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

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
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 Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Charite University, Berlin, Germany

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Hannover Medical School
Heidelberg University
University of Luebeck
University of Giessen

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 1, 2018

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 31, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 31, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 25, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 29, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 1, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
February 24, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 20, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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.

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings