Imatinib in active diffuse cutaneous systemic sclerosis: Results of a six-month, randomized, double-blind, placebo-controlled, proof-of-concept pilot study at a single center

2011 ◽  
Vol 63 (11) ◽  
pp. 3547-3551 ◽  
Author(s):  
Janet Pope ◽  
Donna McBain ◽  
Lisa Petrlich ◽  
Sharon Watson ◽  
Louise Vanderhoek ◽  
...  
Keyword(s):  
Systemic Sclerosis ◽  
Pilot Study ◽  
Proof Of Concept ◽  
Single Center ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Diffuse Cutaneous Systemic Sclerosis

scholarly journals Riociguat in patients with early diffuse cutaneous systemic sclerosis (RISE-SSc): randomised, double-blind, placebo-controlled multicentre trial

2020 ◽  
Vol 79 (5) ◽  
pp. 618-625 ◽  
Author(s):  
Dinesh Khanna ◽  
Yannick Allanore ◽  
Christopher P Denton ◽  
Masataka Kuwana ◽  
Marco Matucci-Cerinic ◽  
...  
Keyword(s):  
Systemic Sclerosis ◽  
Vital Capacity ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Potential Efficacy ◽  
Condition Score ◽  
Modified Rodnan Skin Score ◽  
Diffuse Cutaneous Systemic Sclerosis ◽  
Pulmonary Arterial ◽  
Safety Assessments

ObjectivesRiociguat is approved for pulmonary arterial hypertension and has antiproliferative, anti-inflammatory and antifibrotic effects in animal models of tissue fibrosis. We evaluated the efficacy and safety of riociguat in patients with early diffuse cutaneous systemic sclerosis (dcSSc) at high risk of skin fibrosis progression.MethodsIn this randomised, double-blind, placebo-controlled, phase IIb trial, adults with dcSSc of <18 months' duration and a modified Rodnan skin score (mRSS) 10–22 units received riociguat 0.5 mg to 2.5 mg orally three times daily (n=60) or placebo (n=61). The primary endpoint was change in mRSS from baseline to week 52.ResultsAt week 52, change from baseline in mRSS units was –2.09±5.66 (n=57) with riociguat and –0.77±8.24 (n=52) with placebo (difference of least squares means –2.34 (95% CI –4.99 to 0.30; p=0.08)). In patients with interstitial lung disease, forced vital capacity declined by 2.7% with riociguat and 7.6% with placebo. At week 14, average Raynaud’s condition score had improved ≥50% in 19 (41.3%)/46 patients with riociguat and 13 (26.0%)/50 patients with placebo. Safety assessments showed no new signals with riociguat and no treatment-related deaths.ConclusionsRiociguat did not significantly benefit mRSS versus placebo at the predefined p<0.05. Secondary and exploratory analyses showed potential efficacy signals that should be tested in further trials. Riociguat was well tolerated.

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