Systemic sclerosis & Raynaud's
Belumosudil in diffuse cutaneous systemic sclerosis: a randomized, double-blind, open-label extension, placebo-controlled, phase 2 study
- terminated prematurely
- Terminated prematurely; target enrolment not met
Authors' conclusion
Efficacy signal for belumosudil could not be detected. Signalling pathway modulation analysis supported the mechanism of action of belumosudil. A trend for decreased fibrosis-related biomarkers was observed in the belumosudil-treated group.
Study context
- Population
- Patients with diffuse cutaneous systemic sclerosis (dcSSc) treated with background immunosuppressive therapies31 in open-label period
- Participants
- 35 patients
- Comparison
- Belumosudil 200 mg QD or BID vs placebo, on background immunosuppressive therapies
- Primary endpoint
- CRISS score ≥0.60
- Follow-up
- Week 24
Methodological detailsdouble-blind, placebo-controlled, open-label extension
- Double-blind
- Placebo-controlled
- Open-label extension
Primary result
As reported in the abstract
Each group vs placeboOdds ratio (CI)P value
QD
1.06(0.19–5.82)
P = 0.9472BID
0.39(0.07–2.35)
P = 0.3078Further findings
Fibrosis biomarkers in blood and tissue
- Trends of decreased fibrosis biomarker levels with belumosudil vs placebo
Tissue RNA sequencing in patients with CRISS score ≥0.60
- FOXP3 upregulation and STAT3, IL23A and TGF-β downregulation
Original publication
Belumosudil in diffuse cutaneous systemic sclerosis: a randomized, double-blind, open-label extension, placebo-controlled, phase 2 study. Rheumatology (Oxford). Published 14 March 2025. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →