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

NCT ID: NCT03052283

Last Updated: 2023-04-04

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

2000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2017-01-26

Study Completion Date

2027-12-31

Brief Summary

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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

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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

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Cystic Fibrosis

Study Design

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Observational Model Type

CASE_CONTROL

Study Time Perspective

PROSPECTIVE

Study Groups

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Patients with CF

\>2000 in Germany, France, Italy, Spain, Denmark, Belgium, Portugal, GB, Ireland, USA, Australia, Canada, Brazil, Argentina...

Questionnaire Patient Reported Outcome Measure (PROM)

Intervention Type DIAGNOSTIC_TEST

non-interventional

Age-matched healthy controls

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

Questionnaire Patient Reported Outcome Measure (PROM)

Intervention Type DIAGNOSTIC_TEST

non-interventional

Interventions

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Questionnaire Patient Reported Outcome Measure (PROM)

non-interventional

Intervention Type DIAGNOSTIC_TEST

Other Intervention Names

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routine-care assessments

Eligibility Criteria

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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.)
Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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University of Jena

OTHER

Sponsor Role collaborator

Medizinische Hochschule Brandenburg Theodor Fontane

OTHER

Sponsor Role lead

Responsible Party

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Prof. Dr. Jochen G. Mainz

Director of the Cystic Fibrosis-Center

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Jochen G Mainz, Prof. MD

Role: PRINCIPAL_INVESTIGATOR

Medizinische Hochschule Brandenburg (MHB), University

Locations

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Klinikum Westbrandenburg

Brandenburg, , Germany

Site Status RECRUITING

Medizinische Hochschule Brandenburg (MHB), University

Brandenburg, , Germany

Site Status RECRUITING

Countries

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Germany

Central Contacts

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Jochen G Mainz, Prof. MD

Role: CONTACT

+493381411803

Franziska Duckstein, MA

Role: CONTACT

+493381411841

Facility Contacts

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Jochen G Mainz, Prof. Dr.

Role: primary

+493381411803

Franziska Duckstein, MA

Role: backup

+493381411841

Jochen G Mainz, Prof. Dr.

Role: primary

+493381411803

Franziska Duckstein, MA

Role: backup

References

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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.

Reference Type RESULT
PMID: 28472055 (View on PubMed)

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.

Reference Type RESULT
PMID: 29234058 (View on PubMed)

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.

Reference Type RESULT
PMID: 30887269 (View on PubMed)

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.

Reference Type RESULT
PMID: 34789668 (View on PubMed)

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.

Reference Type RESULT
PMID: 35721187 (View on PubMed)

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.

Reference Type RESULT
PMID: 36710099 (View on PubMed)

Related Links

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https://link.springer.com/article/10.1007%2Fs40271-019-00361-2

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

Other Identifiers

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CFAbd-Score.kid

Identifier Type: OTHER

Identifier Source: secondary_id

CFAbd-day2day

Identifier Type: OTHER

Identifier Source: secondary_id

CFAbd-Score

Identifier Type: -

Identifier Source: org_study_id

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