Chronic graft-versus-host disease (cGvHD) is a serious condition that can happen after a stem cell or bone marrow transplant. The donor's immune cells attack the patient's body, causing inflammation, pain, and damage to organs like the skin, liver, or lungs. For patients with moderate to severe cGvHD who don't improve with or can't tolerate standard front line therapy with steroids, there's a significant unmet need. Steroid-refractory cGvHD is hard to treat, with limited effective options, often leading to ongoing symptoms and reduced quality of life. This Phase II study tests a new treatment combining two oral drugs, ruxolitinib and belumosudil, for these patients. Both drugs have helped cGvHD individually, but this trial explores if they work better together. For the first 28 days (Cycle 1), patients take ruxolitinib (10 mg twice daily). From Cycle 2, they add belumosudil (200 mg once or twice daily, depending on other medications) for 48 weeks (12 cycles) unless their condition worsens or side effects become intolerable. Follow-up visits occur 30 days and 6 months after treatment ends to check health status. The study is non-randomized (all get the same treatment) and open-label (patients and doctors know the drugs used). It aims to see if this combination better controls cGvHD in patients where steroids failed. This could offer hope for better symptom management and improved quality of life for those with limited treatment options.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
63
Patients receive ruxolitinib (10 mg twice daily) alone for one 28-day cycle, then add belumosudil (200 mg once or twice daily if on a PPI) from cycles 2-12 (48 weeks total), unless cGvHD progresses or side effects become intolerable.
Patients receive ruxolitinib (10 mg twice daily) alone for one 28-day cycle, then add belumosudil (200 mg once or twice daily if on a PPI) from cycles 2-12 (48 weeks total), unless cGvHD progresses or side effects become intolerable.
University Health Network
Toronto, Ontario, Canada
Efficacy will be assessed using overall response rate (ORR) as per the NIH cGvHD Consensus Response Criteria at 24 weeks of combination treatment. Tolerability and safety will be assessed by the incidence and severity of adverse events (AEs)
The proportion of responders and its 95% confidence interval (using exact binomial methods) will be calculated.
Time frame: Enrollment to 24 and 48 weeks after combination therapy.
Evaluate the response by assessing ORR at 48 weeks, Failure-free survival (FFS) at 24 & 48 weeks, and durable response rate in patients who achieve complete or partial response at 52 weeks. GvHD symptom burden is measured using modified Lee Symptom Scale
GvHD symptom burden improvement, measured serially measured by the modified Lee Symptom Scale, will be evaluated using repeated measure based on the general linear model. If p-value is less than 0.05, it demonstrates statistically significant change over time within subject.
Time frame: Enrollment to 24 and 48 weeks after combination therapy.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.