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.
OUTLINE: Patients are randomized to 1 of 2 arms. ARM I: Patients receive belumosudil orally (PO) daily (QD) or twice daily (BID) if taken with strong CYP3A inducers or proton pump inhibitors for days 1 through 28 of each cycle. Cycles repeat every 28 days for a total of 11 cycles, followed by one cycle of tapering prior to discontinuation, in the absence of disease progression or unacceptable toxicity. ARM II: Patients receive a placebo PO QD or BID if taken with strong CYP3A inducers or proton pump inhibitors for days 1 through 28 of each cycle. Cycles repeat every 28 days for a total of 11 cycles, followed by one cycle of tapering prior to discontinuation, in the absence of disease progression or unacceptable toxicity. Patients undergo blood sample collection on study. After completion of study treatment, patients are followed up at 30 days, at 60 days if 12 cycles are completed, and then up to 18 months.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
82
Given PO
Undergo blood sample collection
Ancillary studies
Given PO
Moffitt Cancer Center
Tampa, Florida, United States
RECRUITINGDana-Farber Cancer Institute
Boston, Massachusetts, United States
RECRUITINGMemorial Sloan Kettering Cancer Center
New York, New York, United States
RECRUITINGFred Hutch/University of Washington Cancer Consortium
Seattle, Washington, United States
RECRUITINGTime 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.
Time frame: From first dose of study medication to starting a new systemic immunosuppressive agent for cGVHD therapy, up to 12 months
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.
Time frame: From randomization to death, malignancy relapse or addition of a new systemic immune suppressive therapy, up to 12 months or end of study
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.
Time frame: Up to 12 months or end of study
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.
Time frame: Up to 12 months or end of study
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