Recruiting
Phase 1

CPX-351

Sponsor:

M.D. Anderson Cancer Center

Code:

NCT03896269

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

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Liposome-encapsulated Daunorubicin-Cytarabine

Study Details

Brief summary:

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.

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

Study ID

NCT03896269

Start date

May 14, 2019

Status verified date

May, 2026

Completion date

Dec 31, 2027

Anticipated

Primary completion date

Dec 31, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • 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 Criteria:

  • 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

Study Design

Enrollment

38 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Treatment (liposome-encapsulated daunorubicin-cytarabine)

INDUCTION THERAPY: Patients receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5 in the absence of disease progression or unacceptable toxicity. After 2-5 weeks, patients who do not achieve a CR/CRi/CRp, have acceptable or no toxicity, and have stable disease and no disease progression may receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3 in the absence of disease progression or unacceptable toxicity.

CONSOLIDATION THERAPY: Patients who achieve at least a HI response, receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. After 5-8 weeks, patients who do not show clinically significant disease progression or unacceptable toxicity may receive liposome-encapsulated daunorubicin-cytarabine for up to 12 additional cycles.

Interventions

Liposome-encapsulated Daunorubicin-Cytarabine

Given IV

Primary outcome measure

  • Incidence of adverse events (dose-escalation stage) [ Time Frame: Up to 30 days ]
  • Maximum-tolerated dose (MTD) of liposome-encapsulated daunorubicin-cytarabine (dose-escalation stage) [ Time Frame: Up to 28 days ]
  • Incidence of adverse events (dose-expansion stage) [ Time Frame: Up to 30 days ]
  • Objective response rate (ORR) (dose-expansion stage) [ Time Frame: Up to 1.5 years ]

Central Contacts and Locations

Central contacts

Guillermo M Bravo

713-794-3604gmontalban1@mdanderson.org

Locations

M D Anderson Cancer Center

Recruiting

Houston, Texas, United States, 77030

Contacts

Guillermo M. Bravo

713-794-3604

Principal Investigator:

Guillermo M. Bravo

More Information

Sponsor

M.D. Anderson Cancer Center

Last update posted

May 20, 2026

Last verified

May, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by M.D. Anderson Cancer Center on 2026-05-20.