This phase II trial tests how well ruxolitinib and donor (allogeneic) stem cell transplant works in treating patients with myelofibrosis and an enlarged spleen (splenomegaly). Ruxolitinib is in a class of medications called kinase inhibitors. It works to treat myelofibrosis by blocking the signals that cause cancer cells to multiply and has also been shown to shrink the spleen. Stem cell transplant is standard therapy for myelofibrosis, however, patients with splenomegaly are less likely to benefit from a stem cell transplant. An allogeneic stem cell transplant is when healthy stem cells from a donor are infused into a patient. The transplanted cells may help the patient's bone marrow make more healthy cells and platelets. Ruxolitinib before allogeneic stem cell transplant may be effective in treating myelofibrosis patients with splenomegaly.
Additional locations may be listed on ClinicalTrials.gov for NCT06345495.
Locations matching your search criteria
United States
Texas
Houston
M D Anderson Cancer CenterStatus: Active
Contact: Uday R. Popat
Phone: 713-792-8750
PRIMARY OBJECTIVE:
I. Compare the proportion of patients alive, disease free, engrafted, and without poor graft function at 100 days post-transplant with the historical rate of 45%.
SECONDARY OBJECTIVES:
I. Assess overall survival.
II. Assess progression-free survival.
III. Assess graft versus (vs) host disease relapse free survival.
IV. Assess relapse rate.
V. Assess non-relapse mortality.
VI. Assess time to neutrophil and platelet engraftment.
VII. Assess time to red cell transfusion independence.
VIII. Assess graft failure.
IX. Assess acute and chronic graft versus host disease (GVHD).
X. Assess grade 3 - 5 toxicity.
XI. Assess incidence of poor graft function.
XII. Assess need for growth factors (myeloid or thrombopoietic) at 100 days.
XIII. Spleen assessment at baseline, day -1, at 1, 3, 6, and 12 months post stem cell infusion, and at relapse.
XIV. Assess need for transfusions at 100 days.
XV. Assess time to discontinuation of immunosuppressives.
EXPLORATORY OBJECTIVES:
I. Assess immune reconstitution.
II. Assess cytokine profile.
OUTLINE:
Patients receive ruxolitinib orally (PO) twice daily (BID) on days -22 to 60 then once daily (QD) on days 61-80. Patients receive busulfan intravenously (IV) over 180 minutes on days -20, -13 and -6 to -3 and fludarabine IV over 1 hour on days -6 to -3. Patients undergo allogeneic stem cell transplant over 1-2 hours on day 0. Patients also receive cyclophosphamide IV over 3 hours, Mesna IV over 30 minutes on days 3 and 4, and tacrolimus IV continuously and then PO twice daily (BID), once tolerated, for up to 3 months. Patients may optionally receive eltrombopag PO QD or romiplostim injection weekly starting on day 5. Patients also undergo echocardiography (ECHO) and chest x-ray at baseline, abdominal ultrasound at baseline and follow up, and blood sample collection throughout the study. Patients may optionally undergo bone marrow aspiration as clinically indicated.
After completion of study treatment, patients are followed up in months 1, 2, 3, 6, and 12 and then annually in years 2 and 3.
Lead OrganizationM D Anderson Cancer Center
Principal InvestigatorUday R. Popat
