CPX-351 for Myelodysplastic Syndrome
Trial Summary
Do I need to stop my current medications for the CPX-351 trial?
The trial protocol does not specify if you need to stop your current medications. However, if you are on disease-modifying therapy for MDS or investigational agents, you cannot participate in the trial.
What data supports the effectiveness of the drug CPX-351 for treating myelodysplastic syndrome?
CPX-351, a drug used for certain types of acute myeloid leukemia (AML), has shown to improve survival and remission rates compared to traditional chemotherapy in older adults with high-risk AML. This suggests it may also be effective for related conditions like myelodysplastic syndrome, as it uses a similar combination of drugs in a special formulation that enhances their effectiveness.12345
Is CPX-351 generally safe for humans?
CPX-351, also known as Vyxeos, has been studied in patients with acute myeloid leukemia (AML) and has shown a safety profile comparable to traditional chemotherapy, with some common side effects including febrile neutropenia (fever with low white blood cell count), pneumonia, and sepsis. It has been approved by the FDA and EMA for certain types of AML, indicating it is considered safe for use in humans for these conditions.12346
How is the drug CPX-351 different from other treatments for myelodysplastic syndrome?
What is the purpose of this trial?
This is a pilot and feasibility study of transplant eligible, higher risk myelodysplastic syndrome (MDS) patients to determine the safety and tolerability of a lower -dose and higher-dose CPX-351 regimen, with secondary objectives including complete remission (CR) rates and proportion of patients proceeding to transplant.
Research Team
Meagan Jacoby, M.D., Ph.D.
Principal Investigator
Washington University School of Medicine
Eligibility Criteria
This trial is for adults aged 18-70 with higher risk Myelodysplastic Syndrome (MDS) who are eligible for a transplant. They must have good kidney, liver, and heart function, an ECOG performance status ≤2, and no history of Wilson's disease or certain viral infections. Women must not be pregnant and participants should agree to use contraception.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive CPX-351 via IV infusion on Days 1, 3, and 5 of the first induction. A second induction may be considered based on bone marrow biopsy results.
Consolidation
In the absence of disease progression or unacceptable toxicity, patients may continue to consolidation or proceed to alloHCT.
Follow-up
Participants are monitored for safety and effectiveness after treatment, including assessments for event-free survival, relapse-free survival, and overall survival.
Treatment Details
Interventions
- CPX-351
CPX-351 is already approved in United States, European Union for the following indications:
- Newly-diagnosed therapy-related acute myeloid leukemia (t-AML)
- AML with myelodysplasia-related changes (AML-MRC)
- Newly-diagnosed therapy-related acute myeloid leukemia (t-AML)
- AML with myelodysplasia-related changes (AML-MRC)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Washington University School of Medicine
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