Evaluating CFTR function in cystic fibrosis patients treated with CFTR modulators
CFTR Biomarker Studie Bei Patient*Innen Mit Mukoviszidose Und CFTR-Modulatortherapie
This study is testing how well CFTR modulators work for people with cystic fibrosis by looking at changes in their body functions and health over a year.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 500 (estimated) |
| Ages | 6 Months and up |
| Sex | All |
| Sponsor | Charite University, Berlin, Germany Academic / other |
| Locations | 4 sites (Berlin, State of Berlin and 3 other locations) |
| Trial ID | NCT04732910 on ClinicalTrials.gov |
What this trial studies
This observational study assesses the impact of CFTR modulators on CFTR function in patients with cystic fibrosis. It measures biomarkers such as intestinal current measurement (ICM), nasal potential difference (NPD), and sweat chloride levels before treatment and at 12 and 52 weeks post-therapy initiation. Additionally, clinical parameters including lung function and imaging studies will be evaluated to understand the therapy's effects comprehensively. The study aims to gather real-world data on the efficacy of CFTR modulators in improving patient outcomes.
Who should consider this trial
Good fit: Ideal candidates for this study are patients with cystic fibrosis who have been prescribed CFTR modulator therapy by their physician.
Not a fit: Patients currently participating in other investigational drug studies may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could enhance the understanding of CFTR modulator therapies and their effectiveness in improving the health of cystic fibrosis patients.
How similar studies have performed: Other studies have shown promising results with CFTR modulators, indicating a potential for success in this observational approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Decision for cystic fibrosis (CF) transmembrane regulator (CFTR)-modulator therapy by the patient and the caring CF physician * Signed informed consent form (ICF) and, where appropriate, signed assent form. Exclusion Criteria: * Ongoing participation in an investigational drug study (including studies investigating lumacaftor, tezacaftor or ivacaftor)
Where this trial is running
Berlin, State of Berlin and 3 other locations
- Charité - Universitätsmedizin Berlin — Berlin, State of Berlin, Germany (Recruiting)
- Justus-Liebig-University Giessen — Giessen, Germany (Recruiting)
- Hannover Medical School — Hanover, Germany (Recruiting)
- University of Heidelberg — Heidelberg, Germany (Recruiting)
Study contacts
- Principal investigator: Marcus A Mall, MD — Charite University, Berlin, Germany
- Study coordinator: Simon Y Graeber, MD
- Email: simon.graeber@charite.de
- Phone: +4930 450 566 587
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.