RightFit
RecruitingPhase 1

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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests the best dose, side effects, and how well CPX-351 works. CPX-351 combines two chemotherapy drugs. It is for people with high-risk myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML), blood and bone marrow diseases that came back or did not respond to treatment.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Have MDS or CMML confirmed using World Health Organization rules.
  • Have disease rated intermediate-2 or high risk, with more than 5% blasts (young blood cells), or have 10–19% blasts in the bone marrow.
  • Cannot have a stem cell transplant or choose not to have one when joining.
  • Have had azacitidine, decitabine, guadecitabine, or ASTX727, alone or with other study drugs. The disease must not have responded after at least 4 treatment cycles, returned after improvement, or worsened after any number of cycles.
  • You or your legal representative must sign a consent form showing that you understand the study and agree to join.
  • Have bilirubin below 3 mg/dL and liver enzymes AST or ALT no more than 3 times the upper normal limit. A different bilirubin limit may be allowed for Gilbert's syndrome, a condition that raises bilirubin.
  • Have kidney clearance above 30 mL/min, a measure of how well kidneys filter blood, and no end-stage kidney disease.

Who can't join

  • Have severe heart failure or a heart pumping strength below 50%.
  • Had a heart attack within 6 months, or have unstable or uncontrolled chest pain or a history of severe or uncontrolled abnormal rhythms in the heart's lower chambers.
  • Have an uncontrolled infection that is not responding well enough to the right antibiotics.
  • Are pregnant, breastfeeding, or have a positive pregnancy test at screening.
  • Could have children but are unwilling to follow the study's birth control rules throughout the study.
  • Are receiving another study drug, chemotherapy, radiation, or immune-based treatment at the same time or within 14 days before starting study treatment.
  • Have received more than the allowed lifetime dose of anthracyclines, a type of chemotherapy: over 550 mg/m² of daunorubicin or an equal dose of a similar drug, or over 400 mg/m² if you had radiation to the middle of the chest.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility 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

Study sites (1)

TX