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
CFTR Biomarker Studie Bei Patient*Innen Mit Mukoviszidose Und CFTR-Modulatortherapie
1 other identifier
observational
500
1 country
4
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Jul 2018
Longer than P75 for all trials
4 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
July 1, 2018
CompletedFirst Submitted
Initial submission to the registry
January 25, 2021
CompletedFirst Posted
Study publicly available on registry
February 1, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 31, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 31, 2027
February 24, 2026
February 1, 2026
8.8 years
January 25, 2021
February 20, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Intestinal current measurement (ICM)
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 Outcomes (20)
Forced expiratory volume in 1 second (FEV1)
quaterly in the first year, yearly thereafter up to 5 years
Nasal potential Difference (NPD)
12 weeks
Sweat chloride
12, 52 weeks, yearly thereafter up to 5 years
Lung clearance index (LCI)
quaterly in the first year, yearly thereafter up to 5 years
Lung magnetic resonance imaging (MRI)
12, 52 weeks, yearly thereafter up to 5 years
- +15 more secondary outcomes
Interventions
Eligibility Criteria
Cystic Fibrosis patients
You may qualify if:
- 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.
You may not qualify if:
- Ongoing participation in an investigational drug study (including studies investigating lumacaftor, tezacaftor or ivacaftor)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Charite University, Berlin, Germanylead
- Hannover Medical Schoolcollaborator
- Heidelberg Universitycollaborator
- University of Giessencollaborator
- University of Luebeckcollaborator
Study Sites (4)
Charité - Universitätsmedizin Berlin
Berlin, State of Berlin, 13353, Germany
Justus-Liebig-University Giessen
Giessen, Germany
Hannover Medical School
Hanover, Germany
University of Heidelberg
Heidelberg, Germany
Related Publications (4)
Dohna M, Voskrebenzev A, Klimes F, Kaireit TF, Glandorf J, Pallenberg ST, Ringshausen FC, Hansen G, Renz DM, Wacker F, Dittrich AM, Vogel-Claussen J. PREFUL MRI for Monitoring Perfusion and Ventilation Changes after Elexacaftor-Tezacaftor-Ivacaftor Therapy for Cystic Fibrosis: A Feasibility Study. Radiol Cardiothorac Imaging. 2024 Apr;6(2):e230104. doi: 10.1148/ryct.230104.
PMID: 38573129DERIVEDSchaupp L, Addante A, Voller M, Fentker K, Kuppe A, Bardua M, Duerr J, Piehler L, Rohmel J, Thee S, Kirchner M, Ziehm M, Lauster D, Haag R, Gradzielski M, Stahl M, Mertins P, Boutin S, Graeber SY, Mall MA. Longitudinal effects of elexacaftor/tezacaftor/ivacaftor on sputum viscoelastic properties, airway infection and inflammation in patients with cystic fibrosis. Eur Respir J. 2023 Aug 3;62(2):2202153. doi: 10.1183/13993003.02153-2022. Print 2023 Aug.
PMID: 37414422DERIVEDGraeber SY, Renz DM, Stahl M, Pallenberg ST, Sommerburg O, Naehrlich L, Berges J, Dohna M, Ringshausen FC, Doellinger F, Vitzthum C, Rohmel J, Allomba C, Hammerling S, Barth S, Ruckes-Nilges C, Wielputz MO, Hansen G, Vogel-Claussen J, Tummler B, Mall MA, Dittrich AM. Effects of Elexacaftor/Tezacaftor/Ivacaftor Therapy on Lung Clearance Index and Magnetic Resonance Imaging in Patients with Cystic Fibrosis and One or Two F508del Alleles. Am J Respir Crit Care Med. 2022 Aug 1;206(3):311-320. doi: 10.1164/rccm.202201-0219OC.
PMID: 35536314DERIVEDGraeber SY, Vitzthum C, Pallenberg ST, Naehrlich L, Stahl M, Rohrbach A, Drescher M, Minso R, Ringshausen FC, Rueckes-Nilges C, Klajda J, Berges J, Yu Y, Scheuermann H, Hirtz S, Sommerburg O, Dittrich AM, Tummler B, Mall MA. Effects of Elexacaftor/Tezacaftor/Ivacaftor Therapy on CFTR Function in Patients with Cystic Fibrosis and One or Two F508del Alleles. Am J Respir Crit Care Med. 2022 Mar 1;205(5):540-549. doi: 10.1164/rccm.202110-2249OC.
PMID: 34936849DERIVED
Biospecimen
Sputum, Blood
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Marcus A Mall, MD
Charite University, Berlin, Germany
- PRINCIPAL INVESTIGATOR
Simon Y Graeber, MD
Charite University, Berlin, Germany
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Clinician Scientist
Study Record Dates
First Submitted
January 25, 2021
First Posted
February 1, 2021
Study Start
July 1, 2018
Primary Completion (Estimated)
March 31, 2027
Study Completion (Estimated)
December 31, 2027
Last Updated
February 24, 2026
Record last verified: 2026-02