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

NCT03052283 · tracked via the Priya Life Science Germany tracker

Sponsor

Medizinische Hochschule Brandenburg Theodor Fontane

Phase

Observational

Started

2017-01-26

Last updated

2023-04-04

Condition(s) studied

Cystic Fibrosis

Investigational drug(s) / intervention(s)

Questionnaire Patient Reported Outcome Measure (PROM)

Questionnaire Patient Reported Outcome Measure (PROM): non-interventional

Study 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.

Eligibility

Sex

ALL

Min age

—

Max age

—

Healthy volunteers

Accepted

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.)

Primary outcome measure(s)

Development and evaluation of the CFAbd-Score (initially named JenAbdomen CF-Score), a CF-specific PROM following FDA recommendations — 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 — 7

Trial sites (2)

Facility	City	Region	Status
Klinikum Westbrandenburg	Brandenburg an der Havel	Germany	Recruiting
Medizinische Hochschule Brandenburg (MHB), University	Brandenburg an der Havel	Germany	Recruiting

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Other trials for the same condition

A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy Active, not recruiting

Modulate-CF: Cystic Fibrosis Transmembrane Regulator (CFTR) Biomarker Study to Evaluate the Rescue of Mutant CFTR in Patients With Cystic Fibrosis Treated With CFTR-modulators Recruiting

Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del Active, not recruiting

Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older Enrolling by invitation

Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT03052283 on ClinicalTrials.gov ↗ ← All trials in Germany