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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)

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ClinicalTrials.gov Identifier: NCT04732910
Recruitment Status : Recruiting
First Posted : February 1, 2021
Last Update Posted : March 4, 2024
Sponsor:
Collaborators:
Hannover Medical School
Heidelberg University
University of Giessen
Information provided by (Responsible Party):
Simon Graeber, Charite University, Berlin, Germany

Study Description
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Brief 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).

Condition or disease Intervention/treatment
Cystic Fibrosis Drug: Treatment with cystic fibrosis transmembrane regulator (CFTR) modualtors Ivacaftor, Lumacaftor-Ivacaftor, Tezacaftor-Ivacaftor, Elexacaftor-Tezacaftor-Ivacaftor

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 Design
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Study Type : Observational
Estimated Enrollment : 200 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: CFTR Biomarker Studie Bei Patient*Innen Mit Mukoviszidose Und CFTR-Modulatortherapie
Actual Study Start Date : July 1, 2018
Estimated Primary Completion Date : March 31, 2027
Estimated Study Completion Date : December 31, 2027

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor

Groups and Cohorts
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Intervention Details:
  • Drug: Treatment with cystic fibrosis transmembrane regulator (CFTR) modualtors Ivacaftor, Lumacaftor-Ivacaftor, Tezacaftor-Ivacaftor, Elexacaftor-Tezacaftor-Ivacaftor
    observational

Outcome Measures
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Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. Forced expiratory volume in 1 second (FEV1) [ Time Frame: 12, 52, 104 weeks ]
    Absolute change from baseline in percent predicted forced expiratory volume in 1 second (FEV1) in spirometry

  2. 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

  3. Sweat chloride [ Time Frame: 12, 52, 104 weeks ]
    Absolute change from baseline of the chloride concentration in Gibson-Cooke pilocarpine iontophoresis sweat test as a cystic fibrosis transmembrane conductance regulator (CFTR) biomarker

  4. Lung clearance index (LCI) [ Time Frame: 12, 52, 104 weeks ]
    Absolute change from baseline of the lung clearance index (LCI)

  5. Lung magnetic resonance imaging (MRI) [ Time Frame: 12, 52, 104 weeks ]
    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)

  6. 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)

  7. Paranasal sinus magnetic resonance imaging (MRI) [ Time Frame: 12, 52, 104 weeks ]
    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)

  8. Fecal elastase [ Time Frame: 12, 52, 104 weeks ]
    Absolute change from baseline in fecal elastase-1 (FE-1) levels

  9. Weight [ Time Frame: 12, 52, 104 weeks ]
    Absolute change from baseline in weight

  10. Airway Microbiome [ Time Frame: 4, 12, 52, 104 weeks ]
    Absolute change in shannon index representing the alpha-diversity in sputum samples

  11. Sputum Elasticity [ Time Frame: 4, 12, 52, 104 weeks ]
    Absolute change in the elastic modulus (G') in sputum samples measured with a rheometer

  12. Sputum Viscocity [ Time Frame: 4, 12, 52, 104 weeks ]
    Absolute change in the viscous modulus (G'') in sputum samples measured with a rheometer


Biospecimen Retention:   Samples With DNA
Sputum, Blood

Eligibility Criteria
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Ages Eligible for Study:   6 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Cystic Fibrosis patients
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)
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04732910


Contacts
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Contact: Simon Y Graeber, MD +4930 450 566 587 simon.graeber@charite.de

Locations
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Germany
Charité - Universitätsmedizin Berlin Recruiting
Berlin, Germany, 13353
Contact: Simon Graeber, MD    +4930 450 566 587    simon.graeber@charite.de   
Justus-Liebig-University Giessen Recruiting
Gießen, Germany
Contact: Lutz Nährlich, MD         
Hannover Medical School Recruiting
Hanover, Germany
Contact: Burkhard Tümmler, MD         
University of Heidelberg Recruiting
Heidelberg, Germany
Contact: Olaf Sommerburg, MD         
Sponsors and Collaborators
Charite University, Berlin, Germany
Hannover Medical School
Heidelberg University
University of Giessen
Investigators
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Principal Investigator: Simon Y Graeber, MD Charite University, Berlin, Germany
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Simon Graeber, BIH Clinician Scientist, Charite University, Berlin, Germany
ClinicalTrials.gov Identifier: NCT04732910    
Other Study ID Numbers: 20012746
First Posted: February 1, 2021    Key Record Dates
Last Update Posted: March 4, 2024
Last Verified: March 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Simon Graeber, Charite University, Berlin, Germany:
CFTR
ICM
NPD
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
 Genetic Diseases, Inborn
Infant, Newborn, Diseases
Ivacaftor
Elexacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action