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

Last updated: February 20, 2026
Sponsor: Charite University, Berlin, Germany
Overall Status: Active - Recruiting

Phase

N/A

Condition

Cystic Fibrosis

Lung Disease

Scar Tissue

Treatment

Treatment with cystic fibrosis transmembrane regulator (CFTR) modualtors Ivacaftor, Lumacaftor-Ivacaftor, Tezacaftor-Ivacaftor, Elexacaftor-Tezacaftor-Ivacaftor, Vanzacaftor-Tezacaftor-Deutivacaftor

Treatment with cystic fibrosis transmembrane regulator (CFTR) modualtors Ivacaftor, Lumacaftor-Ivacaftor, Tezacaftor-Ivacaftor, Elexacaftor-Tezacaftor-Ivacaftor

Clinical Study ID

NCT04732910
20012746
  • Ages > 6
  • All Genders

Study Summary

This observational study evaluates the effect of therapy with cystic fibrosis transmembrane regulator (CFTR) modulators on CFTR function measured by the CFTR biomarker intestinal current measurement (ICM), nasal potential difference (NPD) and sweat chloride in a post-approval setting in patients with cystic fibrosis (CF).

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Decision for cystic fibrosis (CF) transmembrane regulator (CFTR)-modulator therapyby the patient and the caring CF physician

  • Signed informed consent form (ICF) and, where appropriate, signed assent form.

Exclusion

Exclusion Criteria:

  • Ongoing participation in an investigational drug study (including studiesinvestigating lumacaftor, tezacaftor or ivacaftor)

Study Design

Total Participants: 500
Treatment Group(s): 2
Primary Treatment: Treatment with cystic fibrosis transmembrane regulator (CFTR) modualtors Ivacaftor, Lumacaftor-Ivacaftor, Tezacaftor-Ivacaftor, Elexacaftor-Tezacaftor-Ivacaftor, Vanzacaftor-Tezacaftor-Deutivacaftor
Phase:
Study Start date:
July 01, 2018
Estimated Completion Date:
December 31, 2027

Study Description

Cystic fibrosis transmembrane regulator (CFTR) biomarker (intestinal current measurement (ICM), nasal potential difference (NPD), sweat chloride) before the start of therapy and 12 and 52 weeks after initiation of therapy Clinical parameters (anthropometry, lung function, lung magnetic resonance imaging (MRI), lung computer tomography (CT)) before the start of therapy and after initiation of therapy Assessment of airway secretion specimens before the start of therapy and after initiation of therapy

Connect with a study center

  • Charité - Universitätsmedizin Berlin

    Berlin 2950159, State of Berlin 2950157 13353
    Germany

    Active - Recruiting

  • Charité - Universitätsmedizin Berlin

    Berlin, 13353
    Germany

    Site Not Available

  • Justus-Liebig-University Giessen

    Giessen 2920512,
    Germany

    Active - Recruiting

  • Justus-Liebig-University Giessen

    Gießen,
    Germany

    Site Not Available

  • Hannover Medical School

    Hanover,
    Germany

    Site Not Available

  • Hannover Medical School

    Hanover 2910831,
    Germany

    Active - Recruiting

  • University of Heidelberg

    Heidelberg,
    Germany

    Site Not Available

  • University of Heidelberg

    Heidelberg 2907911,
    Germany

    Active - Recruiting

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