NCT06345495

High Dose Ruxolitinib and Allogeneic Stem Cell Transplantation in Myelofibrosis Patients With Splenomegaly

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: M.D. Anderson Cancer Center · Academic / Other
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

To learn if giving ruxolitinib and busulfan before a stem cell transplant can help to reduce spleen size and help the transplant to succeed.

Detailed description

Primary Objective 1\) 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: 1. Overall survival 2. Progression-free survival 3. Graft vs host disease relapse free survival 4. Relapse rate 5. Non-relapse Mortality 6. Time to Neutrophil and platelet engraftment 7. Time to red cell transfusion independence 8. Graft failure 9. Acute and chronic GVHD 10. Grade 3 -5 Toxicity 11. Incidence of poor graft function5 12. Need for growth factors (myeloid or thrombopoietic) at 100 days 13. Spleen response around day -7, -1, 30, and 100 days 14. Need for transfusions at 100 days 15. Time to discontinuation of immunosuppressives Exploratory Objectives: 1. Immune reconstitution 2. Cytokine profile

Conditions

• Splenomegaly
• Myelofibrosis

Interventions

Type	Name	Description
DRUG	Ruxolitinib	Given by PO
PROCEDURE	Allogeneic Stem Cell Transplantation	Given by Transplant
DRUG	Levetiracetam	Given by PO
DRUG	Eltrombopag	Given by PO
DRUG	Busulfan	Given by IV
DRUG	Romiplostim	Given by IV
DRUG	Fludarabine phosphate	Given by IV
DRUG	Cyclophosphamide	Given by IV
DRUG	Mesna	Given by IV
DRUG	Tacrolimus	Given by IV or PO

Timeline

• Start date: 2024-10-01
• Primary completion: 2027-01-01
• Completion: 2029-01-01
• First posted: 2024-04-03
• Last updated: 2026-03-11

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study