Actively Recruiting
High Dose Ruxolitinib and Allogeneic Stem Cell Transplantation in Myelofibrosis Patients With Splenomegaly
Led by M.D. Anderson Cancer Center · Updated on 2026-03-11
30
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
Sponsors
M
M.D. Anderson Cancer Center
Lead Sponsor
I
Incyte Corporation
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating whether giving ruxolitinib and busulfan before a stem cell transplant can reduce spleen size and improve transplant success for patients with myelofibrosis and splenomegaly. This Phase 2 study aims to compare patient survival and disease-free rates at 100 days after transplant against historical data, while also tracking various clinical outcomes such as graft function, relapse, and toxicity. Participants will receive high-dose ruxolitinib and busulfan prior to undergoing an allogeneic stem cell transplant. Additional medications like levetiracetam, eltrombopag, romiplostim, fludarabine phosphate, cyclophosphamide, mesna, and tacrolimus may also be given as part of the treatment. The treatment phase lasts about 100 days, followed by a one-year follow-up period to monitor patient progress and long-term outcomes. Throughout the study, participants will attend visits for tests and procedures to assess spleen size, blood counts, immune function, and adverse events. Researchers will measure safety and side effects over approximately one year. They will also observe engraftment success, graft-versus-host disease, transfusion needs, and survival rates to understand the treatment impact and transplant outcomes.
CONDITIONS
Brief Title
High Dose Ruxolitinib and Allogeneic Stem Cell Transplantation in Myelofibrosis Patients With Splenomegaly
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants aged 18 to 75 years
- Able to provide written consent
- Diagnosed with primary or secondary myelofibrosis (may have received Jak inhibitors including ruxolitinib)
- Enlarged spleen by palpation or imaging (spleen larger than 12 cm)
- Has a fully matched (8/8 HLA) related or matched unrelated donor
- Adequate kidney function (serum creatinine 3 1.5 mg/dL or eGFR 40 ml/min/1.73 m2)
- Adequate liver function (ALT/AST 3 3 x ULN, direct bilirubin 3 1 mg/dL, no liver cirrhosis or ascites)
- Female participants of childbearing potential must have a negative pregnancy test and agree not to breastfeed during and 3 months after treatment
- Participants of childbearing potential must agree to use highly effective contraception during treatment and 3 months after completion
You will not qualify if you...
- Positive pregnancy test in females of childbearing potential or lactating females
- Ejection fraction less than 40%
- Corrected DLCO less than 50%
- Uncontrolled or active systemic infection (viral, bacterial, or fungal)
- Active hepatitis B, hepatitis C, HIV, or tuberculosis infection requiring treatment
- History of thrombosis such as myocardial infarction, stroke, pulmonary embolism, or deep vein thrombosis in the past 6 months
- History of liver cirrhosis or ascites (per inclusion criteria)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 7 days before transplant
Participants receive high dose Ruxolitinib before transplantation as part of preparation.
Several visits for dosing and monitoring
Duration - About 100 days
Participants undergo allogeneic stem cell transplantation and receive multiple study drugs including immunosuppressive and supportive therapies.
Regular visits including transplant day and follow-up assessments
Duration - Up to 1 year after treatment
Participants are monitored for safety, graft function, and survival outcomes for up to one year after treatment.
Periodic visits for safety and efficacy assessments
Trial Site Locations
Total: 1 location
1
MD Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
U
Uday Popat, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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