Acute Myeloid Leukemia Arising From Previous Myelodysplastic SyndromePhase 2NCT04269213
CPX-351 for the Treatment of Secondary Acute Myeloid Leukemia in Patients Younger Than 60 Years Old
A Phase 2 study testing Liposome-encapsulated Daunorubicin-Cytarabine in people with Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome and Acute Myeloid Leukemia With Myelodysplasia-Related Changes, run by Roswell Park Cancer Institute at 4 sites in United States.
Trial summary
This phase II trial studies how well liposome-encapsulated daunorubicin-cytarabine (CPX-351) works in treating patients with secondary acute myeloid leukemia who are younger than 60 years old. Read more about this study
- Who is it for?
- Ages 18 to 59 years
- Conditions the study is for
- Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Acute Myeloid Leukemia With Myelodysplasia-Related Changes, Secondary Acute Myeloid Leukemia
- What is being tested?
- Liposome-encapsulated Daunorubicin-Cytarabine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 4 sitesUnited States
- Study dates
- 29 Jul 2021to 16 Apr 2026
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 to 59 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Newly diagnosed:
- Therapy-related acute myeloid leukemia (AML)
- AML with antecedent myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML)
- AML with MDS-related changes (as per World Health Organization [WHO])
- Have an Eastern Cooperative Oncology Group (ECOG) performance status of =< 2
- Plasma creatinine =< 1.5 x upper limit of normal (ULN)
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- Total bilirubin < 2.0 mg/dL
- Serum alanine aminotransferase and aspartate aminotransferase < 3 x ULN
- Left ventricular ejection fraction by echocardiogram or multiple-gated acquisition >= 50%
- Participants of child-bearing potential must agree to use adequate contraceptive methods (e.g., hormonal or barrier method of birth control; abstinence) prior to study entry. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately
- Women of childbearing potential must have a negative serum or urine pregnancy test within 72 hours prior to enrollment and commit to two forms of birth control
- Men must use a latex condom during any sexual contact with women of childbearing potential
- Willing to adhere to protocol specific requirements
- Participant or legal representative must understand the investigational nature of this study and sign an Independent Ethics Committee/Institutional Review Board approved written informed consent form prior to receiving any study related procedure
Who cannot join
- Prior treatment of AML
- Known clinically active central nervous system (CNS) leukemia
- Core-binding factor leukemia
- Acute promyelocytic leukemia
- Uncontrolled other malignancy
- Prior anthracycline exposure > 368 mg/m^2 of daunorubicin or equivalent
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- Cardiovascular disease resulting in heart failure (New York Heart Association class III or IV), unstable angina (angina symptoms at rest), or new onset angina (began within the last 3 months) or myocardial infarction within the past 6 months
- Hypersensitivity to cytarabine, daunorubicin, or liposomal drugs
- Known active HIV infection
- Known history of active hepatitis B or C infection
- Pre-existing liver disease (e.g. cirrhosis, chronic hepatitis B or C, nonalcoholic steatohepatitis, sclerosing cholangitis)
- Evidence of ongoing, uncontrolled systemic infection
- Pregnant or breastfeeding women
- Subject with concurrent severe and/or uncontrolled medical or psychiatric conditions that in the opinion of the investigator may impair the participation in the study or the evaluation of safety and/or efficacy
- History of Wilson disease or other copper-handling disorders
- Any condition which in the investigator's opinion deems the participant an unsuitable candidate to receive study drug
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Roswell Park Cancer Institute
Buffalo, New York, 14263
University of Nebraska Medical Center
Omaha, Nebraska, 68198
Allegheny Health Network Cancer Institute - West Penn Hospital
Pittsburgh, Pennsylvania, 15224
SUNY Upstate Medical Center
Syracuse, New York, 13210
About this study
Official title: A Phase II Study of CPX-351 in Younger Patients < 60 Years Old With Secondary Acute Myeloid Leukemia
This phase II trial studies how well liposome-encapsulated daunorubicin-cytarabine (CPX-351) works in treating patients with secondary acute myeloid leukemia who are younger than 60 years old. Drugs used in chemotherapy, such as CPX-351, 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.
Read the detailed description
PRIMARY OBJECTIVE:
- To determine the complete response rate including morphologic complete remission (CR) and morphologic complete remission with incomplete blood count recovery (CRi) as defined by the International Working Group Criteria.
SECONDARY OBJECTIVE:
- To determine CR + CRi duration, event free survival (EFS), overall survival (OS), patients successfully proceeding to allogenic hematopoietic cell transplant, and adverse events (AE).
OUTLINE:
INDUCTION: Patients receive liposome-encapsulated daunorubicin-cytarabine intravenously (IV) over 90 minutes on days 1, 3, and 5 in the absence of disease progression or unacceptable toxicity.
RE-INDUCTION: Patients who do not achieve remission receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3 in the absence of disease progression or unacceptable toxicity.
CONSOLIDATION: Beginning 5-8 weeks after the start of the last induction, patients who achieve CR receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3. Treatment repeats every 45 days for up to 2 cycles in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up at 30 days, and then every 3 months for up to 5 years.
- Conditions
- Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Acute Myeloid Leukemia With Myelodysplasia-Related Changes, Secondary Acute Myeloid Leukemia, Therapy-Related Acute Myeloid Leukemia
- Being tested
- Liposome-encapsulated Daunorubicin-Cytarabine
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Liposome-encapsulated Daunorubicin-CytarabineDrug
Also called CPX-351, Cytarabine-Daunorubicin Liposome for Injection, Liposomal AraC-Daunorubicin CPX-351, Liposomal Cytarabine-Daunorubicin
Given IV
| Study group | Role | What they receive |
|---|---|---|
| Treatment (CPX-351) | Experimental | INDUCTION: Patients receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5 in the absence of disease progression or unacceptable toxicity.
RE-INDUCTION: Patients who do not achieve remission receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3 in the absence of disease progression or unacceptable toxicity.
CONSOLIDATION: Beginning 5-8 weeks after the start of the last induction, patients who achieve CR receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3. Treatment repeats every 45 days for up to 2 cycles in the absence of disease progression or unacceptable toxicity. Receives: Drug: Liposome-encapsulated Daunorubicin-Cytarabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete Response Rate (Morphological Complete Remission [CR] and Incomplete Blood Count Recovery [CRi])
Defined by the International Working Group Criteria. Will be summarized using frequencies and relative frequencies.
Measured: At day 45
Secondary outcomes (5)
CR + CRi Duration
Will be summarized using standard Kaplan-Meier methods, where estimates of the median obtained with 90% confidence intervals.
Measured: Time from overall CR or CRi until relapse or last follow-up, assessed up to 50 months
Event Free Survival
Will be summarized using standard Kaplan-Meier methods, where estimates of the median obtained with 90% confidence intervals.
Measured: Time from treating until disease progression/relapse, death due to disease, or last follow-up, assessed up to 50 months
Overall Survival
Will be summarized using standard Kaplan-Meier methods, where estimates of the median obtained with 90% confidence intervals.
Measured: Time from treatment until death due to any cause or last follow-up, assessed up to 50 months
Allogeneic Hematopoietic Cell Transplant (HSCT) Rate
Transplant rate estimated using a 90% confidence interval obtained using Jeffrey's prior method.
Measured: Up to 50 months
Incidence of Adverse Events
Will report Incidence of adverse events with highest maximum grade seen using frequencies and relative frequencies.
Measured: Up to 50 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Feb 2020
- Study start29 Jul 2021
- Primary completion27 May 2025
- Study completion16 Apr 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Acute Myeloid Leukemia Arising From Previous Myelodysplastic SyndromeStudies of the same condition, open and under way.
- More trials run by Roswell Park Cancer InstituteThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Roswell Park Cancer Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Jazz Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Eunice Wang, MD, Principal investigator, Roswell Park Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04269213
- Sponsor's protocol ID
- I 501719
- Other IDs
- NCI-2019-08946, I 501719
- Status last verified
- Sep 2026
- First submitted
- 11 Feb 2020
- Last updated
- 8 Sep 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of CPX-351 in Younger Patients < 60 Years Old With Secondary Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT04269213
- Acronym / sponsor ID
- I 501719
- Conditions
- Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Acute Myeloid Leukemia With Myelodysplasia-Related Changes, Secondary Acute Myeloid Leukemia, Therapy-Related Acute Myeloid Leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Roswell Park Cancer InstituteUniversity, hospital or other
- Collaborators
- Jazz Pharmaceuticals
- Study start
- 29 Jul 2021 (actual)
- Primary completion
- 27 May 2025 (actual)
- Study completion
- 16 Apr 2026 (actual)
- First posted
- 13 Feb 2020 (actual)
- Results
- Posted 31 Aug 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase II trial studies how well liposome-encapsulated daunorubicin-cytarabine (CPX-351) works in treating patients with secondary acute myeloid leukemia who are younger than 60 years old. Drugs used in chemotherapy, such as CPX-351, 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.
Detailed description
PRIMARY OBJECTIVE:
- To determine the complete response rate including morphologic complete remission (CR) and morphologic complete remission with incomplete blood count recovery (CRi) as defined by the International Working Group Criteria.
SECONDARY OBJECTIVE:
- To determine CR + CRi duration, event free survival (EFS), overall survival (OS), patients successfully proceeding to allogenic hematopoietic cell transplant, and adverse events (AE).
OUTLINE:
INDUCTION: Patients receive liposome-encapsulated daunorubicin-cytarabine intravenously (IV) over 90 minutes on days 1, 3, and 5 in the absence of disease progression or unacceptable toxicity.
RE-INDUCTION: Patients who do not achieve remission receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3 in the absence of disease progression or unacceptable toxicity.
CONSOLIDATION: Beginning 5-8 weeks after the start of the last induction, patients who achieve CR receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3. Treatment repeats every 45 days for up to 2 cycles in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up at 30 days, and then every 3 months for up to 5 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (CPX-351) | Experimental | Drug: Liposome-encapsulated Daunorubicin-CytarabineINDUCTION: Patients receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5 in the absence of disease progression or unacceptable toxicity. RE-INDUCTION: Patients who do not achieve remission receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3 in the absence of disease progression or unacceptable toxicity. CONSOLIDATION: Beginning 5-8 weeks after the start of the last induction, patients who achieve CR receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3. Treatment repeats every 45 days for up to 2 cycles in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 to 59 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (14 listed)
- Newly diagnosed:
- Have an Eastern Cooperative Oncology Group (ECOG) performance status of =< 2
- Plasma creatinine =< 1.5 x upper limit of normal (ULN)
- Total bilirubin < 2.0 mg/dL
- Serum alanine aminotransferase and aspartate aminotransferase < 3 x ULN
Key exclusion criteria (16 listed)
- Prior treatment of AML
- Known clinically active central nervous system (CNS) leukemia
- Core-binding factor leukemia
- Acute promyelocytic leukemia
- Uncontrolled other malignancy
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 |
|---|---|---|
| Liposome-encapsulated Daunorubicin-CytarabineAlso: CPX-351, Cytarabine-Daunorubicin Liposome for Injection, Liposomal AraC-Daunorubicin CPX-351, Liposomal Cytarabine-DaunorubicinGiven IV | Drug | Treatment (CPX-351) |
Primary outcome
Complete Response Rate (Morphological Complete Remission [CR] and Incomplete Blood Count Recovery [CRi])
Time frame: At day 45
Defined by the International Working Group Criteria. Will be summarized using frequencies and relative frequencies.
5 secondary outcomes
CR + CRi Duration
Time frame: Time from overall CR or CRi until relapse or last follow-up, assessed up to 50 months
Event Free Survival
Time frame: Time from treating until disease progression/relapse, death due to disease, or last follow-up, assessed up to 50 months
Overall Survival
Time frame: Time from treatment until death due to any cause or last follow-up, assessed up to 50 months
Allogeneic Hematopoietic Cell Transplant (HSCT) Rate
Time frame: Up to 50 months
Incidence of Adverse Events
Time frame: Up to 50 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Sep 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Eunice Wang, MD principal investigator
Roswell Park Cancer Institute
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Liposome-encapsulated Daunorubicin-Cytarabine in people with Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome and Acute Myeloid Leukemia With Myelodysplasia-Related Changes, run by Roswell Park Cancer Institute at 4 sites 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 14 inclusion and 16 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: finished, as of the sponsor's last update on 8 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites 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?
Yes. Results are posted in the registry (first posted 31 Aug 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Complete Response Rate (Morphological Complete Remission [CR] and Incomplete Blood Count Recovery [CRi]) (time frame: At day 45). 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 8 Sep 2026.