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 (Modulate-CF)

February 20, 2026 updated by: Simon Graeber, Charite University, Berlin, Germany

CFTR Biomarker Studie Bei Patient*Innen Mit Mukoviszidose Und CFTR-Modulatortherapie

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).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed 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

Study Type

Observational

Enrollment (Estimated)

500

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Germany
      • Giessen, Germany
        • Recruiting
        • Justus-Liebig-University Giessen
        • Contact:
          • Lutz Nährlich, MD
      • Hanover, Germany
      • Heidelberg, Germany
        • Recruiting
        • University of Heidelberg
        • Contact:
          • Olaf Sommerburg, MD
    • State of Berlin
      • Berlin, State of Berlin, Germany, 13353
        • Recruiting
        • Charité - Universitätsmedizin Berlin
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 months and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Cystic Fibrosis patients

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Intestinal current measurement (ICM)
Time Frame: 12 weeks
Absolute change from baseline of the chloride secretory ion current induced by cyclic adenosine monophosphate (cAMP) stimulation (forskolin/3-isobutyl-1-methylxanthine (IBMX)) in rectal tissue determined by intestinal current measurement (ICM) as a cystic fibrosis transmembrane conductance regulator (CFTR) biomarker
12 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Nasal potential Difference (NPD)
Time Frame: 12 weeks
Absolute change from baseline total chloride response (zero chloride and isoproterenol) in nasal potential Difference (NPD) as a cystic fibrosis transmembrane conductance regulator (CFTR) biomarker
12 weeks
Lung computer tomography
Time Frame: 52, 104 weeks
Absolute change from baseline in lung computer tomography (CT) score (Brody score ranging from 0 to 40,5 with higher values associated with worsening of the outcome; Brody et al. J Thorac Imaging 2006)
52, 104 weeks
Forced expiratory volume in 1 second (FEV1)
Time Frame: quaterly in the first year, yearly thereafter up to 5 years
Absolute change from baseline in percent predicted forced expiratory volume in 1 second (FEV1) in spirometry
quaterly in the first year, yearly thereafter up to 5 years
Sweat chloride
Time Frame: 12, 52 weeks, yearly thereafter up to 5 years
Absolute change from baseline of the chloride concentration in Gibson-Cooke pilocarpine iontophoresis sweat test as a cystic fibrosis transmembrane conductance regulator (CFTR) biomarker
12, 52 weeks, yearly thereafter up to 5 years
Lung clearance index (LCI)
Time Frame: quaterly in the first year, yearly thereafter up to 5 years
Absolute change from baseline of the lung clearance index (LCI)
quaterly in the first year, yearly thereafter up to 5 years
Lung magnetic resonance imaging (MRI)
Time Frame: 12, 52 weeks, yearly thereafter up to 5 years
Absolute change from baseline in lung magnetic resonance imaging (MRI) score (Heidelberg MRI score ranging from 0 to 72 with higher values associated with worsening of the outcome; Eichinger et al. Eur J Radiol 2012)
12, 52 weeks, yearly thereafter up to 5 years
Paranasal sinus magnetic resonance imaging (MRI)
Time Frame: 12, 52 weeks, yearly thereafter up to 5 years
Absolute change from baseline in paranasal sinus magnetic resonance imaging (MRI) score (Sinunasal MRI score ranging from 0 to 68 with higher values associated with worsening of the outcome; Sommerburg et al. Ann Am Thorac Soc 2020)
12, 52 weeks, yearly thereafter up to 5 years
Fecal elastase
Time Frame: 12, 52 weeks, yearly thereafter up to 5 years
Absolute change from baseline in fecal elastase-1 (FE-1) levels
12, 52 weeks, yearly thereafter up to 5 years
Weight
Time Frame: quaterly in the first year, yearly thereafter up to 5 years
Absolute change from baseline in weight
quaterly in the first year, yearly thereafter up to 5 years
Airway Microbiome
Time Frame: 4, 12, 52 weeks, yearly thereafter up to 5 years
Absolute change in shannon index representing the alpha-diversity in sputum samples
4, 12, 52 weeks, yearly thereafter up to 5 years
Sputum Elasticity
Time Frame: 4, 12, 52 weeks, yearly thereafter up to 5 years
Absolute change in the elastic modulus (G') in sputum samples measured with a rheometer
4, 12, 52 weeks, yearly thereafter up to 5 years
Sputum Viscocity
Time Frame: 4, 12, 52 weeks, yearly thereafter up to 5 years
Absolute change in the viscous modulus (G'') in sputum samples measured with a rheometer
4, 12, 52 weeks, yearly thereafter up to 5 years
Cystic Fibrosis Questionnaire-Revised (CFQ-R)
Time Frame: quarterly in the first year, yearly thereafter up to 5 years
The Cystic Fibrosis Questionnaire-Revised is a disease-specific health-related quality of life instrument for individuals with cystic fibrosis. Scores are standardized on a 0-100 scale. Minimum: 0; Maximum: 100. Higher scores indicate better health-related quality of life. Absolute change from baseline is calculated as follow-up score minus baseline score.
quarterly in the first year, yearly thereafter up to 5 years
Patient Health Questionnaire-9 (PHQ-9)
Time Frame: quarterly in the first year, yearly thereafter up to 5 years
The Patient Health Questionnaire-9 is a 9-item self-report measure assessing depressive symptom severity over the past two weeks. Total score range: 0-27. Minimum: 0; Maximum: 27. Higher scores indicate more severe depressive symptoms. Absolute change from baseline is calculated as follow-up score minus baseline score.
quarterly in the first year, yearly thereafter up to 5 years
Generalized Anxiety Disorder-7 (GAD-7)
Time Frame: quarterly in the first year, yearly thereafter up to 5 years
The Generalized Anxiety Disorder-7 is a 7-item self-report measure assessing anxiety symptom severity over the past two weeks. Total score range: 0-21. Minimum: 0; Maximum: 21. Higher scores indicate more severe anxiety symptoms. Absolute change from baseline is calculated as follow-up score minus baseline score.
quarterly in the first year, yearly thereafter up to 5 years
Child Behavior Checklist for Ages 1½-5 (CBCL/1½-5)
Time Frame: quarterly in the first year, yearly thereafter up to 5 years
The Child Behavior Checklist for Ages 1½-5 is a parent-reported measure assessing emotional and behavioral problems in children aged 18 months to 5 years. The Total Problems raw score ranges from 0-198. Minimum: 0; Maximum: 198. Higher scores indicate more emotional and behavioral problems. Absolute change from baseline is calculated as follow-up score minus baseline score.
quarterly in the first year, yearly thereafter up to 5 years
Child Behavior Checklist for Ages 6-18 (CBCL/6-18)
Time Frame: quarterly in the first year, yearly thereafter up to 5 years
The Child Behavior Checklist for Ages 6-18 is a parent-reported measure of emotional and behavioral problems. Raw total problem scores range from 0-226. Minimum: 0; Maximum: 226. Higher scores indicate more behavioral and emotional problems. Absolute change from baseline is calculated as follow-up score minus baseline score.
quarterly in the first year, yearly thereafter up to 5 years
Youth Self-Report for Ages 11-18 (YSR/11-18)
Time Frame: quarterly in the first year, yearly thereafter up to 5 years
The Youth Self-Report for Ages 11-18 is a self-report measure of emotional and behavioral problems, corresponding to the Child Behavior Checklist. Raw total problem scores range from 0-224. Minimum: 0; Maximum: 224. Higher scores indicate more behavioral and emotional problems. Absolute change from baseline is calculated as follow-up score minus baseline score.
quarterly in the first year, yearly thereafter up to 5 years
Strengths and Difficulties Questionnaire (SDQ)
Time Frame: quarterly in the first year, yearly thereafter up to 5 years
The Strengths and Difficulties Questionnaire is a behavioral screening questionnaire. The Total Difficulties Score ranges from 0-40. Minimum: 0; Maximum: 40. Higher scores indicate more emotional and behavioral difficulties. Absolute change from baseline is calculated as follow-up score minus baseline score.
quarterly in the first year, yearly thereafter up to 5 years
22-Item Sino-Nasal Outcome Test (SNOT-22)
Time Frame: quarterly in the first year, yearly thereafter up to 5 years
The 22-Item Sino-Nasal Outcome Test assesses symptom severity and health-related quality of life in sinonasal disease. The total score ranges from 0-110. Minimum: 0; Maximum: 110. Higher scores indicate more severe symptoms and poorer quality of life. Absolute change from baseline is calculated as follow-up score minus baseline score.
quarterly in the first year, yearly thereafter up to 5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Charite University, Berlin, Germany

Collaborators

Hannover Medical School
Heidelberg University
University of Luebeck
University of Giessen

Investigators

  • Principal Investigator: Simon Y Graeber, MD, Charite University, Berlin, Germany
  • Principal Investigator: Marcus A Mall, MD, Charite University, Berlin, Germany

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 1, 2018

Primary Completion (Estimated)

March 31, 2027

Study Completion (Estimated)

December 31, 2027

Study Registration Dates

First Submitted

January 25, 2021

First Submitted That Met QC Criteria

January 29, 2021

First Posted (Actual)

February 1, 2021

Study Record Updates

Last Update Posted (Actual)

February 24, 2026

Last Update Submitted That Met QC Criteria

February 20, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 20012746

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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