Design of a randomised, placebo-controlled clinical trial of nintedanib in patients with systemic sclerosis-associated interstitial lung disease (SENSCIS™)

Clinical and Experimental Rheumatology

Volumn 35, Issue , 2017, Pages S75-S81

  Distler, Oliver ^a   Kevin K Brown, ^b   Distler, Jörg H W ^c   Assassi, Shervin ^d   Maher, Toby M ^e,f   Cottin, Vincent ^g   Varga, John ^h   Coeck, Carl ⁱ   Gahlemann, Martina ^j   Sauter, Wiebke ^k   Schmidt, Hendrik ^k   Highland, Kristin B ^l  

^a UNIVERSITY HOSPITAL ZURICH   (Switzerland)

^b DEPARTMENT OF MEDICINE   (United States)

^c UNIVERSITY OF ERLANGEN NUREMBERG   (Germany)

^d UNIVERSITY OF TEXAS MEDICAL SCHOOL   (United States)

^e ROYAL BROMPTON HOSPITAL   (United Kingdom)

^f NATIONAL HEART AND LUNG INSTITUTE   (United Kingdom)

^g UNIVERSITÉ LYON 1   (France)

^h NORTHWESTERN UNIVERSITY   (United States)

ⁱ Boehringer Ingelheim   (Belgium)

^j Boehringer Ingelheim (Schweiz) Gmbh   (Switzerland)

^k BOEHRINGER INGELHEIM PHARMA GMBH AND CO KG   (Germany)

^l LERNER RESEARCH INSTITUTE   (United States)

more..

Author keywords

Connective tissue diseases; Quality of life; Respiratory function tests; Systemic sclerosis

Indexed keywords

LOPERAMIDE; METHOTREXATE; MYCOPHENOLIC ACID; NINTEDANIB; PLACEBO; PREDNISONE; INDOLE DERIVATIVE;

ADULT; ARTICLE; COMPUTER ASSISTED TOMOGRAPHY; CONTROLLED STUDY; DIARRHEA; DRUG DOSE REDUCTION; DRUG EFFICACY; DRUG SAFETY; FORCED VITAL CAPACITY; HUMAN; INTERSTITIAL LUNG DISEASE; LUNG DIFFUSION CAPACITY; MAJOR CLINICAL STUDY; MULTICENTER STUDY; PATHOPHYSIOLOGY; PHASE 3 CLINICAL TRIAL; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; ST. GEORGE RESPIRATORY QUESTIONNAIRE; SYSTEMIC SCLEROSIS; CLINICAL TRIAL; COMPLICATION; DOUBLE BLIND PROCEDURE; METHODOLOGY; PROSPECTIVE STUDY; VITAL CAPACITY;

ADULT; DOUBLE-BLIND METHOD; HUMANS; INDOLES; LUNG DISEASES, INTERSTITIAL; PROSPECTIVE STUDIES; RESEARCH DESIGN; SCLERODERMA, SYSTEMIC; VITAL CAPACITY;

EID: 85031689905     PISSN: 0392856X     EISSN: 1593098X     Source Type: Journal    
DOI: None     Document Type: Article

References (42)

1. VARGA J, ABRAHAM D: Systemic sclerosis: a prototypic multisystem fibrotic disorder. J Clin Invest 2007; 117: 557-67
2. STEEN VD, MEDSGER TA: Changes in causes of death in systemic sclerosis, 1972-2002. Ann Rheum Dis 2007; 66: 940-4
3. TYNDALL AJ, BANNERT B, VONK M et al.: Causes and risk factors for death in systemic sclerosis: a study from the EULAR Scleroderma Trials and Research (EUSTAR) database. Ann Rheum Dis 2010; 69: 1809-15
4. STEELE R, HUDSON M, LO E et al.: Clinical decision rule to predict the presence of interstitial lung disease in systemic sclerosis. Arthritis Care Res (Hoboken) 2012; 64: 519-24
5. van den HOOGEN F, KHANNA D, FRANSEN J et al.: 2013 classification criteria for systemic sclerosis: an American College of Rheumatology/ European League against Rheumatism collaborative initiative. Ann Rheum Dis 2013; 72: 1747-55
6. BARON M, SUTTON E, HUDSON M et al.: The relationship of dyspnoea to function and quality of life in systemic sclerosis. Ann Rheum Dis 2008; 67: 644-50
7. THEODORE AC, TSENG CH, LI N, ELASHOFF RM, TASHKIN DP: Correlation of cough with disease activity and treatment with cyclophosphamide in scleroderma interstitial lung disease: findings from the Scleroderma Lung Study. Chest 2012; 142: 614-21
8. KOWAL-BIELECKA O, LANDEWé R, AVOUAC J et al.: EULAR recommendations for the treatment of systemic sclerosis: a report from the EULAR Scleroderma Trials and Research group (EUSTAR). Ann Rheum Dis 2009; 68: 620-8
9. HOYLES RK, ELLIS RW, WELLSBURY J et al.: A multicenter, prospective, randomized, double-blind, placebo-controlled trial of corticosteroids and intravenous cyclophosphamide followed by oral azathioprine for the treatment of pulmonary fibrosis in scleroderma. Arthritis Rheum 2006; 54: 3962-70
10. TASHKIN DP, ELASHOFF R, CLEMENTS PJ et al.: Cyclophosphamide versus placebo in scleroderma lung disease. N Engl J Med 2006; 354: 2655-66
11. TASHKIN DP, ROTH MD, CLEMENTS PJ et al.: Mycophenolate mofetil versus oral cyclophosphamide in scleroderma-related interstitial lung disease (SLS II): a randomised controlled, double-blind, parallel group trial. Lancet Respir Med 2016; 4: 708-19
12. KOWAL-BIELECKA O, FRANSEN J, AVOUAC J et al.: Update of EULAR recommendations for the treatment of systemic sclerosis. Ann Rheum Dis 2016. Epub ahead of print
13. van LAAR JM, FARGE D, SONT JK et al.: Autologous hematopoietic stem cell transplantation vs intravenous pulse cyclophosphamide in diffuse cutaneous systemic sclerosis: a randomized clinical trial. JAMA 2014; 311: 2490-8
14. WEILL D, BENDEN C, CORRIS PA et al.: A consensus document for the selection of lung transplant candidates: 2014 an update from the Pulmonary Transplantation Council of the International Society for Heart and Lung Transplantation. J Heart Lung Transplant 2015; 34: 1-15
15. HILBERG F, ROTH GJ, KRSSAK M et al.: BIBF 1120: triple angiokinase inhibitor with sustained receptor blockade and good antitumor efficacy. Cancer Res 2008; 68: 4774-82
16. WOLLIN L, MAILLET I, QUESNIAUX V et al.: Antifibrotic and anti-inflammatory activity of the tyrosine kinase inhibitor nintedanib in experimental models of lung fibrosis. J Pharmacol Exp Ther 2014; 349: 209-20
17. WOLLIN L, WEX E, PAUTSCH A et al.: Mode of action of nintedanib in the treatment of idiopathic pulmonary fibrosis. Eur Respir J 2015; 45: 1434-45
18. BOEHRINGER INGELHEIM PHARMACEUTICALS, INC. (2014). OFEV™ (nintedanib) prescribing information. Available at: http://bidocs.boehringer-ingelheim. com/BIWebAccess/ViewServlet. ser?docBase=renetnt&folderPath=/ Prescribing+Information/PIs/Ofev/ofev.pdf. Accessed 1 June 2016
19. BOEHRINGER INGELHEIM (2015). OFEV (nintedanib) Summary of Product Characteristics. February 2015. Available at: http://www.ema.europa.eu/ema/index. jsp?curl=pages/medicines/human/medicines/ 003821/human_med_001834. jsp&mid=WC0b01ac058001d124. Accessed 1 June 2016
20. RICHELDI L, COSTABEL U, SELMAN M et al.: Efficacy of a tyrosine kinase inhibitor in idiopathic pulmonary fibrosis. N Engl J Med 2011; 365: 1079-87
21. RICHELDI L, DU BOIS RM, RAGHU G et al.: Efficacy and safety of nintedanib in idiopathic pulmonary fibrosis. N Engl J Med 2014; 370: 2071-82
22. USATEGUI A, DEL REY MJ, PABLOS JL: Fibroblast abnormalities in the pathogenesis of systemic sclerosis. Expert Rev Clin Immunol 2011; 7: 491-8
23. BAGNATO G, HARARI S: Cellular interactions in the pathogenesis of interstitial lung diseases. Eur Respir Rev 2015; 24: 102-14
24. SKHIRTLADZE C, DISTLER O, DEES C et al.: Src kinases in systemic sclerosis: central roles in fibroblast activation and in skin fibrosis. Arthritis Rheum 2008; 58: 1475-84
25. MAURER B, DISTLER A, SULIMAN YA et al.: Vascular endothelial growth factor aggravates fibrosis and vasculopathy in experimental models of systemic sclerosis. Ann Rheum Dis 2014; 73: 1880-7
26. DISTLER O, DISTLER JH, SCHEID A et al.: Uncontrolled expression of vascular endothelial growth factor and its receptors leads to insufficient skin angiogenesis in patients with systemic sclerosis. Circ Res 2004; 95: 109-16
27. ALLIPOUR BIRGANI S, MAILäNDER M, WASLE I et al.: Efficient therapy of ischaemic lesions with VEGF121-fibrin in an animal model of systemic sclerosis. Ann Rheum Dis 2016; 75: 1399-406
28. HUANG J, BEYER C, PALUMBO-ZERR K et al.: Nintedanib inhibits fibroblast activation and ameliorates fibrosis in preclinical models of systemic sclerosis. Ann Rheum Dis 2016; 75: 883-90
29. IOANNOU M, PYRPASOPOULOU A, SIMOS G et al.: Upregulation of VEGF expression is associated with accumulation of HIF-1a in the skin of naïve scleroderma patients. Mod Rheumatol 2013; 23: 1245-8
30. LIAKOULI V, CIPRIANI P, MARRELLI A et al.: Angiogenic cytokines and growth factors in systemic sclerosis. Autoimmun Rev 2011; 10: 590-4
31. ASSASSI S, SHARIF R, LASKY RE et al.: Predictors of interstitial lung disease in early systemic sclerosis: a prospective longitudinal study of the GENISOS cohort. Arthritis Res Ther 2010; 12: R166
32. CLEMENTS PJ, LACHENBRUCH PA, SEIBOLD JR et al.: Skin thickness score in systemic sclerosis: an assessment of interobserver variability in 3 independent studies. J Rheumatol 1993; 20: 1892-6
33. JONES PW, QUIRK FH, BAVEYSTOCK CM: The St George's Respiratory Questionnaire. Respir Med 1991; 85: 25-31
34. SCHMIDINGER M: Understanding and managing toxicities of vascular endothelial growth factor (VEGF) inhibitors. EJC Suppl 2013; 11: 172-91
35. CORTE T, BONELLA F, CRESTANI B et al.: Safety, tolerability and appropriate use of nintedanib in idiopathic pulmonary fibrosis. Respir Res 2015; 16: 116
36. KHANNA D, MITTOO S, AGGARWAL R et al.: Connective Tissue Disease-associated Interstitial Lung Diseases (CTD-ILD)-Report from OMERACT CTD-ILD Working Group. J Rheumatol 2015; 42: 2168-71
37. SAKETKOO LA, MITTOO S, HUSCHER D et al.: Connective tissue disease related interstitial lung diseases and idiopathic pulmonary fibrosis: provisional core sets of domains and instruments for use in clinical trials. Thorax 2014; 69: 428-36
38. MERKEL PA, CLEMENTS PJ, REVEILLE JD et al; OMERACT 6: Current status of outcome measure development for clinical trials in systemic sclerosis. Report from OMERACT 6. J Rheumatol 2003; 30: 1630-47
39. FURST DE, CLEMENTS PJ, STEEN VD et al.: The modified Rodnan skin score is an accurate reflection of skin biopsy thickness in systemic sclerosis. J Rheumatol 1998; 25: 84-8
40. SWIGRIS JJ, ESSER D, CONOSCENTI CS, BROWN KK: The psychometric properties of the St George's Respiratory Questionnaire (SGRQ) in patients with idiopathic pulmonary fibrosis: a literature review. Health Qual Life Outcomes 2014; 12: 124
41. SWIGRIS JS, ESSER D, WILSON H et al.: Psychometric properties of the St. George's Respiratory Questionnaire in patients with IPF. Eur Respir J 2017; 18; 49 (1)
42. BERETTA L, SANTANIELLO A, LEMOS A et al.: Validity of the Saint George's Respiratory Questionnaire in the evaluation of the health-related quality of life in patients with interstitial lung disease secondary to systemic sclerosis. Rheumatology (Oxford) 2007; 46: 296-301