A Phase 1 interventional study of CPX-351 and Research skin biopsy in Myelodysplastic Syndromes, sponsored by Washington University School of Medicine. Active, not recruiting at 3 sites in United States. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2026-04-14.
Sponsored by Washington University School of Medicine · Phase 1, Interventional, and Treatment
This is a pilot and feasibility study of transplant eligible, higher risk myelodysplastic syndrome (MDS) patients to determine the safety and tolerability of a lower -dose and higher-dose CPX-351 regimen, with secondary objectives including complete remission (CR) rates and proportion of patients proceeding to transplant.
2,124 studies on the registry are indexed under Myelodysplastic Syndromes; 320 are open to participants now.
This study's enrollment of 20 is below the median of 39 across 1,740 interventional studies indexed under Myelodysplastic Syndromes.
Browse Myelodysplastic Syndromes studies →Washington University School of Medicine is the lead sponsor of 1,764 studies on the registry; 270 are open to participants now.
Of its 325 completed or terminated interventional studies of FDA-regulated products, 212 (65%) have results posted.
Counted across the registry records on this site, refreshed daily.
Adequate renal and hepatic function as defined below:
*Total bilirubin ≤ 2.0 x IULN*
Note: If, in the opinition of the treatment physician, the bilirubin is elevated secondary to hemolysis or Gilbert's disease, the patient may be eligible after discussion with the Washington University PI.
Exclusion Criteria:
* CPX-351 will be given according to the assigned dose level over a minimum of a 90-minutes via IV infusion on Days 1, 3, and 5 of the first induction * If the treating physician elects to perform a day 14 bone marrow biopsy then, a second induction may be considered for patients in the absence of a chemoablated, hypocellular marrow on the Day 14 bone marrow assessment, if the patient has failed to achieve a marrow CR, and it is deemed safe to administer by the treating physician. The second induction uses a modified schedule in which CPX-351 will be given according to the assigned dose level on Days 1 and 3 * In the absence of disease progression or unacceptable toxicity, the patient may continue to consolidation at the discretion of the treating physician or the patient may proceed to alloHCT after induction at the discretion of the treating physician
Drug: CPX-351 · Procedure: Research skin biopsy · Procedure: Research blood draw · Procedure: Research bone marrow aspirate
-CPX-351 will be provided by Jazz Pharmaceuticals
Also known as: Vyxeos™, Daunorubicin and cytarabine
-And/or buccal swab * Pre-treatment * Post-induction (no earlier than Day 28 and no later than Day 56 from last induction)
* Pre-treatment * Post-induction (no earlier than Day 28 and no later than Day 56 from last induction) * Post-consolidation 1 (if applicable) * Post-consolidation 2 (if applicable) * Post-transplant Day 30 (if applicable) * Post-transplant Day 100 (if applicable)
* Pre-treatment * Post-induction (no earlier than Day 28 and no later than Day 56 from last induction) * Post-consolidation 1 (if applicable) * Post-consolidation 2 (if applicable) * Post-transplant Day 30 (if applicable) * Post-transplant Day 100 (if applicable)
Safety and tolerability of a CPX-351 regimen in a transplant eligible, higher risk MDS population as measured by the proportion of participants who experience an adverse event by patient, type of event, and grade of event
Time frame: Through 56 days after the last dose
Overall response rate in MDS patients treated with CPX-351
* Overall response rate = complete remission + marrow complete remission + partial response + hematologic improvement * Patients will be assessed for response according to modified International Working Group (IWG) criteria for MDS
Time frame: 56 days after the last dose
Best overall response in MDS patients treated with CPX-351
-Patients will be assessed for response according to modified International Working Group (IWG) criteria for MDS
Time frame: 56 days after the last dose
Remission duration in MDS patients treated with CPX-351
* Defined as the interval from the date complete remission is documented to the date of recurrence. This is determined only for patients achieving a complete remission. * Patients will be assessed for response according to modified International Working Group (IWG) criteria for MDS
Time frame: Through 5 years
Relapse-free survival in MDS patients treated with CPX-351
-Patients will be assessed for response according to modified International Working Group (IWG) criteria for MDS
Time frame: Through 5 years
Progression-free survival in MDS patients treated with CPX-351
* Defined as the interval from the date of first dose of study drug to disease progression or death from MDS. * Patients will be assessed for response according to modified International Working Group (IWG) criteria for MDS
Time frame: Through 5 years
Overall survival in MDS patients treated with CPX-351
-Defined as the date of first dose of study drug to the date of death from any cause.
Time frame: Through 5 years
Complete remission + marrow complete remission rates in patients treated with CPX-351
-Patients will be assessed for response according to modified International Working Group (IWG) criteria for MDS
Time frame: 56 days after the last dose
Post-induction mortality in MDS patients treated with CPX-351
-Rate of death
Time frame: Day 30
Post-induction mortality in MDS patients treated with CPX-351
-Rate of death
Time frame: Day 60
Safety and feasibility of CPX-351 consolidation therapy in MDS patients as measured by the proportion of patients who experience an adverse event by patient, type of event, and grade of event
Time frame: Through 56 days after the last dose
Proportion of MDS patients treated with CPX-351 proceeding to allogeneic hematopoietic cell transplant
Time frame: Through 56 days after the last dose
Overall survival in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
-Defined as the date of first dose of study drug to the date of death from any cause.
Time frame: Day 100
Overall survival in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
-Defined as the date of first dose of study drug to the date of death from any cause.
Time frame: 1 year
Non-relapse mortality in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
Time frame: Day 100
Non-relapse mortality in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
Time frame: 1 year
Event-free survival in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
* Defined as the interval from the date of first dose of study drug to date of treatment failure, recurrence, or death due to any cause. * Patients will be assessed for response according to modified International Working Group (IWG) criteria for MDS
Time frame: Day 100
Event-free survival in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
* Defined as the interval from the date of first dose of study drug to date of treatment failure, recurrence, or death due to any cause. * Patients will be assessed for response according to modified International Working Group (IWG) criteria for MDS
Time frame: 1 year
Plan to share: No
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Washington University School of Medicine