Cystic FibrosisCFAbd-ScoreNCT03052283
Development and Validation of a Disease Specific PROM to Assess Abdominal Involvement in Patients With CF (CFAbd-Score)
An observational study of Cystic Fibrosis, run by Medizinische Hochschule Brandenburg Theodor Fontane at 2 sites in Germany.
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. Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Questionnaire Patient Reported Outcome Measure (PROM)
- How many people
- 2,000 participants
- Where
- 2 sitesGermany
- Study dates
- 26 Jan 2017to 31 Dec 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: Patients with CF of all ages
Who may be able to join
- 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
Who cannot join
- 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 locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Germany2 sites, 2 recruiting
- Recruiting
Klinikum Westbrandenburg
Brandenburg an der Havel, 14770
Jochen G Mainz, Prof. Dr., +493381411803, jochen.mainz@mhb-fontane.de
Franziska Duckstein, MA, +493381411841, f.duckstein@klinikum-brandenburg.de
- Recruiting
Medizinische Hochschule Brandenburg (MHB), University
Brandenburg an der Havel, 14770
Jochen G Mainz, Prof. Dr., +493381411803, jochen.mainz@mhb-fontane.de
Franziska Duckstein, MA, f.duckstein@klinikum-brandenburg.de
About this study
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)
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
- Conditions
- Cystic Fibrosis
- Being tested
- Questionnaire Patient Reported Outcome Measure (PROM)
- Keywords
- Cystic fibrosis, Abdomen, Pancreas, Liver, Gut, Symptom Score, Patient reported outcome measure (PROM), diabetes, CFTR-modulator, Tricafta, Kaftrio
- Medical subject headings
- Cystic Fibrosis, Diabetes Mellitus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Questionnaire Patient Reported Outcome Measure (PROM)Diagnostic test
Also called routine-care assessments
non-interventional
| Study group | Role | What they receive |
|---|---|---|
| Patients with CF | >2000 in Germany, France, Italy, Spain, Denmark, Belgium, Portugal, GB, Ireland, USA, Australia, Canada, Brazil, Argentina... Receives: Diagnostic Test: Questionnaire Patient Reported Outcome Measure (PROM) | |
| Age-matched healthy controls | >100 in Germany >100 in each of the other participating countries Receives: Diagnostic Test: Questionnaire Patient Reported Outcome Measure (PROM) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 7 years
Development and evaluation of a diary for assessment and quantification of abdominal symptoms, a CF-specific PROM following FDA recommendations
Measured: 7
Secondary outcomes (3)
Abdominal symptoms in cystic fibrosis and their relation to genotype, history, clinical and laboratory findings
relating CFAbd-Scores to history, clinical- and laboratory findings
Measured: 7 years
Assessing the Relation of Ultrasound Findings and Abdominal Symptoms obtained with the CFAbd-Score in Cystic Fibrosis Patients
abdominal ultrasound
Measured: 3 years
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
Measured: 7 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Feb 2017
- Study start26 Jan 2017
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 2,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Medizinische Hochschule Brandenburg Theodor Fontane
- Sponsor type
- University, hospital or other
- Collaborators
- University of Jena
- Responsible party
- Principal investigator, Prof. Dr. Jochen G. Mainz, Medizinische Hochschule Brandenburg Theodor Fontane
- Investigators
- Jochen G Mainz, Prof. MD, Principal investigator, Medizinische Hochschule Brandenburg (MHB), University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03052283
- Sponsor's protocol ID
- CFAbd-Score
- Other IDs
- CFAbd-Score.kid, CFAbd-day2day
- Acronym
- CFAbd-Score
- Status last verified
- Mar 2023
- First submitted
- 9 Feb 2017
- Last updated
- 4 Apr 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
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 28472055Tabori 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 29234058Jaudszus 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 30887269Jaudszus 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 34789668Mainz 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.
PubMed 35721187Caley 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 36710099Sadrieh 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.
PubMed 40701924
Study at a glance
From the registry record, last updated by the sponsor on 4 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT03052283
- Acronym / sponsor ID
- CFAbd-Score / CFAbd-Score
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic fibrosis, Abdomen, Pancreas, Liver, Gut, Symptom Score, Patient reported outcome measure (PROM), diabetes
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 2,000 participants (estimated)
- Lead sponsor
- Medizinische Hochschule Brandenburg Theodor FontaneUniversity, hospital or other
- Collaborators
- University of Jena
- Study start
- 26 Jan 2017 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 14 Feb 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients with CF | – | Diagnostic Test: Questionnaire Patient Reported Outcome Measure (PROM)>2000 in Germany, France, Italy, Spain, Denmark, Belgium, Portugal, GB, Ireland, USA, Australia, Canada, Brazil, Argentina... |
| Age-matched healthy controls | – | Diagnostic Test: Questionnaire Patient Reported Outcome Measure (PROM)>100 in Germany >100 in each of the other participating countries |
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with CF of all ages
Key inclusion criteria (6 listed)
- 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.
- Age-matched
Key exclusion criteria (4 listed)
- Lacking ability to cooperate of patients /their families, respectively
- Other diseases causing abdominal symptoms (Inflammatory bowel disease (IBD), alpha 1 antitrypsin deficiency, celiac disease, food allergy, gastroenteritis, etc.)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Questionnaire Patient Reported Outcome Measure (PROM)Also: routine-care assessmentsnon-interventional | Diagnostic test | Age-matched healthy controls, Patients with CF |
Primary outcome
Development and evaluation of the CFAbd-Score (initially named JenAbdomen CF-Score), a CF-specific PROM following FDA recommendations
Time frame: 7 years
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
Development and evaluation of a diary for assessment and quantification of abdominal symptoms, a CF-specific PROM following FDA recommendations
Time frame: 7
3 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Apr 2023.
- Results publication: PubMed 28472055Tabori 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.
- Results publication: PubMed 29234058Tabori 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.
- Results publication: PubMed 30887269Jaudszus 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.
- Results publication: PubMed 34789668Jaudszus 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.
- Results publication: PubMed 35721187Mainz 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.
- Results publication: PubMed 36710099Caley 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.
- Derived from this study: PubMed 40701924Sadrieh 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.
- Validity and Reliability of a Novel Multimodal Questionnaire for the Assessment of Abdominal Symptoms in People with Cystic Fibrosis (CFAbd-Score).Link provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Jochen G Mainz, Prof. MD principal investigator
Medizinische Hochschule Brandenburg (MHB), University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by Medizinische Hochschule Brandenburg Theodor Fontane at 2 sites in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 4 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jochen G Mainz, Prof. MD (+493381411803, jochen.mainz@mhb-fontane.de), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Development and evaluation of the CFAbd-Score (initially named JenAbdomen CF-Score), a CF-specific PROM following FDA recommendations (time frame: 7 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03052283, which also lists 7 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Apr 2023.