Refractory Acute Myeloid LeukemiaPhase 2NCT04668885
CPX-351 as a Novel Approach for the Treatment of Older Patients With AML and MDS
A Phase 2 study testing CPX-351 in people with Refractory Acute Myeloid Leukemia and Relapsed Acute Myelomonocytic Leukemia, run by Case Comprehensive Cancer Center at 1 site in United States.
Trial summary
The purpose of this study is to evaluate how effective lower doses of CPX-351 are in older participants with relapsed/refractory acute myeloid leukemia (AML) who are not eligible to receive intensive chemotherapy and in participants with myelodysplastic syndromes (MDS) after Hypomethylating Agents (HMA) failure. Read more about this study
- Who is it for?
- Ages 60 years and older
- Conditions the study is for
- Refractory Acute Myeloid Leukemia, Relapsed Acute Myelomonocytic Leukemia, Myelodysplastic Syndromes
- What is being tested?
- CPX-351
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteUnited States
- Study dates
- 14 Jan 2021to 19 Jan 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 60 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Primary refractory or relapsed AML (defined by 2016 World Health Organization [WHO] criteria) patients who are not suitable for or not willing to receive intensive chemotherapy as evaluated by the treating physician. Primary refractory disease is defined as:
- Failure to achieve a CR, CRi, or mLFS (defined as <5% Bone Marrow (BM) blasts) after receiving 1 or 2 cycles of remission induction chemotherapy.
- Failure to achieve a CR, CRi, or MLFS (defined as <5% BM blasts) after receiving 4 cycles of non-intensive chemotherapy or whose disease progressed at any time point during the treatment.
- Participants with MDS (according to 2016 WHO criteria) who did not respond to treatment with azacitidine, decitabine, or combination of HMA with another drug or lost their response to initial therapy with HMA.
- Eastern Cooperative Oncology Group (ECOG) performance status <=2
- Adequate hepatic (serum total bilirubin < 1.5 x ULN, Serum glutamic pyruvic transaminase (SGPT) and/or serum glutamate oxaloacetate transaminase (SGOT) <2.5 x ULN) and renal function (creatinine < 1.5mg/dL).
- Participants must be willing and able to review, understand, and provide written consent before starting therapy.
Who cannot join
- Active signs or symptoms of central nervous system (CNS) involvement by malignancy (lumbar puncture [LP] not required).
- Prior 7+3 remission induction chemotherapy for MDS or AML
- More than 2 lines of prior non-intensive therapy.
- New York Heart Association (NYHA) class III or IV heart disease, active ischemia or any other uncontrolled cardiac condition such as angina pectoris, clinically significant cardiac arrhythmia on rhythm control strategy or on pacemaker, uncontrolled hypertension (blood pressure > 160 systolic and > 110 diastolic not responsive to antihypertensive medication)
- Acute myocardial infarction in the previous 12 weeks (from the start of treatment).
- Any serious medical condition, laboratory abnormality, or psychiatric illness that, in the view of the treating physician, would place the participant at an unacceptable risk if he or she were to participate in the study or would prevent that person from giving informed consent.
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- Any active malignancy (unrelated, non-hematological malignancy) diagnosed within the past 6 months of starting the study drug (other than curatively treated carcinoma-in-situ of the cervix or non-melanoma skin cancer).
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to CPX-351.
- Subjects with uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, uncontrolled cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- Known history of HIV or active hepatitis B or C.
- No major surgery within 2 weeks prior to study enrollment.
- Pregnant or breast feeding
- Male and female participants who are fertile who do not agree to use an effective barrier methods of birth control (i.e. abstinence) to avoid pregnancy while receiving study treatment and for 30 days after last dose of study treatment. Non-childbearing is defined as > 1 year postmenopausal or surgically sterilized.
- Acute promyelocytic leukemia (APL)
Trial locations and contacts
1 site in 1 country.
United States1 site
Cleveland Clinic, Case Comprehensive Cancer Center
Cleveland, Ohio, 44106-5065
About this study
Official title: Lower Doses of CPX-351 as a Novel Approach for the Treatment of Older Patients With Relapsed or Refractory Acute Myeloid Leukemia and Myelodysplastic Syndromes
The purpose of this study is to evaluate how effective lower doses of CPX-351 are in older participants with relapsed/refractory acute myeloid leukemia (AML) who are not eligible to receive intensive chemotherapy and in participants with myelodysplastic syndromes (MDS) after Hypomethylating Agents (HMA) failure.
Read the detailed description
Currently, elderly patients with AML and high risk MDS, who are ineligible to receive induction chemotherapy and fail HMA +/- combination, have very poor outcomes and there is no FDA-approved therapy outside of some targeted therapies which can only be applied to a small patient population. CPX-351 is an investigational (experimental) drug that works by combining two anti-cancer drugs cytarabine and daunorubicin. CPX-351 is experimental because it is only FDA approved for the treatment of adults with two types of AML: newly diagnosed therapy-related AML or AML with myelodysplasia-related changes.
This is an open label clinical trial of lower doses of CPX-351 in relapsed/primary refractory older AML and MDS patients ineligible to receive intensive chemotherapy. The first arm is for particpants with primary refractory/relapsed AML and the second arm is for higher risk MDS participants after HMA failure.
- Conditions
- Refractory Acute Myeloid Leukemia, Relapsed Acute Myelomonocytic Leukemia, Myelodysplastic Syndromes
- Being tested
- CPX-351
- Medical subject headings
- Leukemia, Myeloid, Acute, Leukemia, Myelomonocytic, Acute, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CPX-351Drug
Induction phase: CPX-351 15 mg/m^2 on days 1 and 3 of each 28-day cycle for up to a total of 6 cycles in the absence of unacceptable toxicity
Maintenance phase: CPX-351 7.5 mg/m^2 on days 1 and 3 for two cycles alternating with 15 mg/m^2 for one cycle. Participants may receive up to 12 cycles of maintenance phase in the absence of unacceptable toxicity.
| Study group | Role | What they receive |
|---|---|---|
| Primary refractory/relapsed AML | Experimental | Lower dose CPX-351 in participants with primary refractory/relapsed AML. Participants will receive an induction and maintenance phase of CPX-351 Receives: Drug: CPX-351 |
| MDS after HMA failure | Experimental | Lower dose CPX-351 in participants with MDS after HMA failure. Participants will receive an induction and maintenance phase of CPX-351 Receives: Drug: CPX-351 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate (ORR) per 2003 International Working Group (IWG) criteria
ORR = complete response (CR) + CR with incomplete blood count recovery (CRi) + partial remission (PR) as defined by 2003 IWG criteria for AML patients, and ORR = CR + PR + hematologic improvement (HI) and as defined per 2006 IWG criteria for MDS patients
Measured: At 6 months
Overall response rate (ORR) per 2003 IWG criteria
ORR = complete response (CR) + CR with incomplete blood count recovery (CRi) + partial remission (PR) as defined by 2003 IWG criteria for AML patients, and ORR = CR + PR + hematologic improvement (HI) and as defined per 2006 IWG criteria for MDS patients
Measured: At 1 year
Overall response rate (ORR) per 2003 IWG criteria
ORR = complete response (CR) + CR with incomplete blood count recovery (CRi) + partial remission (PR) as defined by 2003 IWG criteria for AML patients, and ORR = CR + PR + hematologic improvement (HI) and as defined per 2006 IWG criteria for MDS patients
Measured: At 1.5 years
Overall response rate (ORR) per 2003 IWG criteria
ORR = complete response (CR) + CR with incomplete blood count recovery (CRi) + partial remission (PR) as defined by 2003 IWG criteria for AML patients, and ORR = CR + PR + hematologic improvement (HI) and as defined per 2006 IWG criteria for MDS patients
Measured: At 2 years
Overall response rate (ORR) per 2003 IWG criteria
ORR = complete response (CR) + CR with incomplete blood count recovery (CRi) + partial remission (PR) as defined by 2003 IWG criteria for AML patients, and ORR = CR + PR + hematologic improvement (HI) and as defined per 2006 IWG criteria for MDS patients
Measured: At 2.5 years
Overall response rate (ORR) per 2003 IWG criteria
ORR = complete response (CR) + CR with incomplete blood count recovery (CRi) + partial remission (PR) as defined by 2003 IWG criteria for AML patients, and ORR = CR + PR + hematologic improvement (HI) and as defined per 2006 IWG criteria for MDS patients
Measured: At 3 years
Overall response rate (ORR) per 2003 IWG criteria
ORR = complete response (CR) + CR with incomplete blood count recovery (CRi) + partial remission (PR) as defined by 2003 IWG criteria for AML patients, and ORR = CR + PR + hematologic improvement (HI) and as defined per 2006 IWG criteria for MDS patients
Measured: At 3.5 years
Overall response rate (ORR) per 2003 IWG criteria
ORR = complete response (CR) + CR with incomplete blood count recovery (CRi) + partial remission (PR) as defined by 2003 IWG criteria for AML patients, and ORR = CR + PR + hematologic improvement (HI) and as defined per 2006 IWG criteria for MDS patients
Measured: At 4 years
Overall response rate (ORR) per 2003 IWG criteria
ORR = complete response (CR) + CR with incomplete blood count recovery (CRi) + partial remission (PR) as defined by 2003 IWG criteria for AML patients, and ORR = CR + PR + hematologic improvement (HI) and as defined per 2006 IWG criteria for MDS patients
Measured: At 4.5 years
Overall response rate (ORR) per 2003 IWG criteria
ORR = complete response (CR) + CR with incomplete blood count recovery (CRi) + partial remission (PR) as defined by 2003 IWG criteria for AML patients, and ORR = CR + PR + hematologic improvement (HI) and as defined per 2006 IWG criteria for MDS patients
Measured: At 5 years
Secondary outcomes (40)
Time to response (TTR)
TTR is measured from start of treatment to the date of achieving a response. Wilcoxon rank test will be used for comparison of continuous variables.
Measured: At 6 months
Time to response (TTR)
TTR is measured from start of treatment to the date of achieving a response. Wilcoxon rank test will be used for comparison of continuous variables.
Measured: At 1 year
Time to response (TTR)
TTR is measured from start of treatment to the date of achieving a response. Wilcoxon rank test will be used for comparison of continuous variables.
Measured: At 1.5 years
Time to response (TTR)
TTR is measured from start of treatment to the date of achieving a response. Wilcoxon rank test will be used for comparison of continuous variables.
Measured: At 2 years
Time to response (TTR)
TTR is measured from start of treatment to the date of achieving a response. Wilcoxon rank test will be used for comparison of continuous variables.
Measured: At 2.5 years
Time to response (TTR)
TTR is measured from start of treatment to the date of achieving a response. Wilcoxon rank test will be used for comparison of continuous variables.
Measured: At 3 years
Time to response (TTR)
TTR is measured from start of treatment to the date of achieving a response. Wilcoxon rank test will be used for comparison of continuous variables.
Measured: At 3.5 years
Time to response (TTR)
TTR is measured from start of treatment to the date of achieving a response. Wilcoxon rank test will be used for comparison of continuous variables.
Measured: At 4 years
Time to response (TTR)
TTR is measured from start of treatment to the date of achieving a response. Wilcoxon rank test will be used for comparison of continuous variables.
Measured: At 4.5 years
Time to response (TTR)
TTR is measured from start of treatment to the date of achieving a response. Wilcoxon rank test will be used for comparison of continuous variables.
Measured: At 5 years
Duration of response (DOR)
DOR measured from the time of achieving a response to time of disease progression. Wilcoxon rank test will be used for comparison of continuous variables.
Measured: At 6 months
Duration of response (DOR)
DOR measured from the time of achieving a response to time of disease progression. Wilcoxon rank test will be used for comparison of continuous variables.
Measured: At 1 year
Duration of response (DOR)
DOR measured from the time of achieving a response to time of disease progression. Wilcoxon rank test will be used for comparison of continuous variables.
Measured: At 1.5 years
Duration of response (DOR)
DOR measured from the time of achieving a response to time of disease progression. Wilcoxon rank test will be used for comparison of continuous variables.
Measured: At 2 years
Duration of response (DOR)
DOR measured from the time of achieving a response to time of disease progression. Wilcoxon rank test will be used for comparison of continuous variables.
Measured: At 2.5 years
Duration of response (DOR)
DOR measured from the time of achieving a response to time of disease progression. Wilcoxon rank test will be used for comparison of continuous variables.
Measured: At 3 years
Duration of response (DOR)
DOR measured from the time of achieving a response to time of disease progression. Wilcoxon rank test will be used for comparison of continuous variables.
Measured: At 3.5 years
Duration of response (DOR)
DOR measured from the time of achieving a response to time of disease progression. Wilcoxon rank test will be used for comparison of continuous variables.
Measured: At 4 years
Duration of response (DOR)
DOR measured from the time of achieving a response to time of disease progression. Wilcoxon rank test will be used for comparison of continuous variables.
Measured: At 4.5 years
Duration of response (DOR)
DOR measured from the time of achieving a response to time of disease progression. Wilcoxon rank test will be used for comparison of continuous variables.
Measured: At 5 years
Event-free survival (EFS)
EFS measured from date of first dose to the date of treatment failure, relapse, or death from any cause. Wilcoxon rank test will be used for comparison of continuous variables
Measured: At 6 months
Event-free survival (EFS)
EFS measured from date of first dose to the date of treatment failure, relapse, or death from any cause. Wilcoxon rank test will be used for comparison of continuous variables
Measured: At 1 year
Event-free survival (EFS)
EFS measured from date of first dose to the date of treatment failure, relapse, or death from any cause. Wilcoxon rank test will be used for comparison of continuous variables
Measured: At 1.5 years
Event-free survival (EFS)
EFS measured from date of first dose to the date of treatment failure, relapse, or death from any cause. Wilcoxon rank test will be used for comparison of continuous variables
Measured: At 2 years
Event-free survival (EFS)
EFS measured from date of first dose to the date of treatment failure, relapse, or death from any cause. Wilcoxon rank test will be used for comparison of continuous variables
Measured: At 2.5 years
Event-free survival (EFS)
EFS measured from date of first dose to the date of treatment failure, relapse, or death from any cause. Wilcoxon rank test will be used for comparison of continuous variables
Measured: At 3 years
Event-free survival (EFS)
EFS measured from date of first dose to the date of treatment failure, relapse, or death from any cause. Wilcoxon rank test will be used for comparison of continuous variables
Measured: At 3.5 years
Event-free survival (EFS)
EFS measured from date of first dose to the date of treatment failure, relapse, or death from any cause. Wilcoxon rank test will be used for comparison of continuous variables
Measured: At 4 years
Event-free survival (EFS)
EFS measured from date of first dose to the date of treatment failure, relapse, or death from any cause. Wilcoxon rank test will be used for comparison of continuous variables
Measured: At 4.5 years
Event-free survival (EFS)
EFS measured from date of first dose to the date of treatment failure, relapse, or death from any cause. Wilcoxon rank test will be used for comparison of continuous variables
Measured: At 5 years
Overall Survival (OS)
OS measured from start of treatment to death or last follow up. OS function will be estimated using the Kaplan-Meier method
Measured: At 6 months
Overall Survival (OS)
OS measured from start of treatment to death or last follow up. OS function will be estimated using the Kaplan-Meier method
Measured: At 1 year
Overall Survival (OS)
OS measured from start of treatment to death or last follow up. OS function will be estimated using the Kaplan-Meier method
Measured: At 1.5 years
Overall Survival (OS)
OS measured from start of treatment to death or last follow up. OS function will be estimated using the Kaplan-Meier method
Measured: At 2 years
Overall Survival (OS)
OS measured from start of treatment to death or last follow up. OS function will be estimated using the Kaplan-Meier method
Measured: At 2.5 years
Overall Survival (OS)
OS measured from start of treatment to death or last follow up. OS function will be estimated using the Kaplan-Meier method
Measured: At 3 years
Overall Survival (OS)
OS measured from start of treatment to death or last follow up. OS function will be estimated using the Kaplan-Meier method
Measured: At 3.5 years
Overall Survival (OS)
OS measured from start of treatment to death or last follow up. OS function will be estimated using the Kaplan-Meier method
Measured: At 4 years
Overall Survival (OS)
OS measured from start of treatment to death or last follow up. OS function will be estimated using the Kaplan-Meier method
Measured: At 4.5 years
Overall Survival (OS)
OS measured from start of treatment to death or last follow up. OS function will be estimated using the Kaplan-Meier method
Measured: At 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Dec 2020
- Study start14 Jan 2021
- Primary completion19 Jan 2026
- Study completion19 Jan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Refractory Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by Case Comprehensive Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Case Comprehensive Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Sudipto Mukherjee, MD, PhD, Principal investigator, Cleveland Clinic, Case Comprehensive Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04668885
- Sponsor's protocol ID
- CASE1920
- Status last verified
- Jun 2026
- First submitted
- 9 Dec 2020
- Last updated
- 12 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: There are no plans to share individual patient data in order to protect the confidentiality of the participants who enroll on this study
Study at a glance
From the registry record, last updated by the sponsor on 12 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Lower Doses of CPX-351 as a Novel Approach for the Treatment of Older Patients With Relapsed or Refractory Acute Myeloid Leukemia and Myelodysplastic Syndromes
- ClinicalTrials.gov ID
- NCT04668885
- Acronym / sponsor ID
- CASE1920
- Conditions
- Refractory Acute Myeloid Leukemia, Relapsed Acute Myelomonocytic Leukemia, Myelodysplastic Syndromes
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Case Comprehensive Cancer CenterUniversity, hospital or other
- Study start
- 14 Jan 2021 (actual)
- Primary completion
- 19 Jan 2026 (actual)
- Study completion
- 19 Jan 2027 (estimated)
- First posted
- 16 Dec 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate how effective lower doses of CPX-351 are in older participants with relapsed/refractory acute myeloid leukemia (AML) who are not eligible to receive intensive chemotherapy and in participants with myelodysplastic syndromes (MDS) after Hypomethylating Agents (HMA) failure.
Detailed description
Currently, elderly patients with AML and high risk MDS, who are ineligible to receive induction chemotherapy and fail HMA +/- combination, have very poor outcomes and there is no FDA-approved therapy outside of some targeted therapies which can only be applied to a small patient population. CPX-351 is an investigational (experimental) drug that works by combining two anti-cancer drugs cytarabine and daunorubicin. CPX-351 is experimental because it is only FDA approved for the treatment of adults with two types of AML: newly diagnosed therapy-related AML or AML with myelodysplasia-related changes.
This is an open label clinical trial of lower doses of CPX-351 in relapsed/primary refractory older AML and MDS patients ineligible to receive intensive chemotherapy. The first arm is for particpants with primary refractory/relapsed AML and the second arm is for higher risk MDS participants after HMA failure.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Primary refractory/relapsed AML | Experimental | Drug: CPX-351Lower dose CPX-351 in participants with primary refractory/relapsed AML. Participants will receive an induction and maintenance phase of CPX-351 |
| MDS after HMA failure | Experimental | Drug: CPX-351Lower dose CPX-351 in participants with MDS after HMA failure. Participants will receive an induction and maintenance phase of CPX-351 |
Who may qualify
- Ages
- 60 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Primary refractory or relapsed AML (defined by 2016 World Health Organization [WHO] criteria) patients who are not suitable for or not willing to receive intensive chemotherapy as evaluated by the treating physician. Primary refractory disease is defined as:
- Participants with MDS (according to 2016 WHO criteria) who did not respond to treatment with azacitidine, decitabine, or combination of HMA with another drug or lost their response to initial therapy with HMA.
- Eastern Cooperative Oncology Group (ECOG) performance status <=2
- Adequate hepatic (serum total bilirubin < 1.5 x ULN, Serum glutamic pyruvic transaminase (SGPT) and/or serum glutamate oxaloacetate transaminase (SGOT) <2.5 x ULN) and renal function (creatinine < 1.5mg/dL).
- Participants must be willing and able to review, understand, and provide written consent before starting therapy.
Key exclusion criteria (14 listed)
- Active signs or symptoms of central nervous system (CNS) involvement by malignancy (lumbar puncture [LP] not required).
- Prior 7+3 remission induction chemotherapy for MDS or AML
- More than 2 lines of prior non-intensive therapy.
- New York Heart Association (NYHA) class III or IV heart disease, active ischemia or any other uncontrolled cardiac condition such as angina pectoris, clinically significant cardiac arrhythmia on rhythm control strategy or on pacemaker, uncontrolled hypertension (blood pressure > 160 systolic and > 110 diastolic not responsive to antihypertensive medication)
- Acute myocardial infarction in the previous 12 weeks (from the start of treatment).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CPX-351Induction phase: CPX-351 15 mg/m^2 on days 1 and 3 of each 28-day cycle for up to a total of 6 cycles in the absence of unacceptable toxicity Maintenance phase: CPX-351 7.5 mg/m^2 on days 1 and 3 for two cycles alternating with 15 mg/m^2 for one cycle. Participants may receive up to 12 cycles of maintenance phase in the absence of unacceptable toxicity. | Drug | MDS after HMA failure, Primary refractory/relapsed AML |
Primary outcome
Overall response rate (ORR) per 2003 International Working Group (IWG) criteria
Time frame: At 6 months
ORR = complete response (CR) + CR with incomplete blood count recovery (CRi) + partial remission (PR) as defined by 2003 IWG criteria for AML patients, and ORR = CR + PR + hematologic improvement (HI) and as defined per 2006 IWG criteria for MDS patients
Overall response rate (ORR) per 2003 IWG criteria
Time frame: At 1 year
ORR = complete response (CR) + CR with incomplete blood count recovery (CRi) + partial remission (PR) as defined by 2003 IWG criteria for AML patients, and ORR = CR + PR + hematologic improvement (HI) and as defined per 2006 IWG criteria for MDS patients
Overall response rate (ORR) per 2003 IWG criteria
Time frame: At 1.5 years
ORR = complete response (CR) + CR with incomplete blood count recovery (CRi) + partial remission (PR) as defined by 2003 IWG criteria for AML patients, and ORR = CR + PR + hematologic improvement (HI) and as defined per 2006 IWG criteria for MDS patients
Overall response rate (ORR) per 2003 IWG criteria
Time frame: At 2 years
ORR = complete response (CR) + CR with incomplete blood count recovery (CRi) + partial remission (PR) as defined by 2003 IWG criteria for AML patients, and ORR = CR + PR + hematologic improvement (HI) and as defined per 2006 IWG criteria for MDS patients
Overall response rate (ORR) per 2003 IWG criteria
Time frame: At 2.5 years
ORR = complete response (CR) + CR with incomplete blood count recovery (CRi) + partial remission (PR) as defined by 2003 IWG criteria for AML patients, and ORR = CR + PR + hematologic improvement (HI) and as defined per 2006 IWG criteria for MDS patients
Overall response rate (ORR) per 2003 IWG criteria
Time frame: At 3 years
ORR = complete response (CR) + CR with incomplete blood count recovery (CRi) + partial remission (PR) as defined by 2003 IWG criteria for AML patients, and ORR = CR + PR + hematologic improvement (HI) and as defined per 2006 IWG criteria for MDS patients
Overall response rate (ORR) per 2003 IWG criteria
Time frame: At 3.5 years
ORR = complete response (CR) + CR with incomplete blood count recovery (CRi) + partial remission (PR) as defined by 2003 IWG criteria for AML patients, and ORR = CR + PR + hematologic improvement (HI) and as defined per 2006 IWG criteria for MDS patients
Overall response rate (ORR) per 2003 IWG criteria
Time frame: At 4 years
ORR = complete response (CR) + CR with incomplete blood count recovery (CRi) + partial remission (PR) as defined by 2003 IWG criteria for AML patients, and ORR = CR + PR + hematologic improvement (HI) and as defined per 2006 IWG criteria for MDS patients
Overall response rate (ORR) per 2003 IWG criteria
Time frame: At 4.5 years
ORR = complete response (CR) + CR with incomplete blood count recovery (CRi) + partial remission (PR) as defined by 2003 IWG criteria for AML patients, and ORR = CR + PR + hematologic improvement (HI) and as defined per 2006 IWG criteria for MDS patients
Overall response rate (ORR) per 2003 IWG criteria
Time frame: At 5 years
ORR = complete response (CR) + CR with incomplete blood count recovery (CRi) + partial remission (PR) as defined by 2003 IWG criteria for AML patients, and ORR = CR + PR + hematologic improvement (HI) and as defined per 2006 IWG criteria for MDS patients
40 secondary outcomes
Time to response (TTR)
Time frame: At 6 months
Time to response (TTR)
Time frame: At 1 year
Time to response (TTR)
Time frame: At 1.5 years
Time to response (TTR)
Time frame: At 2 years
Time to response (TTR)
Time frame: At 2.5 years
Time to response (TTR)
Time frame: At 3 years
Time to response (TTR)
Time frame: At 3.5 years
Time to response (TTR)
Time frame: At 4 years
Time to response (TTR)
Time frame: At 4.5 years
Time to response (TTR)
Time frame: At 5 years
Duration of response (DOR)
Time frame: At 6 months
Duration of response (DOR)
Time frame: At 1 year
Duration of response (DOR)
Time frame: At 1.5 years
Duration of response (DOR)
Time frame: At 2 years
Duration of response (DOR)
Time frame: At 2.5 years
Duration of response (DOR)
Time frame: At 3 years
Duration of response (DOR)
Time frame: At 3.5 years
Duration of response (DOR)
Time frame: At 4 years
Duration of response (DOR)
Time frame: At 4.5 years
Duration of response (DOR)
Time frame: At 5 years
Event-free survival (EFS)
Time frame: At 6 months
Event-free survival (EFS)
Time frame: At 1 year
Event-free survival (EFS)
Time frame: At 1.5 years
Event-free survival (EFS)
Time frame: At 2 years
Event-free survival (EFS)
Time frame: At 2.5 years
Event-free survival (EFS)
Time frame: At 3 years
Event-free survival (EFS)
Time frame: At 3.5 years
Event-free survival (EFS)
Time frame: At 4 years
Event-free survival (EFS)
Time frame: At 4.5 years
Event-free survival (EFS)
Time frame: At 5 years
Overall Survival (OS)
Time frame: At 6 months
Overall Survival (OS)
Time frame: At 1 year
Overall Survival (OS)
Time frame: At 1.5 years
Overall Survival (OS)
Time frame: At 2 years
Overall Survival (OS)
Time frame: At 2.5 years
Overall Survival (OS)
Time frame: At 3 years
Overall Survival (OS)
Time frame: At 3.5 years
Overall Survival (OS)
Time frame: At 4 years
Overall Survival (OS)
Time frame: At 4.5 years
Overall Survival (OS)
Time frame: At 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jun 2026.
- Individual participant dataSharing planned: No. There are no plans to share individual patient data in order to protect the confidentiality of the participants who enroll on this study
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sudipto Mukherjee, MD, PhD principal investigator
Cleveland Clinic, Case Comprehensive Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CPX-351 in people with Refractory Acute Myeloid Leukemia and Relapsed Acute Myelomonocytic Leukemia, run by Case Comprehensive Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 12 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall response rate (ORR) per 2003 International Working Group (IWG) criteria (time frame: At 6 months). The study lists 10 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Jun 2026.