, 2023, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT03052283
Lead sponsor
Medizinische Hochschule Brandenburg Theodor Fontane
Collaborators
University of Jena
Responsible party
Prof. Dr. Jochen G. Mainz (Director of the Cystic Fibrosis-Center, Medizinische Hochschule Brandenburg Theodor Fontane) — Principal investigator
First posted
Feb 14, 2017
Start date
Jan 26, 2017
Primary completion
Dec 31, 2026 (estimated)
Completion
Dec 31, 2027 (estimated)
Last update
Apr 4, 2023

Study contacts

Jochen G Mainz, Prof. MD
Contact
jochen.mainz@mhb-fontane.de
+493381411803
Franziska Duckstein, MA
Contact
f.duckstein@klinikum-brandenburg.de
+493381411841
Jochen G Mainz, Prof. MD
principal investigator · Medizinische Hochschule Brandenburg (MHB), University

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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