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

ConditionsBlasts 10-19 Percent of Bone Marrow Nucleated CellsBlasts More Than 5 Percent of Bone Marrow Nucleated CellsHigh Risk Chronic Myelomonocytic LeukemiaRecurrent Chronic Myelomonocytic LeukemiaRecurrent High Risk Myelodysplastic SyndromeRefractory Chronic Myelomonocytic LeukemiaRefractory High Risk Myelodysplastic Syndrome
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorM.D. Anderson Cancer Center

About this trial

This phase I trial studies best dose and side effects of liposome-encapsulated daunorubicin-cytarabine (CPX-351) and how well it works in treating patients with high risk myelodysplastic syndrome or chronic myelomonocytic leukemia that has come back or has not responded to treatment. Drugs used in chemotherapy, such as liposome-encapsulated daunorubicin-cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Single group

Treatments tested in this trial

  • Liposome-encapsulated Daunorubicin-Cytarabine

    Drug

    Given IV

Treatment groups

38 Participants
are divided into 1 treatment group
Group A: Treatment (liposome-encapsulated daunorubicin-cytarabine)Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence of adverse events (dose-escalation stage)

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.

Time frame
Up to 30 days
2

Maximum-tolerated dose (MTD) of liposome-encapsulated daunorubicin-cytarabine (dose-escalation stage)

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.

Time frame
Up to 28 days
3

Incidence of adverse events (dose-expansion stage)

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.

Time frame
Up to 30 days
4

Objective response rate (ORR) (dose-expansion stage)

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.

Time frame
Up to 1.5 years

Secondary outcomes

1

Overall response rate

Time frame
Up to 1.5 years
2

Overall survival

The distribution of time-to-event endpoints will be estimated using the method of Kaplan and Meier. Comparisons of time-to-event endpoints by important subgroups will be made using the log-rank tests.

Time frame
Up to 1.5 years
3

Duration of response

Will be summarized using descriptive statistics such as mean, standard deviation, median and range.

Time frame
Up to 1.5 years
4

Relapse-free survival

The distribution of time-to-event endpoints will be estimated using the method of Kaplan and Meier. Comparisons of time-to-event endpoints by important subgroups will be made using the log-rank tests.

Time frame
Up to 1.5 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

M.D. Anderson Cancer Center

Lead sponsor

National Cancer Institute (NCI)

Collaborator

Source ClinicalTrials.gov
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