Ruxolitinib + CPX-351 for Acute Myeloid Leukemia
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores a new treatment combination for individuals with secondary acute myeloid leukemia, a type of blood cancer. Researchers aim to determine the optimal dose of a drug called ruxolitinib when combined with CPX-351, a mix of two chemotherapy drugs designed to target cancer cells more effectively. They hope this combination will outperform CPX-351 alone. Suitable candidates have been diagnosed with certain blood disorders that have progressed, such as myelofibrosis or essential thrombocythemia, and have not responded to previous treatments. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new combination therapy.
Will I have to stop taking my current medications?
Yes, you will need to stop taking any prior MPN-associated therapy, such as hydroxyurea or JAK2 inhibitors, before starting the trial. However, no specific time without these medications (washout period) is required. Additionally, you must discontinue anti-platelet agents or anticoagulants before starting the study treatment.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that CPX-351, a combination of two chemotherapy drugs in tiny fat-based particles, has a safety profile similar to standard chemotherapy. It effectively targets cancer cells. Previous studies found CPX-351 to be well-tolerated, with safety comparable to traditional treatments for acute myeloid leukemia.
Ruxolitinib, another part of the treatment, is already used for other conditions, providing some assurance about its safety in humans. Although tested in combination with CPX-351, its use in other conditions suggests it is generally well-tolerated.
In this trial, researchers are examining the safety of using ruxolitinib with CPX-351. As the trial is in its early stages, it is still determining the best dose and any side effects. However, based on existing knowledge from other uses and studies, both drugs have shown a promising level of safety for treating leukemia.12345Why are researchers excited about this trial's treatments?
Unlike the standard treatments for acute myeloid leukemia, which typically involve chemotherapy drugs like daunorubicin and cytarabine, the combination of Ruxolitinib and CPX-351 introduces a unique approach. Ruxolitinib works differently by targeting specific proteins involved in cancer cell growth, offering a new mechanism of action. Additionally, CPX-351 is a liposome-encapsulated formulation of daunorubicin and cytarabine, which allows for more precise delivery of these drugs to cancer cells, potentially enhancing effectiveness and reducing side effects. Researchers are excited because this combination could improve outcomes for patients who do not respond well to traditional therapies.
What evidence suggests that this trial's treatments could be effective for acute myeloid leukemia?
Research shows that ruxolitinib can fight leukemia independently. In some studies, combining it with other treatments led to better results. CPX-351 significantly improved survival in patients with acute myeloid leukemia compared to standard treatments. It is FDA-approved and demonstrated a major survival benefit, especially for secondary AML, a type of blood cancer that develops from another blood disorder. This trial studies the combination of ruxolitinib and CPX-351, as together they might be more effective against cancer cells. Both treatments have promising evidence for their effectiveness.678910
Who Is on the Research Team?
Uma Borate, MD
Principal Investigator
The Ohio State Comprehensive Cancer Center
Are You a Good Fit for This Trial?
This trial is for patients with advanced myeloproliferative neoplasms, including those previously diagnosed with conditions like polycythemia vera or essential thrombocythemia. Participants must be able to perform daily activities (ECOG status 0-2), have a heart function within normal limits, and agree to use contraception. Exclusions include ongoing participation in another clinical trial, certain leukemia types, uncontrolled infections or illnesses, known drug hypersensitivity, and recent surgery.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Induction
Patients receive CPX-351 IV on days 1, 3, and 5 and ruxolitinib PO BID on days 6-28 of cycle 1
Re-Induction
Patients with significant residual disease may receive additional CPX-351 and ruxolitinib
Consolidation
Patients with ≤ 5% blasts in bone marrow receive CPX-351 and ruxolitinib for up to 2 cycles
Maintenance Therapy
Patients receive ruxolitinib for up to 8 cycles if they have not undergone SCT
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Liposome-encapsulated Daunorubicin-Cytarabine
- Ruxolitinib
Liposome-encapsulated Daunorubicin-Cytarabine is already approved in United States for the following indications:
Find a Clinic Near You
Who Is Running the Clinical Trial?
Uma Borate
Lead Sponsor
Ohio State University Comprehensive Cancer Center
Lead Sponsor
Jazz Pharmaceuticals
Industry Sponsor
Bruce C. Cozadd
Jazz Pharmaceuticals
Chief Executive Officer since 2009
BA in Economics from Yale University, MBA from Stanford University
Dr. Austin
Jazz Pharmaceuticals
Chief Medical Officer since 2023
MD from the Royal College of Surgeons in Ireland
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