This phase II trial compares the effect of belumosudil to a placebo in treating patients with chronic graft versus host disease. Chronic graft versus host disease remains a major complication of stem cell transplantation and can involve multiple organ systems. Belumosudil is a ROCK2 selective inhibitor that works to reduce the immune system response causing the chronic graft versus host disease. Giving belumosudil may better treat patients with chronic graft versus host disease and prevent the need for starting additional immune suppressive medications.
At least one diagnostic or distinctive cGVHD manifestation(s), with a clinical diagnosis of cGVHD,but patients do not need to meet National Institute of Health (NIH) criteria for cGVHD
If eye involvement only, cGVHD must be confirmed on exam by an ophthalmologist or optometrist
No new immune suppressive therapy added within preceding 2 weeks prior to study enrollment for any indication
Continuation of agents previously given as either GVHD prophylaxis or acute/late acute GVHD therapy are permitted. Modification of dose of these agents for targeting of therapeutic drug levels is permitted, as are decreases in existing prednisone or prednisone equivalent dose based on routine clinical tapering practices. Increases in prednisone or prednisone equivalents are not allowed in the 2 weeks prior to enrollment
Any systemic immune suppressive treatment for cGVHD (topical or local therapies are allowed)
Plan to start systemic immune suppressive therapy for cGVHD or increase steroid dose within 14 days after planned start of study medication
0.25 mg/kg/day or higher prednisone or prednisone equivalent dose at time of screening
History of non-compliance that in the investigator's opinion would interfere with study participation
Parallel
Belumosudil
DrugGiven PO
Biospecimen Collection
Procedure/SurgeryUndergo blood sample collection
Electronic Health Record Review
Other interventionAncillary studies
Placebo Administration
DrugGiven PO
Time to start of subsequent systemic immune suppressive treatment for chronic graft versus host disease (cGVHD)
Systemic therapies include any systemic agent given for a cGVHD indication, including extracorporeal photopheresis. Will use Gray's test. Point estimates of new systemic immunosuppressive use will be obtained using cumulative incidence estimates.
Event-free survival
Point estimates will be obtained using the method of Kaplan and Meier and the log-rank test will be used to assess the difference between treatment groups.
Overall survival
Point estimates will be obtained using the method of Kaplan and Meier and the log-rank test will be used to assess the difference between treatment groups.
Rate of relapse
Point estimates will be obtained using the method of Kaplan and Meier and the log-rank test will be used to assess the difference between treatment groups.
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Fred Hutchinson Cancer Center
Lead sponsor
Sanofi
Collaborator
