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Ruxolitinib Versus Prednisone for the Treatment of Patients with Chronic Graft vs Host Disease Needing Systemic Therapy
Trial Status: active
This phase II trial compares the effect of ruxolitinib versus standard of care prednisone for the treatment of patients with chronic graft versus host disease (cGVHD) needing systemic therapy. GVHD is a common problem that occurs after allogeneic hematopoietic cell transplant (HCT). Sometimes the transplanted cells from a donor can attack the body's normal cells (called graft-versus-host disease). Giving ruxolitinib or prednisone the transplant may be an effective treatment. Ruxolitinib blocks proteins called JAK 1 and 2 which may lead to a reduction in inflammation. Prednisone is in a class of medications called corticosteroids. It is used to reduce inflammation and lower the body's immune response. Giving ruxolitinib may be effective in treating patients with cGVHD needing systemic therapy.
Inclusion Criteria
Age ≥ 18 years
Karnofsky performance status ≥ 60%
Patients with a diagnosis of chronic GVHD per NIH diagnostic criteria who are in need for first systemic therapy as per treating physician’s discretion, overlap chronic GVHD will be allowed
No new immune suppressive therapy added within preceding 2 weeks prior to study enrolment.
* Continuation of agents previously given as either GVHD prophylaxis or acute GVHD therapy are permitted
Able to take oral medications
Absolute neutrophil count ≥ 1,000/mcL
Platelets ≥ 30,000/mcL
Hemoglobin ≥ 7 g/dL
Bilirubin ≤ 3 times institutional upper limit of normal (ULN) unless attributable to GVHD
Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of study drug administration
Ability to understand and the willingness to sign a written informed consent document
Exclusion Criteria
Previously treated with systemic immune suppressive therapy for chronic GVHD (where the indication for start of that systemic immune suppressive therapy was chronic GVHD)
Patients with clinically significant or uncontrolled cardiovascular disease, including unstable angina, acute myocardial infarction, or stroke within 6 months, New York Heart Association class III or IV heart failure will be excluded
Relapse malignancy post- transplant
Active hepatitis B, hepatitis C and HIV will be excluded
Any uncontrolled infection at the time if enrollment will be excluded
History of allergic reactions attributed to compounds of similar chemical or biologic composition to ruxolitinib
Participants with psychiatric illness/social situations that would limit compliance with study requirements
Pregnant women and lactating women are excluded from this study because of the potential for teratogenic or abortifacient effects and an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with ruxolitinib, breastfeeding should be discontinued if the mother is treated with ruxolitinib
Current or history of active tuberculosis
Additional locations may be listed on ClinicalTrials.gov for NCT06660355.
Locations matching your search criteria
United States
Florida
Miami
University of Miami Miller School of Medicine-Sylvester Cancer Center
I. To compare treatment success (National Institute of Health [NIH] complete response [CR]/partial response [PR] without treatment failure [defined by death and next line systemic immune suppressive therapy]) at (cycle 7 day 1 [C7D1]) between ruxolitinib and prednisone treatment arms.
SECONDARY OBJECTIVE:
I. To compare treatment success rate (NIH CR/PR without treatment failure (defined by death and next-line systemic immune suppressive therapy) + <0.25mg/kg prednisone dose) at (C7D1) between ruxolitinib and prednisone treatment arms.
EXPLORATORY/TERTIARY OBJECTIVES:
I. To determine overall response rate at C7D1 (CR + PR) using NIH consensus criteria.
II. To determine time to second line therapy and failure free survival.
III. To examine best response rate within 6 months (by C7D1).
IV. To describe dose and duration of prednisone therapy.
V. To study patient-reported outcomes (inclusive of symptom score, patient-reported treatment response, and quality of life).
VI. Determine the cumulative prednisone exposure mg/kg by 6 months (C7D1) and compare across study arms.
OUTLINE: Patients are assigned to 1 of 2 arms.
ARM I: Patients receive ruxolitinib orally (PO) twice daily (BID) on days 1-28 of each cycle. Cycles repeat every 28 days for 6 cycles but may continue for up to 24 cycles for responding patients in the absence of progressive disease or unacceptable toxicity. Patients with complete response, starting at cycle 7, may taper by taking a smaller dose of ruxolitinib PO BID for 8 weeks, followed by ruxolitinib daily (QD) for 8 weeks. Patients undergo blood sample collection throughout the study.
ARM II: Patients receive prednisone PO QD for at least 3-6 weeks, depended upon clinical improvement per standard of care. Patients undergo blood sample collection throughout the study.
After completion of study treatment, patients are followed up at 30 days.
