NCT03052283

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
2,000

participants targeted

Target at P75+ for all trials

Timeline
20mo left

Started Jan 2017

Longer than P75 for all trials

Geographic Reach
1 country

2 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress85%
Jan 2017Dec 2027

Study Start

First participant enrolled

January 26, 2017

Completed
14 days until next milestone

First Submitted

Initial submission to the registry

February 9, 2017

Completed
5 days until next milestone

First Posted

Study publicly available on registry

February 14, 2017

Completed
9.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2026

Expected
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2027

Last Updated

April 4, 2023

Status Verified

March 1, 2023

Enrollment Period

9.9 years

First QC Date

February 9, 2017

Last Update Submit

March 31, 2023

Conditions

Cystic Fibrosis

Keywords

Cystic fibrosisAbdomenPancreasLiverGutSymptom ScorePatient reported outcome measure (PROM)diabetesCFTR-modulatorTricaftaKaftrio

Outcome Measures

Primary Outcomes (2)

  • 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

    7 years

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

    7

Secondary Outcomes (3)

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

    7 years

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

    3 years

  • Effects of a new therapy with CFTR-modulator on abdominal symtoms, quantified with the CFAbd-Score

    7 years

Study Arms (2)

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

Questionnaire Patient Reported Outcome Measure (PROM)DIAGNOSTIC_TEST

non-interventional

Also known as: routine-care assessments
Age-matched healthy controlsPatients with CF

Eligibility Criteria

Sexall
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Patients with CF of all ages

You may qualify if:

  • 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

You may not qualify if:

  • 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.)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Klinikum Westbrandenburg

Brandenburg, 14770, Germany

RECRUITING

Medizinische Hochschule Brandenburg (MHB), University

Brandenburg, 14770, Germany

RECRUITING

Related Publications (6)

  • 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: 28472055RESULT

  • 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: 29234058RESULT

  • 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: 30887269RESULT

  • 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: 34789668RESULT

  • 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.

    PMID: 35721187RESULT

  • 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: 36710099RESULT

Related Links

Biospecimen

Retention: SAMPLES WITHOUT DNA

Stool

MeSH Terms

Conditions

Cystic FibrosisDiabetes Mellitus

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, DiseasesGlucose Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesEndocrine System Diseases

Study Officials

  • Jochen G Mainz, Prof. MD

    Medizinische Hochschule Brandenburg (MHB), University

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Jochen G Mainz, Prof. MD

CONTACT

+493381411803jochen.mainz@mhb-fontane.de

Franziska Duckstein, MA

CONTACT

+493381411841f.duckstein@klinikum-brandenburg.de

Study Design

Study Type
observational
Observational Model
CASE CONTROL
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Director of the Cystic Fibrosis-Center

Study Record Dates

First Submitted

February 9, 2017

First Posted

February 14, 2017

Study Start

January 26, 2017

Primary Completion (Estimated)

December 31, 2026

Study Completion (Estimated)

December 31, 2027

Last Updated

April 4, 2023

Record last verified: 2023-03

Data Sharing

IPD Sharing
Will not share

Locations

DE(2)