Ruxolitinib for Myelofibrosis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests whether adding ruxolitinib, a medication that may halt harmful immune responses and cancer growth, to standard treatments can aid older patients with myelofibrosis or similar conditions during and after a stem cell transplant. The goal is to determine if this combination can prevent graft-versus-host disease (GVHD), where transplanted cells attack the body's own cells, and improve overall recovery. Patients dealing with myelofibrosis or related conditions who are candidates for a stem cell transplant might be suitable participants. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.
Do I have to stop taking my current medications for the trial?
The trial information does not specify if you need to stop taking your current medications. However, you must be willing to take ruxolitinib for at least 8 weeks before the transplant and continue it for up to 12 months after, followed by a 6-month taper.
Is there any evidence suggesting that ruxolitinib is likely to be safe for humans?
Research has shown that ruxolitinib is generally well-tolerated by people with myelofibrosis (MF). Studies have identified common side effects, such as low blood cell counts, particularly anemia (low red blood cells) and thrombocytopenia (low platelets). These side effects can be managed and typically do not worsen significantly over time. For over ten years, ruxolitinib has been used to treat MF and has improved symptoms and increased survival rates. Although ruxolitinib affects blood cell counts, regular check-ups and adjustments can help manage these effects.12345
Why are researchers excited about this study treatment for myelofibrosis?
Ruxolitinib is unique because it targets the JAK1 and JAK2 pathways, which are often overactive in myelofibrosis, a rare bone marrow disorder. Unlike standard treatments that primarily manage symptoms, ruxolitinib directly tackles the underlying disease mechanism, potentially offering more comprehensive disease control. Researchers are excited about ruxolitinib because it can be used in combination with other therapies, like high-intensity or reduced-intensity conditioning regimens, which might enhance the overall treatment effectiveness and improve patient outcomes.
What evidence suggests that ruxolitinib might be an effective treatment for myelofibrosis?
Research has shown that ruxolitinib effectively treats myelofibrosis (MF). In one study, about 44% of patients with MF experienced either full or partial relief from symptoms, a reduction in spleen size, or improved blood counts. Another study found that ruxolitinib not only reduced spleen size and symptoms but also extended patients' lives. Additionally, finding the right dose of ruxolitinib has been linked to better results and manageable side effects for MF patients. In this trial, participants will receive ruxolitinib as part of their treatment regimen, which may help prevent graft versus host disease (GVHD) and improve outcomes for patients undergoing blood stem cell transplants.36789
Who Is on the Research Team?
Rachel Salit, MD
Principal Investigator
Fred Hutch/University of Washington Cancer Consortium
Are You a Good Fit for This Trial?
This trial is for older patients with myelofibrosis or MDS/MPN overlap syndromes. Participants must be eligible for a stem cell transplant from a donor and able to receive medications like ruxolitinib, tacrolimus, and methotrexate.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Pre-Conditioning Treatment
Patients receive ruxolitinib or an alternate JAK-inhibitor for at least 8 weeks prior to the start of HCT conditioning
Conditioning and Transplantation
Patients receive high or reduced intensity conditioning chemotherapy followed by stem cell infusion
Post-Transplant Treatment
Patients receive ruxolitinib and GVHD prophylaxis with tacrolimus and methotrexate
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Methotrexate
- Ruxolitinib
Trial Overview
The study tests adding ruxolitinib to standard GVHD prevention in older patients undergoing donor stem cell transplants. It aims to see if this approach is safe and effective in preventing GVHD compared to the usual treatment without ruxolitinib.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
PART 1: Patients receive ruxolitinib or alternate JAK-inhibitor for at least 8 weeks prior to the start of HCT conditioning. PART 2: Starting on day -4, patients receive ruxolitinib 5mg PO BID for 12 months then PO QD until 18 months. Patients receive high intensity conditioning with cyclophosphamide IV on days -7 and -6 and busulfan IV over 3 hours on days -5 to -2 or reduced intensity conditioning with fludarabine IV over 30 minutes on days -6 to -2 and melphalan IV over 15-30 minutes on days -3 and -2. Patients receive stem cells infusion on day 0. Starting on day -3, patients receive tacrolimus IV over 1-2 hours or PO every 12 hours for at least 100 days. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Additionally, patients undergo urine sample collection, echocardiography, and pulmonary function testing prior to conditioning and blood sample collection, bone marrow biopsy and aspiration, and spleen ultrasound or CT throughout the study.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Fred Hutchinson Cancer Center
Lead Sponsor
Incyte Corporation
Industry Sponsor
Steven Stein
Incyte Corporation
Chief Medical Officer since 2015
MD from University of Witwatersrand
Hervé Hoppenot
Incyte Corporation
Chief Executive Officer since 2014
MBA from ESSEC Business School
Citations
Long-term safety and effectiveness of ruxolitinib in patients ...
After 6 months of ruxolitinib treatment, 26.2% of patients had a spleen response and 52.0% of patients had less symptoms. We could not calculate ...
2.
ashpublications.org
ashpublications.org/bloodneoplasia/article/2/2/100082/535814/Outcomes-for-ruxolitinib-only-versus-combinationOutcomes for ruxolitinib only versus combination with ...
In this phase 2 trial, 44% of patients achieved either complete (CR) or partial responses (PR), including symptom, spleen size, hematologic, ...
Ten years of treatment with ruxolitinib for myelofibrosis
Ruxolitinib has been shown to not only improve splenomegaly and the burdensome symptoms associated with MF but also to improve overall survival (OS).
A Review of Real-World Experience With Ruxolitinib for ...
Data from the COMFORT trials indicated that ruxolitinib treatment was associated with significantly longer OS compared with placebo or best available treatment.
5.
curetoday.com
curetoday.com/view/optimal-jakafi-dosing-associated-with-improved-outcomes-in-myelofibrosisOptimal Jakafi Dosing Associated With Improved ...
Optimal dosing of Jakafi (ruxolitinib) was associated with improved efficacy and manageable safety in patients with myelofibrosis, according to study findings.
Safety Profile in Intermediate or High-Risk Myelofibrosis (MF)
Jakafi® (ruxolitinib) adverse reactions · The most frequently observed adverse reactions were thrombocytopenia and anemia · Perform a pretreatment CBC, and ...
Efficacy, safety, and survival with ruxolitinib in patients with ...
Ruxolitinib continued to be generally well tolerated; there was no pattern of worsening grade ≥3 anemia or thrombocytopenia with longer-term ruxolitinib ...
8.
ashpublications.org
ashpublications.org/blood/article/144/Supplement%201/1786/529894/Comparing-the-Efficacy-and-Safety-of-RuxolitinibComparing the Efficacy and Safety of Ruxolitinib in Patients ...
Ruxolitinib is an effective treatment for patients with lower risk MF in the classification of MIPSS-70, MIPSS+V2.0, DIPSS-Plus, DIPSS≤2 and MYSEC-PM.
Efficacy and safety of ruxolitinib in patients with lower risk ...
By week 48, 26 (65.0%) patients achieved a ≥ 50% decrease in palpable spleen length. 32 (80.0%) patients achieved a ≥ 50% reduction from ...
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