Effect of the CFTR-modulating Triple Therapy Elexacaftor - Tezacaftor - Ivacaftor
1 other identifier
observational
130
1 country
1
Brief Summary
The aim of this study is to investigate the frequency distribution, cytokine profile and function of peripheral, mononuclear leukocyte populations (monocytes, NK cells, T/B lymphocytes) and their correlation to clinical and biochemical parameters in patients with cystic fibrosis receiving CFTR modulatory triple therapy consisting of elexacaftor, tezacaftor and ivacaftor and to compare it with patients without CFTR modulatory therapy and healthy control subjects.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for all trials
Started Dec 2020
Typical duration for all trials
1 active site
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
December 30, 2020
CompletedFirst Submitted
Initial submission to the registry
September 27, 2022
CompletedFirst Posted
Study publicly available on registry
October 12, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 18, 2023
CompletedStudy Completion
Last participant's last visit for all outcomes
October 18, 2023
CompletedOctober 12, 2022
October 1, 2022
2.8 years
September 27, 2022
October 7, 2022
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Peripheral Blood Immunograms
Relative and absolute peripheral blood immune cell count as determined by multicolor flow cytometry
prior ETI
Peripheral Blood Immunograms
Relative and absolute peripheral blood immune cell count as determined by multicolor flow cytometry
6 months ETI
Secondary Outcomes (45)
Shear Wave Velocity (SWV)
prior ETI
Shear Wave Velocity (SWV)
6 months ETI
Attenuation Coefficient (AC)
prior ETI
Attenuation Coefficient (AC)
6 months ETI
Serum bile acids
prior ETI
- +40 more secondary outcomes
Study Arms (4)
longitudinal
Inclusion of patients with diagnosed CF prior ETI therapy, follow-up visit after 6 months
under ETI
Patients with diagnosed CF already receiving ETI therapy for 6 months
no ETI
Patients with diagnosed CF that have refused an ETI treatment or are not eligible for ETI therapy
Healthy Individuals
Healthy, age- and gender-matched probands
Interventions
Elexacaftor / Ivacaftor / Tezacaftor is a triple drug therapy that modulates CFTR availability at (apical) cell membranes and increases opening probability.
Eligibility Criteria
All patients who are under the care of the CF Center Erlangen at the time of the study and who meet the inclusion criteria will be considered for the patient collective. Recruitment of healthy subjects will be performed during routine blood sampling in patients with pulmonary disease during outpatient appointments as well as during inpatient stays after prior informed consent has been obtained.
You may qualify if:
- Patients (m/f/d) with molecularly genetically confirmed cystic fibrosis aged 6 years and older.
You may not qualify if:
- Written informed consent
- For study arm "Kaftrio® ongoing": Kaftrio® therapy for at least 6 months
- For study arm "Kaftrio® longitudinal": no Kaftrio® therapy started yet
- Use of inhaled or systemic glucocorticoids as part of a permanent medication regimen
- Pregnancy
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
University Hospital Erlange, Department of Pediatrics
Erlangen, Bavaria, 91054, Germany
Related Publications (4)
Keating D, Marigowda G, Burr L, Daines C, Mall MA, McKone EF, Ramsey BW, Rowe SM, Sass LA, Tullis E, McKee CM, Moskowitz SM, Robertson S, Savage J, Simard C, Van Goor F, Waltz D, Xuan F, Young T, Taylor-Cousar JL; VX16-445-001 Study Group. VX-445-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles. N Engl J Med. 2018 Oct 25;379(17):1612-1620. doi: 10.1056/NEJMoa1807120. Epub 2018 Oct 18.
PMID: 30334692BACKGROUNDZemanick ET, Taylor-Cousar JL, Davies J, Gibson RL, Mall MA, McKone EF, McNally P, Ramsey BW, Rayment JH, Rowe SM, Tullis E, Ahluwalia N, Chu C, Ho T, Moskowitz SM, Noel S, Tian S, Waltz D, Weinstock TG, Xuan F, Wainwright CE, McColley SA. A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One F508del Allele. Am J Respir Crit Care Med. 2021 Jun 15;203(12):1522-1532. doi: 10.1164/rccm.202102-0509OC.
PMID: 33734030BACKGROUNDMcDonald TV, Nghiem PT, Gardner P, Martens CL. Human lymphocytes transcribe the cystic fibrosis transmembrane conductance regulator gene and exhibit CF-defective cAMP-regulated chloride current. J Biol Chem. 1992 Feb 15;267(5):3242-8.
PMID: 1371114BACKGROUNDSchnell A, Jungert J, Klett D, Hober H, Kaiser N, Ruppel R, Geppert A, Tremel C, Sobel J, Plattner E, Schmitt-Grohe S, Zirlik S, Strobel D, Neurath MF, Knieling F, Rauh M, Woelfle J, Hoerning A, Regensburger AP. Increase of liver stiffness and altered bile acid metabolism after triple CFTR modulator initiation in children and young adults with cystic fibrosis. Liver Int. 2023 Apr;43(4):878-887. doi: 10.1111/liv.15544. Epub 2023 Feb 28.
PMID: 36797990DERIVED
Biospecimen
Serum Peripheral Mononuclear Blood Cells (PBMCs)
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- CASE CONTROL
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Principle Investigator
Study Record Dates
First Submitted
September 27, 2022
First Posted
October 12, 2022
Study Start
December 30, 2020
Primary Completion
October 18, 2023
Study Completion
October 18, 2023
Last Updated
October 12, 2022
Record last verified: 2022-10
Data Sharing
- IPD Sharing
- Will not share