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Riociguat in patients with early diffuse cutaneous systemic sclerosis (RISE-SSc): open-label, long-term extension of a phase 2b, randomised, placebo-controlled trial
O. Distler, Y. Allanore, CP. Denton, M. Kuwana, M. Matucci-Cerinic, JE. Pope, T. Atsumi, R. Bečvář, L. Czirják, E. Hachulla, T. Ishii, O. Ishikawa, SR. Johnson, E. De Langhe, C. Stagnaro, V. Riccieri, E. Schiopu, RM. Silver, V. Smith, V. Steen,...
Language English Country England, Great Britain
Document type Clinical Trial, Phase II, Journal Article, Randomized Controlled Trial
- MeSH
- Scleroderma, Diffuse * drug therapy MeSH
- Humans MeSH
- Patients MeSH
- Pyrazoles adverse effects MeSH
- Pyrimidines * MeSH
- Research Design MeSH
- Check Tag
- Humans MeSH
- Male MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
- Clinical Trial, Phase II MeSH
- Randomized Controlled Trial MeSH
BACKGROUND: The phase 2b Riociguat Safety and Efficacy in Patients with Diffuse Cutaneous Systemic Sclerosis (RISE-SSc) trial investigated riociguat versus placebo in early diffuse cutaneous systemic sclerosis. The long-term extension evaluated safety and exploratory treatment effects for an additional year. METHODS: Patients were enrolled to RISE-SSc between Jan 15, 2015, and Dec 8, 2016. Those who completed the 52-week, randomised, parallel-group, placebo-controlled, double-blind phase were eligible for the long-term extension. Patients originally assigned to riociguat continued therapy (riociguat-riociguat group). Those originally assigned to placebo were switched to riociguat (placebo-riociguat group), adjusted up to 2·5 mg three times daily in a 10-week, double-blind dose-adjustment phase, followed by an open-label phase. Statistical analyses were descriptive. Safety including adverse events and serious adverse events was assessed in the long-term safety analysis set (all patients randomly assigned and treated with study medication in the double-blind phase who continued study medication in the long-term extension). The RISE-SSc trial is registered with ClinicalTrials.gov, NCT02283762. FINDINGS: In total, 87 (72%) of 121 patients in the main RISE-SSc study entered the long-term extension (riociguat-riociguat, n=42; placebo-riociguat, n=45). 65 (75%) of 87 patients were women, 22 (25%) were men, and 62 (71%) were White. Overall, 82 (94%) of 87 patients in the long-term extension had an adverse event; most (66 [76%] of 87) were of mild to moderate severity, with no increase in pulmonary-related serious adverse events in patients with interstitial lung disease. INTERPRETATION: No new safety signals were observed with long-term riociguat in patients with early diffuse cutaneous systemic sclerosis. Study limitations include the absence of a comparator group in this open-label extension study. FUNDING: Bayer and Merck Sharp & Dohme.
Clinical Research Innovation and Education Center Tohuko University Sendai Japan
Department of Allergy and Rheumatology Nippon Medical School Tokyo Japan
Department of Experimental and Clinical Medicine University of Florence Florence Italy
Department of Internal Medicine Ghent University Hospital Ghent Belgium
Department of Rheumatology and Immunology Medical School University of Pécs Pécs Hungary
Department of Rheumatology CHU Bordeaux University Hospital Bordeaux France
Department of Rheumatology Ghent University Hospital Ghent Belgium
Department of Rheumatology St Vincent's Hospital Melbourne VIC Australia
Department of Rheumatology University Hospital Zurich University of Zurich Zurich Switzerland
Department of Rheumatology University of Debrecen Debrecen Hungary
Division of Medicine Centre for Rheumatology UCL London UK
Division of Rheumatology and Immunology Medical University of South Carolina Charleston SC USA
Division of Rheumatology Georgetown University Washington DC USA
Ishii Hospital Division of Dermatology Isezaki Gunma Japan
Medical College of Georgia at Augusta University Augusta GA USA
Research and Development Bayer Wuppertal Germany
Rheumatology A Department Cochin Hospital APHP Paris Descartes University Paris France
Rheumatology Unit Department of Clinical and Experimental Medicine University of Pisa Pisa Italy
Schulich School of Medicine Division of Rheumatology University of Western Ontario London ON Canada
Unit for Molecular Immunology and Inflammation VIB Inflammation Research Center Ghent Belgium
Unit of Immunology Rheumatology Allergy and Rare Diseases IRCCS San Raffaele Hospital Milan Italy
References provided by Crossref.org
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- $a Distler, Oliver $u Department of Rheumatology, University Hospital Zurich, University of Zurich, Zurich, Switzerland. Electronic address: oliver.distler@usz.ch
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