Clinical trial · Interventional
CPX-351 in Treating Patients With Relapsed or Refractory High Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia
Phase 1 Dose Escalation Study of CPX-351 for Patients With Int-2 or High Risk IPSS Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia After Failure to Hypomethylating Agents
NCT03896269CI-TRIAL-00112182recruitingPhase 1ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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
Conditions (7)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Blasts 10-19 Percent of Bone Marrow Nucleated Cells | — | UNRESOLVED | — |
| Blasts More Than 5 Percent of Bone Marrow Nucleated Cells | — | UNRESOLVED | — |
| High Risk Chronic Myelomonocytic Leukemia | — | UNRESOLVED | — |
| Recurrent Chronic Myelomonocytic Leukemia | Chronic Myelomonocytic Leukemia | CURATED_BROADER | 0.78 |
| Recurrent High Risk Myelodysplastic Syndrome | — | UNRESOLVED | — |
| Refractory Chronic Myelomonocytic Leukemia | Chronic Myelomonocytic Leukemia | CURATED_BROADER | 0.78 |
| Refractory High Risk Myelodysplastic Syndrome | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Liposome-encapsulated Daunorubicin-Cytarabine | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Treatment (liposome-encapsulated daunorubicin-cytarabine)
- description
- 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.
- interventionNames
- Drug: Liposome-encapsulated Daunorubicin-Cytarabine
Primary outcomes (4)
- measure
- Incidence of adverse events (dose-escalation stage)
- timeFrame
- Up to 30 days
- description
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
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
References
Publications (1)
- DERIVEDMontalban-Bravo G, Jabbour E, Borthakur G, Kadia T, Ravandi F, Chien K, Pemmaraju N, Hammond D, Dong XQ, Huang X, Schneider H, John R, Kanagal-Shamana R, Loghavi S, Kantarjian H, Garcia-Manero G. Phase 1/2 study of CPX-351 for patients with Int-2 or high risk International Prognostic Scoring System myelodysplastic syndromes and chronic myelomonocytic leukaemia after failure to hypomethylating agents. Br J Haematol. 2024 Mar;204(3):898-909. doi: 10.1111/bjh.19193. Epub 2023 Nov 10. PMID 37946611