Tiotropium Respimat® in cystic fibrosis: Phase 3 and Pooled phase 2/3 randomized trials

Felix Ratjen; Paul Koker; David E. Geller; Berengere Langellier-Cocteaux; Florence Le Maulf; Sabine Kattenbeck; Petra Moroni-Zentgraf; J. Stuart Elborn

doi:10.1016/j.jcf.2015.03.004

Tiotropium Respimat® in cystic fibrosis: Phase 3 and Pooled phase 2/3 randomized trials

Felix Ratjen^*, Paul Koker, David E. Geller, Berengere Langellier-Cocteaux, Florence Le Maulf, Sabine Kattenbeck, Petra Moroni-Zentgraf, J. Stuart Elborn

^*Corresponding author for this work

Research output: Contribution to journal › Article › peer-review

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Abstract

Background: Tiotropium Respimat® improved lung function in a phase 2 trial in patients with cystic fibrosis (CF). We investigated its efficacy and safety in a phase 3 trial, including a pre-specified pooled analysis of the phase 2 and 3 trials.

Methods: 12-week, randomized, double-blind, placebo-controlled trial of tiotropium Respimat® 5. μg once daily in patients with CF (N = 463).

Results: Co-primary efficacy endpoints showed no statistical difference between tiotropium and placebo: percent-predicted forced expiratory volume in 1s (FEV₁) area under the curve from 0-4h (AUC_0-4h) (95% CI): 1.64% (0.27,3.55; p=0.092); percent-predicted trough FEV₁ (95% CI) 1.40% (0.50,3.30; p=0.15). Adverse events were similar between groups. Pooled phase 2/3 trial results showed a treatment difference in favor of tiotropium: percent-predicted FEV₁ AUC_0-4h (95% CI): 2.62% (1.34,3.90).

Conclusion: Tiotropium was well tolerated in patients with CF; lung function improvements compared with placebo were not statistically significant in the phase 3 trial. Clinical trials: These studies are registered with clinical trial identifier numbers NCT00737100 and NCT01179347NCT00737100NCT01179347. These studies are also registered with the EudraCT number: 2008-001156-43 and 2010-019802-17.

Original language	English
Pages (from-to)	608-614
Number of pages	7
Journal	Journal of Cystic Fibrosis
Volume	14
Issue number	5
Early online date	26 Mar 2015
DOIs	https://doi.org/10.1016/j.jcf.2015.03.004
Publication status	Published - Sept 2015

Bibliographical note

Keywords

Bronchodilator
Cystic fibrosis
Respimat
Tiotropium

ASJC Scopus subject areas

Pulmonary and Respiratory Medicine
Pediatrics, Perinatology, and Child Health

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Tiotropium Respimat® in cystic fibrosis: Phase 3 and Pooled phase 2/3 randomized trials
© 2014 The Authors
Submitted manuscript, 480 KBLicence: Unspecified

Cite this

@article{973bb8b272d7477d98d0a7bc22e532a8,

title = "Tiotropium Respimat{\textregistered} in cystic fibrosis: Phase 3 and Pooled phase 2/3 randomized trials",

abstract = "Background: Tiotropium Respimat{\textregistered} improved lung function in a phase 2 trial in patients with cystic fibrosis (CF). We investigated its efficacy and safety in a phase 3 trial, including a pre-specified pooled analysis of the phase 2 and 3 trials. Methods: 12-week, randomized, double-blind, placebo-controlled trial of tiotropium Respimat{\textregistered} 5. μg once daily in patients with CF (N = 463). Results: Co-primary efficacy endpoints showed no statistical difference between tiotropium and placebo: percent-predicted forced expiratory volume in 1s (FEV1) area under the curve from 0-4h (AUC0-4h) (95% CI): 1.64% (0.27,3.55; p=0.092); percent-predicted trough FEV1 (95% CI) 1.40% (0.50,3.30; p=0.15). Adverse events were similar between groups. Pooled phase 2/3 trial results showed a treatment difference in favor of tiotropium: percent-predicted FEV1 AUC0-4h (95% CI): 2.62% (1.34,3.90). Conclusion: Tiotropium was well tolerated in patients with CF; lung function improvements compared with placebo were not statistically significant in the phase 3 trial. Clinical trials: These studies are registered with clinical trial identifier numbers NCT00737100 and NCT01179347NCT00737100NCT01179347. These studies are also registered with the EudraCT number: 2008-001156-43 and 2010-019802-17.",

keywords = "Bronchodilator, Cystic fibrosis, Respimat, Tiotropium",

author = "Felix Ratjen and Paul Koker and Geller, {David E.} and Berengere Langellier-Cocteaux and {Le Maulf}, Florence and Sabine Kattenbeck and Petra Moroni-Zentgraf and Elborn, {J. Stuart}",

note = "Copyright {\textcopyright} 2015. Published by Elsevier B.V.",

year = "2015",

month = sep,

doi = "10.1016/j.jcf.2015.03.004",

language = "English",

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T1 - Tiotropium Respimat® in cystic fibrosis: Phase 3 and Pooled phase 2/3 randomized trials

AU - Ratjen, Felix

AU - Koker, Paul

AU - Geller, David E.

AU - Langellier-Cocteaux, Berengere

AU - Le Maulf, Florence

AU - Kattenbeck, Sabine

AU - Moroni-Zentgraf, Petra

AU - Elborn, J. Stuart

PY - 2015/9

Y1 - 2015/9

N2 - Background: Tiotropium Respimat® improved lung function in a phase 2 trial in patients with cystic fibrosis (CF). We investigated its efficacy and safety in a phase 3 trial, including a pre-specified pooled analysis of the phase 2 and 3 trials. Methods: 12-week, randomized, double-blind, placebo-controlled trial of tiotropium Respimat® 5. μg once daily in patients with CF (N = 463). Results: Co-primary efficacy endpoints showed no statistical difference between tiotropium and placebo: percent-predicted forced expiratory volume in 1s (FEV1) area under the curve from 0-4h (AUC0-4h) (95% CI): 1.64% (0.27,3.55; p=0.092); percent-predicted trough FEV1 (95% CI) 1.40% (0.50,3.30; p=0.15). Adverse events were similar between groups. Pooled phase 2/3 trial results showed a treatment difference in favor of tiotropium: percent-predicted FEV1 AUC0-4h (95% CI): 2.62% (1.34,3.90). Conclusion: Tiotropium was well tolerated in patients with CF; lung function improvements compared with placebo were not statistically significant in the phase 3 trial. Clinical trials: These studies are registered with clinical trial identifier numbers NCT00737100 and NCT01179347NCT00737100NCT01179347. These studies are also registered with the EudraCT number: 2008-001156-43 and 2010-019802-17.

AB - Background: Tiotropium Respimat® improved lung function in a phase 2 trial in patients with cystic fibrosis (CF). We investigated its efficacy and safety in a phase 3 trial, including a pre-specified pooled analysis of the phase 2 and 3 trials. Methods: 12-week, randomized, double-blind, placebo-controlled trial of tiotropium Respimat® 5. μg once daily in patients with CF (N = 463). Results: Co-primary efficacy endpoints showed no statistical difference between tiotropium and placebo: percent-predicted forced expiratory volume in 1s (FEV1) area under the curve from 0-4h (AUC0-4h) (95% CI): 1.64% (0.27,3.55; p=0.092); percent-predicted trough FEV1 (95% CI) 1.40% (0.50,3.30; p=0.15). Adverse events were similar between groups. Pooled phase 2/3 trial results showed a treatment difference in favor of tiotropium: percent-predicted FEV1 AUC0-4h (95% CI): 2.62% (1.34,3.90). Conclusion: Tiotropium was well tolerated in patients with CF; lung function improvements compared with placebo were not statistically significant in the phase 3 trial. Clinical trials: These studies are registered with clinical trial identifier numbers NCT00737100 and NCT01179347NCT00737100NCT01179347. These studies are also registered with the EudraCT number: 2008-001156-43 and 2010-019802-17.

KW - Bronchodilator

KW - Cystic fibrosis

KW - Respimat

KW - Tiotropium

U2 - 10.1016/j.jcf.2015.03.004

DO - 10.1016/j.jcf.2015.03.004

M3 - Article

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SN - 1569-1993

VL - 14

SP - 608

EP - 614

JO - Journal of Cystic Fibrosis

JF - Journal of Cystic Fibrosis

IS - 5

ER -

Tiotropium Respimat® in cystic fibrosis: Phase 3 and Pooled phase 2/3 randomized trials

Abstract

Bibliographical note

Keywords

ASJC Scopus subject areas

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