№ files_lp_4_process_2_74730
File format: docx Character count: 1412 File size: 21 KB
Year: 2026
Region: Multicenter study
Topic: Cystic fibrosis treatment outcomes
Document type: Clinical study table
Institution: University medical centers
Population: Patients with cystic fibrosis
Treatment groups: Never treated with CFTR modulators; Switched from lumacaftor/ivacaftor to ETI
Parameters measured: Age, sex, sweat chloride, FEV1, FVC, BMI, pancreatic insufficiency, diabetes, chronic infections, CRP, ESR, leukocyte and neutrophil counts, platelets, IgG, albumin, hemoglobin, HbA1c, glucose
Statistical methods: Chi-square, t-test, Mann-Whitney test
Data source: ETI treatment initiation visit
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Correlation between patient-derived intestinal organoids and clinical responses to CFTR modulators in people with cystic fibrosis homozygous for F508del
Authors: Noelia Rodriguez Mier; Senne Cuyx; Kris De Boeck; Marijke Proesmans; Mieke Boon; Lieven Dupont; Kaline Arnauts; Elke De Wachter; Stephanie Van Biervliet; Monique Lequesne; Vicky Nowé; Hedwige Boboli; Anabela Santo Ramalho; François Vermeulen; on behalf of the Belgian Organoid Project
Affiliations: KU Leuven; University Hospital Leuven; University Hospital Brussels; Ghent University Hospital; Antwerp University Hospital; GZA Hospital; CHR Citadelle
Country: Belgium
Research field: Cystic fibrosis; CFTR modulators; organoid research; personalized medicine
Study population: 60 people with cystic fibrosis homozygous for F508del
Genetic focus: F508del CFTR mutation (homozygous)
Interventions studied: Elexacaftor/tezacaftor/ivacaftor; Tezacaftor/ivacaftor
Methods: Patient-derived intestinal organoids from rectal biopsies; forskolin-induced swelling assay; comparison with clinical outcomes
Clinical outcomes assessed: Lung function; sweat chloride levels; body weight; pulmonary exacerbations
Type of document: Scientific research article
Source: PubMed
DOI / Identifier: 40883119
Supplementary material for CFTR activity enhancement by GLPG2222 with and without ivacaftor in FLAMINGO and ALBATROSS trials
Year: 2023
Study type: Randomized clinical trials
Drug investigated: GLPG2222
Combination therapy: With and without ivacaftor
Measured outcomes: Sweat chloride concentration, ppFEV1, FEV1 absolute changes
Study centers: Multiple international hospitals across Belgium, Netherlands, Serbia, Spain, United Kingdom, United States, Australia, Czech Republic, Germany, Ireland
Principal investigators: Scott C Bell, Peter J Barry, Kris De Boeck, Pavel Drevinek, J Stuart Elborn, Barry J Plant, Predag Minić, Eva Van Braeckel, Stijn Verhulst, Karine Muller, Desirée Kanters, Susan Bellaire, Herman de Kock, David E Geller, Katja Conrath, Olivier Van de Steen, Kors van der Ent
Participant groups: FLAMINGO (N=59), ALBATROSS (+ ivacaftor; N=37)
Safety population: Patients with cystic fibrosis and associated comorbidities
Data presentation: Supplementary figures and tables including waterfall plots and mean FEV1 changes
Topics: modulator ETI CFTR