Evaluating CFTR function in cystic fibrosis patients treated with CFTR modulators

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.