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Clinical Trials/NCT03052283
NCT03052283RecruitingNot Applicable

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)

Medizinische Hochschule Brandenburg Theodor Fontane2 sites in 1 country2,000 target enrollmentStarted: January 26, 2017Last updated:
Conditions

Trial Snapshot

Phase
Not Applicable
Status
Recruiting
Sponsor
Enrollment
2,000
Locations
2
Primary Endpoint
Development and evaluation of a diary for assessment and quantification of abdominal symptoms, a CF-specific PROM following FDA recommendations

Study Overview

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

Study Design

Study Type
Observational
Observational Model
Case Control
Time Perspective
Prospective

Eligibility Criteria

Sex
All
Accepts Healthy Volunteers
Yes

Inclusion Criteria

  • 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

  • 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.)

Outcomes

Primary Outcomes

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

Time Frame: 7

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

Secondary Outcomes

  • 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)

Investigators

Sponsor
Medizinische Hochschule Brandenburg Theodor Fontane
Sponsor Class
Other
Responsible Party
Principal Investigator
Principal Investigator

Prof. Dr. Jochen G. Mainz

Director of the Cystic Fibrosis-Center

Medizinische Hochschule Brandenburg Theodor Fontane

Study Sites (2)

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