Cystic Fibrosis Transmembrane Regulator (CFTR) Biomarker Study to Evaluate the Rescue of Mutant CFTR in Patients With Cystic Fibrosis Treated With CFTR-modulators
NCT ID: NCT04732910
Last Updated: 2024-03-04
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
200 participants
OBSERVATIONAL
2018-07-01
2027-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
PROSPECTIVE
Interventions
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Treatment with cystic fibrosis transmembrane regulator (CFTR) modualtors Ivacaftor, Lumacaftor-Ivacaftor, Tezacaftor-Ivacaftor, Elexacaftor-Tezacaftor-Ivacaftor
observational
Eligibility Criteria
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Inclusion Criteria
* Signed informed consent form (ICF) and, where appropriate, signed assent form.
Exclusion Criteria
6 Months
ALL
No
Sponsors
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Hannover Medical School
OTHER
Heidelberg University
OTHER
University of Giessen
OTHER
Charite University, Berlin, Germany
OTHER
Responsible Party
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Simon Graeber
BIH Clinician Scientist
Principal Investigators
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Simon Y Graeber, MD
Role: PRINCIPAL_INVESTIGATOR
Charite University, Berlin, Germany
Locations
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Charité - Universitätsmedizin Berlin
Berlin, , Germany
Justus-Liebig-University Giessen
Giessen, , Germany
Hannover Medical School
Hanover, , Germany
University of Heidelberg
Heidelberg, , Germany
Countries
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Central Contacts
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Facility Contacts
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Lutz Nährlich, MD
Role: primary
Burkhard Tümmler, MD
Role: primary
Olaf Sommerburg, MD
Role: primary
References
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Schaupp 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.
Graeber 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.
Graeber 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.
Other Identifiers
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20012746
Identifier Type: -
Identifier Source: org_study_id
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