Using CPX-351 to treat patients with high-risk blood cancers
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
This study is testing a new treatment called CPX-351 to see if it can safely help people with high-risk blood cancers like myelodysplastic syndrome or chronic myelomonocytic leukemia.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 38 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | M.D. Anderson Cancer Center Academic / other |
| Drugs / interventions | chemotherapy, immunotherapy, radiation |
| Locations | 1 site (Houston, Texas) |
| Trial ID | NCT03896269 on ClinicalTrials.gov |
What this trial studies
This phase I trial investigates the safety, tolerability, and efficacy of liposome-encapsulated daunorubicin-cytarabine (CPX-351) in patients with relapsed or refractory high-risk myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML). The study involves a dose-escalation approach to determine the maximum tolerated dose and assess the overall response rate, survival, and safety profile of the treatment. Patients will receive the drug intravenously over specified days, with follow-up assessments to evaluate treatment outcomes.
Who should consider this trial
Good fit: Ideal candidates include patients diagnosed with intermediate-2 or high-risk MDS or CMML who have not responded to previous therapies and are not eligible for stem cell transplant.
Not a fit: Patients with low-risk MDS or CMML or those who have not exhausted other treatment options may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with high-risk MDS or CMML who have limited treatment alternatives.
How similar studies have performed: Other studies have shown promise with similar chemotherapy approaches, but this specific combination is being evaluated for the first time in this patient population.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Houston, Texas
- M D Anderson Cancer Center — Houston, Texas, United States (Recruiting)
Study contacts
- Principal investigator: Guillermo M Bravo — M.D. Anderson Cancer Center
- Study coordinator: Guillermo M Bravo
- Email: gmontalban1@mdanderson.org
- Phone: 713-794-3604
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.