Clinical trial NCT04732910: Cystic Fibrosis Transmembrane Regulator (CFTR) Biomarker Study to Evaluate the Rescue of Mutant CFTR in Patients With Cystic Fibrosis Treated With CFTR-modulators
Official title: CFTR Biomarker Studie Bei Patient*Innen Mit Mukoviszidose Und CFTR-Modulatortherapie
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Cystic Fibrosis
Keywords: CFTR
ICM
NPD
Lead sponsor: Charite University, Berlin, Germany
Sponsor class: OTHER
Collaborators: name: Hannover Medical School
name: Heidelberg University
name: University of Giessen
First posted: 2021-02-01
Enrollment: 200
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Cystic Fibrosis patients

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).

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

Eligibility 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)

Primary outcomes:
measure: Intestinal current measurement (ICM)
timeFrame: 12 weeks
Secondary outcomes:
measure: Forced expiratory volume in 1 second (FEV1)
timeFrame: 12, 52, 104 weeks
measure: Nasal potential Difference (NPD)
timeFrame: 12 weeks
measure: Sweat chloride
timeFrame: 12, 52, 104 weeks
measure: Lung clearance index (LCI)
timeFrame: 12, 52, 104 weeks
measure: Lung magnetic resonance imaging (MRI)
timeFrame: 12, 52, 104 weeks
measure: Lung computer tomography
timeFrame: 52, 104 weeks
measure: Paranasal sinus magnetic resonance imaging (MRI)
timeFrame: 12, 52, 104 weeks
measure: Fecal elastase
timeFrame: 12, 52, 104 weeks
measure: Weight
timeFrame: 12, 52, 104 weeks
measure: Airway Microbiome
timeFrame: 4, 12, 52, 104 weeks
measure: Sputum Elasticity
timeFrame: 4, 12, 52, 104 weeks
measure: Sputum Viscocity
timeFrame: 4, 12, 52, 104 weeks
Other outcomes:
Locations:
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin
city: Gießen
country: Germany
facility: Justus-Liebig-University Giessen
city: Hanover
country: Germany
facility: Hannover Medical School
city: Heidelberg
country: Germany
facility: University of Heidelberg
Study officials:
affiliation: Charite University, Berlin, Germany
name: Simon Y Graeber, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.