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NCT Number: NCT03052283

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

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Diabetes Mellitus Digestive System Diseases Endocrine System Diseases Genetic Diseases, Inborn Glucose Metabolism Disorders Infant, Newborn, Diseases Lung Diseases Metabolic Diseases Nutritional and Metabolic Diseases Pancreatic Diseases Respiratory Tract Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Klinikum Westbrandenburg, Brandenburg an der Havel, Germany

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About this study

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

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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.)

Treatment and study plan

Questionnaire Patient Reported Outcome Measure (PROM)

Diagnostic Test

non-interventional

Other names: routine-care assessments

Primary outcomes

  1. 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

  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

    Time frame: 7 years

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

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

    Time frame: 3 years

    abdominal ultrasound

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

    Time frame: 7 years

    Assessing the CFAbd-Score´s sensitivity to identify and quantify changes due to effective therapeutic interventions, following FDA-guidelines for validation of a PROM

Study contacts

Contact information is provided by the study sponsor or research team.

Franziska Duckstein, MA

CONTACT

[email protected]

+493381411841

Jochen G Mainz, Prof. MD

CONTACT

[email protected]

+493381411803

Sponsors and collaborators

Lead sponsor

Medizinische Hochschule Brandenburg Theodor Fontane

Other

Collaborators

  • University of Jena

Registry information

Official study 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)

Acronym: CFAbd-Score

Important dates

Study start
2017
Primary completion
2026
Study completion
2027
First posted
Feb 14, 2017
Registry last updated
Apr 4, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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