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Led by M.D. Anderson Cancer Center · Updated on 2026-05-20
38
Participants Needed
1
Research Sites
N/A
Total Duration
Researchers are studying the use of liposome-encapsulated daunorubicin-cytarabine CPX-351 to find the best dose and understand side effects in treating patients with intermediate-2 or high-risk myelodysplastic syndrome MDS or chronic myelomonocytic leukemia CMML that has returned or not responded to other treatments. This phase I trial focuses on safety, tolerability, maximum tolerated dose, and preliminary effectiveness of CPX-351, a chemotherapy drug that works by stopping cancer cells from growing or spreading. Patients receive CPX-351 intravenously over 90 minutes during an induction phase on days 1, 3, and 5. If the disease is stable and side effects are acceptable, patients may receive additional doses on days 1 and 3 after 2 to 5 weeks. Those who respond with at least hematological improvement enter a consolidation phase, receiving treatment every 28 days for up to 12 cycles, with possible extension for 12 more cycles if no significant disease progression or toxicity occurs. During the study, participants will be monitored for side effects, response to treatment, and disease progression. Follow-up visits occur 30 days after treatment and then every six months. Researchers will measure adverse events, response rates, survival outcomes, and relapse-free survival over a period of up to 1.5 years to evaluate the drugs safety and preliminary efficacy.
CONDITIONS
CPX-351 in Treating Patients With Relapsed or Refractory High Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia
G
Guillermo M Bravo
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M.D. Anderson Cancer Center
Lead Sponsor
National Cancer Institute (NCI)
Collaborating Sponsor
This table compares participation in this trial with other options available for this condition. The information is drawn from published research and the trial’s study details and is provided for educational purposes only.
| Factors | CPX-351 Study Regimen | Current-Care Salvage Therapy |
|---|---|---|
| What it is | A study regimen consisting of CPX-351, a fixed-combination liposome-encapsulated daunorubicin and cytarabine, administered intravenously to adults with relapsed or refractory high-risk MDS or CMML after prior hypomethylating agent therapy. | Alternative salvage therapy, typically a high-dose cytarabine-based regimen, is commonly used for adults with relapsed or refractory high-risk MDS or CMML after prior hypomethylating agent failure according to practice guidelines. |
| Role in Trial | CPX-351 is evaluated as the investigational therapy in a single-arm phase 1/2 trial for disease control and response. | High-dose cytarabine-based salvage therapy serves as the guideline-recommended current-care approach outside this study for similar patients. |
| Primary Use | Tested for potential disease control, response, and survival extension in relapsed or refractory high-risk MDS or CMML after failing prior hypomethylating agents. | Used to attempt disease control and achieve response in the same patient population when other therapies fail. |
| How it is given or performed | CPX-351 is administered intravenously, with dosing determined by the phase 1/2 trial protocol for safety and response. | High-dose cytarabine-based regimens are typically administered intravenously, with schedules defined by guideline-supported protocols. |
| Evidence Status | Early-phase evidence from a single-center phase 1/2 trial; data on efficacy and safety in this population is still maturing. | Supported by guideline consensus and numerous published studies as the recommended salvage option for disease control when HMA treatment fails. Outcomes remain suboptimal for this population. |
| Important Limitations | Single-arm early-phase design, no direct comparative efficacy or safety data; trial population is limited to post-hypomethylating agent failure and transplant-ineligible patients. | High-dose cytarabine-based regimens offer limited response rates and significant toxicity; overall outcomes are poor after HMA failure and many patients may not tolerate intensive therapy. |
Disclaimer: This comparison is created with the help of artificial intelligence(AI) and may contain errors or missing information. It is not medical advice and does not mean one option is better or safer than another. Clinical trials are still being studied, and joining a trial is your choice. Please consult your healthcare professional before making any decisions.
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Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 1.5 years
Participants receive liposome-encapsulated daunorubicin-cytarabine intravenously as induction therapy followed by consolidation therapy if appropriate.
Treatment cycles with IV infusions on specific days repeated every 28 days for up to 24 cycles; initial induction therapy includes infusions on days 1, 3, and 5; subsequent cycles have infusions on days 1 and 3
Duration - Up to 1.5 years after treatment completion
Participants are followed up after completion of study treatment to monitor safety and outcomes.
Visits at 30 days post-treatment and then every 6 months
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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Guillermo Montalban-Bravo, Elias Jabbour, Gautam Borthakur...
https://pubmed.ncbi.nlm.nih.gov/37946611/M.D. Anderson Cancer Center
Lead Sponsor
National Cancer Institute (NCI)
Collaborating Sponsor
