Testing Two Different Doses of Tiotropium Respimat® in Cystic Fibrosis: Phase 2 Randomized Trial Results
| Autor: | Bradley, Jm, Koker, P, Deng, Q, Moroni Zentgraf, P, Ratjen, F, Geller, De, Elborn, Js, Clements, B, Greville, H, Middleton, P, Serisier, D, van Asperen, P, Dupont, L, Knoop, C, Malfroot, A, Belleguic, C, Chiron, R, Derelle, J, Dominique, S, Dusser, D, Ginies, Jl, Guillot, M, Horeau-Langlard, D, Le Bourgeois, M, Leroy, S, Marguet, C, Munck, A, Pautard, Jc, Reix, P, Uffredi, Ml, Ballmann, M, Bargon, J, Fischer, R, Herth, F, Heuer, He, Griese, M, Kamin, W, Kohlhäufl, M, Nährlich, L, Riethmüller, J, Sorichter, S, Wagner, T, Bennett, L, Crawford, M, Crossley, J, Derry, D, Doull, I, Elborn, J, Everard, M, Groggins, C, Johnson, M, Lee, T, Rayner, R, Reid, A, Smyth, A, Southern, K, Thomas, D, Walshaw, M, Whitehouse, J, Braggion, C, De Rose, V, Gagliardini, R, Minicucci, L, Reijers, M, Tiddens, Ha, O'Carroll, M, Wong, J, Barreto, C, Cardim, P, Loureiro, M, Rocha, H, Vaz, Ml, Asherova, I, Chepurnaya, M, Chuchalin, Ag, Ilkovich, M, Kapranov, N, Neretina, A, Orlov, A, Simonova, O, Ahrens, R, Anbar, R, Biller, J, Carveth, H, Daines, C, Fiel, S, Flume, P, Froh, D, 3rd, Harris J., Howenstine, M, Millard, Dl, Nasr, S, Parker, H, Pian, M, Royall, J, Sabogal, C, Salathe, M, Sannuti, A, Schaeffer, D, Strausbaugh, S, Toder, D, Reyes, S, Whittaker, L, Zanni, R. |
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| Jazyk: | angličtina |
| Rok vydání: | 2014 |
| Předmět: |
Male
Respimat Cystic Fibrosis Pulmonology lcsh:Medicine Cystic fibrosis Pediatrics Pulmonary function testing law.invention Clinical trials Randomized controlled trial law Bronchodilator Forced Expiratory Volume lcsh:Science Child Medicine(all) Multidisciplinary Agricultural and Biological Sciences(all) Area under the curve Tiotropium bromide Respiratory Function Tests Research Design Anesthesia Female Genetic Dominance Phase II clinical investigation medicine.drug Research Article Adult medicine.medical_specialty Adolescent General Science & Technology medicine.drug_class Clinical Research Design Pediatric Pulmonology Scopolamine Derivatives Placebo Research and Analysis Methods Young Adult Double-Blind Method MD Multidisciplinary Administration Inhalation medicine Genetics Humans Tiotropium Bromide Medicine and health sciences Autosomal Recessive Traits business.industry Biochemistry Genetics and Molecular Biology(all) lcsh:R Biology and Life Sciences Human Genetics medicine.disease Fibrosis respiratory tract diseases Clinical medicine Physical therapy Tiotropium Cystic Fibrosis Study Group lcsh:Q business Developmental Biology |
| Zdroj: | PLoS ONE Bradley, J M, Koker, P, Deng, Q, Moroni-Zentgraf, P, Ratjen, F, Geller, D E & Elborn, S 2014, ' Testing two different doses of tiotropium Respimat® in cystic fibrosis: Phase 2 randomized trial results ', PLoS ONE, vol. 9, no. 9, e106195, pp. 1-9 . https://doi.org/10.1371/journal.pone.0106195 PLoS ONE, Vol 9, Iss 9, p e106195 (2014) |
| ISSN: | 1932-6203 0073-7100 |
| DOI: | 10.1371/journal.pone.0106195 |
| Popis: | Background Tiotropium is a once-daily, long-acting anticholinergic bronchodilator with the potential to alleviate airway obstruction in cystic fibrosis. Our objective was to evaluate the efficacy and safety of 2.5 and 5 µg once-daily tiotropium delivered via the Respimat Soft Mist Inhaler vs. placebo in people with cystic fibrosis. Methods This phase 2, 12-week, randomized, double-blind, placebo-controlled parallel-group study of tiotropium Respimat as add-on to usual cystic fibrosis maintenance therapy included people with cystic fibrosis with pre-bronchodilator forced expiratory volume in 1 second (FEV1) ≥25% predicted. Co-primary efficacy end points were change from baseline in percent-predicted FEV1 area under the curve from 0 to 4 hours (FEV1 AUC0–4h), and trough FEV1 at the end of week 12. Findings A total of 510 subjects with cystic fibrosis aged 5–69 years were randomized. Both doses of tiotropium resulted in significant improvement compared with placebo in the co-primary efficacy end points at the end of week 12 (change from baseline in percent-predicted FEV1 AUC0–4h: 2.5 µg: 2.94%, 95% confidence interval 1.19–4.70, p = 0.001; 5 µg: 3.39%, 95% confidence interval 1.67–5.12, p = 0.0001; in percent-predicted trough FEV1∶2.5 µg: 2.24%, p = 0.2; 5 µg: 2.22%, p = 0.02). There was a greater benefit with tiotropium 5 vs. 2.5 µg. No treatment-related adverse events or unexpected safety findings were observed in patients taking tiotropium. Conclusions Tiotropium significantly improved lung function in people with cystic fibrosis. The improvement was greater with the higher dose than the lower dose, with no difference in adverse events. Trial Registration ClinicalTrials.gov NCT00737100 EudraCT 2008-001156-43. |
| Databáze: | OpenAIRE |
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