Macitentan for the treatment of idiopathic pulmonary fibrosis: The randomised controlled MUSIC trial

Ganesh Raghu, Rachel Million-Rousseau, Adele Morganti, Loïc Perchenet, Juergen Behr, N. Goh, A. Glanville, M. Musk, P. Hopkins, D. C. Lien, C. Chan, J. D. Rolf, P. Wilcox, P. G. Cox, H. Manganas, V. Cottin, D. Valeyre, B. Walleart, S. Andreas, C. NeurohrA. Guenther, N. Schönfeld, A. Koch, M. Kramer, R. Breuer, I. Ben-Dov, G. Fink, Y. Schwarz, C. Albera, M. Confalonieri, C. Saltini, S. Harari, M. Flezar, M. Greenblatt, G. J. Ras, F. Morell, J. L. Alvarez-Sala, A. Xaubet, A. Sueiro, M. J. Linares, M. Sköld, O. Kayacan, N. Mogulkoc, A. Chan, J. Chapman, J. Parambil, N. Ettinger, J. Golden, K. C. Meyer, J. J. Swigris, G. L. Yung, D. Antin-Ozerkis, P. K. Mohabir, L. J. Wesselius, J. De Andrade, F. Cordova, Z. Safdar, M. Wencel

Research output: Contribution to journalArticlepeer-review

172 Scopus citations


Idiopathic pulmonary fibrosis is a progressive, fatal disease. This prospective, randomised, double-blind, multicentre, parallel-group, placebo-controlled phase II trial (NCT00903331) investigated the efficacy and safety of the endothelin receptor antagonist macitentan in idiopathic pulmonary fibrosis. Eligible subjects were adults with idiopathic pulmonary fibrosis of <3 years duration and a histological pattern of usual interstitial pneumonia on surgical lung biopsy. The primary objective was to demonstrate that macitentan (10 mg once daily) positively affected forced vital capacity versus placebo. Using a centralised system, 178 subjects were randomised (2:1) to macitentan (n=119) or placebo (n=59). The median change from baseline up to month 12 in forced vital capacity was -0.20 L in the macitentan arm and -0.20 L in the placebo arm. Overall, no differences between treatments were observed in pulmonary function tests or time to disease worsening or death. Median exposures to macitentan and placebo were 14.5 months and 15.0 months, respectively. Alanine and/or aspartate aminotransferase elevations over three times upper limit of normal arose in 3.4% of macitentan-treated subjects and 5.1% of placebo recipients. In conclusion, the primary objective was not met. Long-term exposure to macitentan was well tolerated with a similar, low incidence of elevated hepatic aminotransferases in each treatment group.

Original languageEnglish (US)
Pages (from-to)1622-1632
Number of pages11
JournalEuropean Respiratory Journal
Issue number6
StatePublished - Dec 1 2013

ASJC Scopus subject areas

  • Pulmonary and Respiratory Medicine


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