18 and older, any sex, with Myelodysplastic Syndrome (MDS). Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Efficacy of CPX-351 as Measured by Overall Response Rate (ORR)Primary· day 28 +/- 7 days of induction
Efficacy of CPX as measured by ORR as defined by IWG 2006 criteria for MDS participants at end of induction. IWG 2006 responses that must be for at least 4 weeks include Complete Remission (CR), Partial Remission (PR), Marrow CR, Stable Disease (SD), Failure, Relapse after CR or PR (PD), or cytogenetic response. Hematologic Improvement (HI), which must be for at least 8 weeks, includes erythroid response (pretreatment, \< 11 g/dL), Platelet response (pretreatment, \< 100x109/L), Neutrophil response (pretreatment, \< 1 x109/L), or Progression or relapse after HI.
Group
Value
95% CI
Intravenous CPX-351 With Potential Maintenance Therapy
1
Intravenous CPX-351 With Potential Maintenance Therapy
0
Intravenous CPX-351 With Potential Maintenance Therapy
0
Intravenous CPX-351 With Potential Maintenance Therapy
0
Time to Response (TTR) Associated With CPX-351Secondary· day 28 +/- 7 days of induction
TTR associated with CPX-351 in participant with MDS at the end of induction. TTR defined by the time between starting the treatment and the time of achieving best response.
Group
Value
95% CI
Intravenous CPX-351 With Potential Maintenance Therapy
28
Duration of Response (DOR) in Participants Achieving a ResponseSecondary· up to 1 year after end of treatment
DOR in participants achieving a response defined by the time between first response (day C1 D28 +/-7 days from induction) and the day of loss of response
Group
Value
95% CI
Intravenous CPX-351 With Potential Maintenance Therapy
458
Event-free Survival (EFS)Secondary· up to 1 year after end of treatment
EFS probability of all participants enrolled in this trial from start of treatment and up to 1 year after the end of treatment.
Group
Value
95% CI
Intravenous CPX-351 With Potential Maintenance Therapy
1
Overall Survival (OS)Secondary· up to 1 year after end of treatment
OS probability of all participants enrolled in this trial from start of treatment and up to 1 year after the end of treatment.
Group
Value
95% CI
Intravenous CPX-351 With Potential Maintenance Therapy
1
Adverse events — posted to ClinicalTrials.gov
Time frame: 2.5 years or until death, whichever occurs first..
Reporting threshold: 2%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Intravenous CPX-351 With Potential Maintenance Therapy
The purpose of this study is to evaluate the efficacy of treatment with CPX-351 (an FDA approved drug for the treatment of AML) in individuals with MDS while using a new stratification tool to predict outcomes of participants following HMA failure. This approach is intended to gain a better understanding and insight into identifying new opportunities for drug approvals in this setting.
Publications & conference data
4 peer-reviewed publications reference this trial (live from Europe PMC):
NCT07008638 — Phase I/II Clinical Trial of Proteasome Inhibitor in Combination With CPX-351 for the Treatment of Newly-Diagnosed TP53-
· Phase 1, PHASE2
· recruiting
NCT05829434 — Magrolimab Plus Intensive Chemotherapy in Newly Diagnosed AML or HR-MDS
· Phase 2
· withdrawn
NCT04982354 — Induction Therapy for Patients With FLT3 Mutated Acute Myeloid Leukemia
· Phase 1, PHASE2
· withdrawn
NCT04992949 — Evaluation of CPX-351 Monotherapy in Acute Myeloid Leukemia Secondary to Myeloproliferative Neoplasm
· Phase 2
· completed
NCT05169307 — CPX-351 Real-World Effectiveness and Safety Study
· completed
Other recruiting trials for Myelodysplastic Syndrome (MDS)
Currently open trials in the same condition.
NCT07008820 — Patient Reported Outcomes and Patient Voice Among Patients Diagnosed With Low Risk Myelodysplastic Syndrome (LR-MDS) or
· recruiting
NCT03613532 — Venetoclax Added to Fludarabine + Busulfan Prior to Transplant and to Maintenance Therapy for AML, MDS, and MDS/MPN
· Phase 1
· active not recruiting
NCT03173937 — Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilic
· Phase 1, PHASE2
· recruiting
NCT02790515 — Provision of TCRγδ T Cells and Memory T Cells Plus Selected Use of Blinatumomab in Naïve T-cell Depleted Haploidentical
· Phase 2
· active not recruiting
Other Case Comprehensive Cancer Center trials
Trials by the same sponsor.
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· NA
· not yet recruiting
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· NA
· recruiting
NCT07084779 — Investigation of Impact of AI on Prostate Cancer Workflow
· NA
· recruiting
NCT06904482 — Co-Transplant of an Unmodified Haplo-Identical Graft With Cord Blood
· Phase 2
· recruiting
NCT07044362 — Histotripsy Plus Chemotherapy vs Chemotherapy Alone for Advanced Colorectal Liver Metastasis
· NA
· recruiting
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Case Comprehensive Cancer Center
Last refreshed: 15 August 2024
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03957876.