Pirfenidone in patients with unclassifiable progressive fibrosing interstitial lung disease: design of a double-blind, randomised, placebo-controlled phase II trial

被引:50
作者
Maher, Toby M. [1 ,2 ]
Corte, Tamera J. [3 ,4 ]
Fischer, Aryeh [5 ]
Kreuter, Michael [6 ,7 ]
Lederer, David J. [8 ]
Molina-Molina, Maria [9 ,10 ]
Axmann, Judit [11 ]
Kirchgaessler, Klaus-Uwe [11 ]
Cottin, Vincent [12 ,13 ]
机构
[1] Royal Brompton Hosp, NIHR Resp Clin Res Facil, London, England
[2] Imperial Coll London, Natl Heart & Lung Inst, Fibrosis Res Grp, London, England
[3] Royal Prince Alfred Hosp, Dept Resp Med, Camperdown, NSW, Australia
[4] Univ Sydney, Med Sch, Camperdown, NSW, Australia
[5] Univ Colorado, Dept Med, Sch Med, Aurora, CO USA
[6] Heidelberg Univ, Member German Ctr Lung Res, Thoraxklin, Ctr Interstitial & Rare Lung Dis, Heidelberg, Germany
[7] Heidelberg Univ, Member German Ctr Lung Res, Thoraxklin, Dept Pulmonol & Crit Care Med, Heidelberg, Germany
[8] Columbia Univ, Med Ctr, Div Pulm Allergy & Crit Care Med, New York, NY USA
[9] Univ Hosp Bellvitge, Inst Invest Biomed Bellvitge IDIBELL, Barcelona, Spain
[10] Ctr Invest Biomed Red Enfermedades Resp CIBERES, Madrid, Spain
[11] F Hoffmann La Roche Ltd, Basel, Switzerland
[12] Louis Pradel Hosp, Natl Reference Ctr Rare Pulm Dis, Lyon, France
[13] Univ Claude Bernard Lyon 1, Hosp Civils Lyon, Lyon, France
来源
BMJ OPEN RESPIRATORY RESEARCH | 2018年 / 5卷 / 01期
关键词
D O I
10.1136/bmjresp-2018-000289
中图分类号
R56 [呼吸系及胸部疾病];
学科分类号
摘要
Introduction Despite extensive multidisciplinary team (MDT) assessment, some patients have interstitial lung disease (ILD) that is considered unclassifiable (uILD), for which there are currently no approved treatments. This study will assess the efficacy and safety of the antifibrotic pirfenidone in treating uILD. Methods and analysis This double-blind, randomised, placebo-controlled phase II trial is enrolling adults with fibrosing ILD, including uILD that fulfils proposed research criteria for interstitial pneumonia with autoimmune features (IPAF), that cannot be classified with moderate or high confidence to any category of ILD following MDT discussion. Study participants must have >10% fibrosis on high-resolution CT scan within the previous 12 months, forced vital capacity (FVC) >= 45% and diffusing capacity of the lung for carbon monoxide >= 30% of predicted values. Study participants will be randomised to receive 801 mg pirfenidone or placebo three times daily for 24 weeks. The efficacy of pirfenidone vs placebo will be assessed by daily measurement of FVC using a handheld spirometer over the treatment period. Other functional parameters, patient-reported outcomes, samples for biomarker analysis and safety endpoints will be collected. Additionally, the study will assess the efficacy and safety of pirfenidone with and without concomitant mycophenolate mofetil treatment and in study participants with or without IPAF. Ethics and dissemination This trial is being conducted in accordance with the International Conference on Harmonisation E6 guideline for Good Clinical Practice, Declaration of Helsinki and local laws for countries in which the research is conducted.
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页数:10
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