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CPX-351 for High-Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia

This study is testing a chemotherapy drug called liposome-encapsulated daunorubicin-cytarabine (CPX-351) for patients with high-risk myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) that has returned or hasn't responded to previous treatments. The main goals are to find the safest dose of CPX-351 and to see how well it works. You might be able to join if you have been diagnosed with high-risk MDS or CMML, are 18 or older, and are not eligible for or choose not to have a stem cell transplant. The study is currently unclear about its recruitment status and plans to enroll 38 patients.

Study design
This is a dose-escalation study, meaning it starts with a lower dose and gradually increases it to find the safest and most effective amount. It is an interventional study, but the phase is not specified.
What's involved
Participants will receive liposome-encapsulated daunorubicin-cytarabine intravenously (IV) on days 1, 3, and 5 of each treatment cycle. This will continue as long as the treatment is working and side effects are manageable.
Compensation
Not stated in the trial record.
Follow-up
Adverse events will be measured for up to 30 days after treatment, and the maximum-tolerated dose will be determined within 28 days.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT03896269

CPX-351 in Treating Patients With Relapsed or Refractory High Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia

Recruiting
PHASE1Ages 18+InterventionalTreatment
M.D. Anderson Cancer Center
~38 participants
Updated 2026-05-20 on ClinicalTrials.gov
What's tested:Liposome-encapsulated Daunorubicin-Cytarabine

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of adverse events (dose-escalation stage)
Measured over Up to 30 days
+3 more outcomes measured

Conditions

Blasts 10-19 Percent of Bone Marrow Nucleated Cells
Blasts More Than 5 Percent of Bone Marrow Nucleated Cells
High Risk Chronic Myelomonocytic Leukemia
Recurrent Chronic Myelomonocytic Leukemia
Recurrent High Risk Myelodysplastic Syndrome
Refractory Chronic Myelomonocytic Leukemia
Refractory High Risk Myelodysplastic Syndrome

Explore related trials

Chronic Myelomonocytic LeukemiaMyelodysplastic Syndrome

NCT03896269

Where you'd take part

This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • M D Anderson Cancer Center

    Houston, Texasstudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Guillermo M Bravo · PRINCIPAL_INVESTIGATOR · M.D. Anderson Cancer Center

Who to contact

Guillermo M Bravo
[email protected]
713-794-3604
Email the study team

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Eligibility criteria

Inclusion

Diagnosis of MDS or chronic myelomonocytic leukemia (CMML) according to World Health Organization (WHO)
Patients are either not eligible for or choose not to proceed with a stem cell transplant at the time of enrollment
MDS and CMML classified by International Prognostic Scoring System (IPSS) as intermediate-2/high risk with excess blasts \> 5%, or with 10-19% bone marrow blasts
No response following at least 4 cycles of therapy or relapse after initial CR, partial response (PR), or HI or progression after any number of cycles of either azacitidine, decitabine, guadecitabine or ASTX727 (oral decitabine) as single agents or in combination with other investigational agents
Patient (or patient's legally authorized representative) must have signed an informed consent document indicating that the patient understands the purpose of and procedures required for the study and is willing to participate in the study
Total bilirubin \< 3 mg/dL (will allow less than 5 x upper limit of normal \[ULN\] if Gilbert's at investigator's discretion)
Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) =\< 3 x ULN
Serum creatinine clearance \> 30 mL/min and no end/stage renal disease
Hydroxyurea for control of leukocytosis or use of hematopoietic growth factors (eg, granulocyte-colony stimulating factor \[G-CSF\], granulocyte-macrophage colony-stimulating factor \[GM-CSF\], procrit, aranesp, thrombopoietins) is allowed at any time prior to or during study if considered to be in the best interest of the patient

Exclusion

New York Heart Association (NYHA) class III or IV congestive heart failure or left ventricular ejection fraction (LVEF) \< 50 by echocardiogram or multigated acquisition (MUGA) scan
History of myocardial infarction within the last 6 months or unstable/uncontrolled angina pectoris or history of severe and/or uncontrolled ventricular arrhythmias
Uncontrolled infection not adequately responding to appropriate antibiotics
Female patients who are pregnant or lactating
Patients with reproductive potential who are unwilling to following contraception requirements (including condom use for males with sexual partners, and for females: prescription oral contraceptives \[birth control pills\], contraceptive injections, intrauterine devices \[IUD\], double-barrier method \[spermicidal jelly or foam with condoms or diaphragm\], contraceptive patch, or surgical sterilization) throughout the study
Female patients with reproductive potential who have a positive urine or blood beta-human chorionic gonadotropin (beta HCG) pregnancy test at screening
Patients receiving any other concurrent investigational agent or chemotherapy, radiotherapy, or immunotherapy (within 14 days of initiating study treatment)
Prior cumulative anthracycline exposure of \> 550 mg/m\^2 daunorubicin or equivalent, or \> 400 mg/m\^2 in patients who received radiation therapy to the mediastinum

What this trial measures

  • Incidence of adverse events (dose-escalation stage)Up to 30 days

    Safety data of the patients will be summarized using descriptive statistics such as mean, standard deviation, median, and range. Toxicity type, severity, and attribution will be summarized for each patient using frequency tables.

  • Maximum-tolerated dose (MTD) of liposome-encapsulated daunorubicin-cytarabine (dose-escalation stage)Up to 28 days

    Defined as the highest dose level in which a dose-limiting toxicity (DLT) occurs within at most 1 out of 6 patients treated. DLTs are defined as any related non-hematological Common Terminology Criteria for Adverse Events grade \>= 3 adverse events that prevent further administration of the agent at that same dose level.

  • Incidence of adverse events (dose-expansion stage)Up to 30 days

    Safety data of the patients will be summarized using descriptive statistics such as mean, standard deviation, median, and range. Toxicity type, severity, and attribution will be summarized for each patient using frequency tables.

  • Objective response rate (ORR) (dose-expansion stage)Up to 1.5 years

    Defined as complete response (CR), partial response, CR with incomplete bone marrow recovery, marrow CR or hematological improvement. Will be estimated along with 95% confidence intervals treated at the MTD (i.e., including those treated at the MTD during dose escalation and dose expansion phases). The association between ORR and patient's clinical characteristics will be examined by Wilcoxon's rank sum test or Fisher's exact test, as appropriate.