High Risk Acute Myeloid LeukemiaPhase 3301NCT01696084
Phase III Study of CPX-351 Versus 7+3 in Patients 60-75 Years Old With Untreated High Risk (Secondary) Acute Myeloid Leukemia
A Phase 3 study testing CPX-351 and 7+3 (cytarabine and daunorubicin) in people with High Risk Acute Myeloid Leukemia, run by Jazz Pharmaceuticals at 43 sites in 2 countries.
Trial summary
To confirm the efficacy of CPX-351 compared to 7+3 as first line therapy in elderly patients (60-75 yrs) with high risk (secondary) Acute Myeloid Leukemia. The primary efficacy endpoint will be overall survival. Read more about this study
- Who is it for?
- Ages 60 to 75 years
- Conditions the study is for
- High Risk Acute Myeloid Leukemia
- What is being tested?
- CPX-351 and 7+3 (cytarabine and daunorubicin)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 309 participants
- Where
- 43 sitesin 2 countries
- Study dates
- 13 Dec 2012to 31 Dec 2015
- Phase
- Phase 3
- 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 60 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Ability to understand and voluntarily give informed consent
- Age 60-75 years at the time of diagnosis of AML
- Pathological diagnosis of AML according to WHO criteria (with at least 20% blasts in the peripheral blood or bone marrow)
- Confirmation of:
- Therapy related AML: t-AML must have a documented history of prior cytotoxic therapy or ionizing radiotherapy for an unrelated disease
- AML with a history of myelodysplasia: MDSAML must have bone marrow documentation of prior MDS
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- AML with a history of CMMoL: CMMoLAML must have bone marrow documentation of prior CMMoL
- De novo AML with karyotypic abnormalities characteristic of MDS: de novoAML must have cytogenetics with abnormalities per WHO.
- Eastern Cooperative Oncology Group (ECOG) performance status 0-2
- Able to adhere to the study visit schedule and other protocol requirements
- Laboratory values fulfilling the following:
- Serum creatinine < 2.0 mg/dL
- Serum total bilirubin < 2.0 mg/dL, patients with Gilbert's Syndrome should contact the medical monitor
- Serum alanine aminotransferase or aspartate aminotransferase < 3 times the ULN Note: If elevated liver enzymes, above the ULN, are related to disease; contact medical monitor to discuss.
- Cardiac ejection fraction ≥ 50% by echocardiography or MUGA
- Patients with second malignancies in remission may be eligible if there is clinical evidence of disease stability for a period of greater than 6 months off cytotoxic chemotherapy, documented by imaging, tumor marker studies, etc., at screening. Patients maintained on long-term non-chemotherapy treatment, e.g., hormonal therapy, are eligible.
Who cannot join
- Except for CMMoL, patients with history of myeloproliferative neoplasms (MPN) (defined as a history of essential thrombocytosis or polycythemia vera, or idiopathic myelofibrosis prior to the diagnosis of AML) or combined MDS/MPN are not eligible.
- Acute promyelocytic leukemia [t(15;17)] or favorable cytogenetics, including t(8;21) or inv16 if known at the time of randomization.
- Clinical evidence of active CNS leukemia
- Patients with active (uncontrolled, metastatic) second malignancies are excluded.
- Prior treatment intended for induction therapy of AML; only hydroxyurea is permitted for control of blood counts. For example, a patient with MDS that changes HMA dose and schedule after the diagnosis of AML is excluded. AML-type therapy, such as cytarabine alone (>1g/m2/day) or cytarabine plus an anthracycline as well as prior HSCT are also excluded.
- Administration of any therapy for MDS (conventional or investigational) must be completed by 2 weeks prior to of the first dose of study drug; in the event of rapidly proliferative disease use of hydroxyurea is permitted until 24 hours before the start of study treatment. Toxicities associated with prior MDS therapy must have recovered to grade 1 or less prior to start of treatment.
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- Any major surgery or radiation therapy within four weeks.
- Patients with prior cumulative anthracycline exposure of greater than 368 mg/m2 daunorubicin (or equivalent).
- Any serious medical condition, laboratory abnormality or psychiatric illness that would prevent obtaining informed consent
- Patients with myocardial impairment of any cause (e.g. cardiomyopathy, ischemic heart disease, significant valvular dysfunction, hypertensive heart disease, and congestive heart failure) resulting in heart failure by New York Heart Association Criteria (Class III or IV staging)
- Active or uncontrolled infection. Patients with an infection receiving treatment (antibiotic, antifungal or antiviral treatment) may be entered into the study but must be afebrile and hemodynamically stable for ≥72 hrs.
- Current evidence of invasive fungal infection (blood or tissue culture); patients with recent fungal infection must have a subsequent negative cultures to be eligible; known HIV (new testing not required) or evidence of active hepatitis B or C infection (with rising transaminase values)
- Hypersensitivity to cytarabine, daunorubicin or liposomal products
- History of Wilson's disease or other copper-metabolism disorder
Trial locations and contacts
43 sites in 2 countries.
43 sites in 2 countries
United States39 sites
University of Michigan
Ann Arbor, Michigan, 48109
Northside Hospital
Atlanta, Georgia, 30342
University of Alabama at Birmingham
Birmingham, Alabama, 35294
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599-1651
Medical University of South Carolina
Charleston, South Carolina, 29425
Northwestern University
Chicago, Illinois, 60208
Rush University Medical Center
Chicago, Illinois, 60612
University of Chicago
Chicago, Illinois, 60637
University of Missouri
Columbia, Missouri, 65211
Baylor Research Insitute
Dallas, Texas, 75246
Duke University Medical Center
Durham, North Carolina, 27710
University of Florida
Gainesville, Florida, 32611
Hackensack University Medical Center
Hackensack, New Jersey, 07601
M.D. Anderson Cancer Center
Houston, Texas, 770303
Franciscan St. Francis Health
Indianapolis, Indiana, 46237
University of Kansas Medical Center
Kansas City, Kansas, 66160
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire, 03756
North Shore LIJ Health System
Long Island City, New York
UCLA
Los Angeles, California, 90095
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
University of Minnesota
Minneapolis, Minnesota, 55455
Sarah Cannon Research Institute
Nashville, Tennessee, 37203
Vanderbilt University
Nashville, Tennessee, 37232
Yale University
New Haven, Connecticut, 06510
Columbia University
New York, New York, 10032
Cornell U, Weill Medical College
New York, New York, 10065
Fox Chase Cancer Center
Philadelphia, Pennsylvania, 19111
UPMC
Pittsburgh, Pennsylvania, 15219
Providence Portland Medical Center
Portland, Oregon, 97213
Oregon Health and Science University
Portland, Oregon, 97239
University of CA San Diego
San Diego, California, 92037-0706
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109
Washington University
St Louis, Missouri, 63110
Stanford University
Stanford, California, 94305
H. Lee Moffitt Cancer Center
Tampa, Florida, 33612
Montefiore Medical Center
The Bronx, New York, 10467
New York Medical College
Valhalla, New York, 10595
Wake Forest University Health Services
Winston-Salem, North Carolina, 27157
Canada4 sites
University of Alberta Hospital
Edmonton, Alberta, T6G 2C8
Hopital Maisonneuve-Rosemont
Montreal, Quebec
Princess Margaret Hospital
Toronto, Ontario, M5G2M9
British Columbia Cancer Center
Vancouver, British Columbia, V5Z 1M9
About this study
Official title: Phase III, Multicenter, Randomized, Trial of CPX-351 (Cytarabine:Daunorubicin) Liposome Injection Versus Cytarabine and Daunorubicin in Patients 60-75 Years of Age With Untreated High Risk (Secondary) AML
To confirm the efficacy of CPX-351 compared to 7+3 as first line therapy in elderly patients (60-75 yrs) with high risk (secondary) Acute Myeloid Leukemia. The primary efficacy endpoint will be overall survival.
- Conditions
- High Risk Acute Myeloid Leukemia
- Being tested
- CPX-351, 7+3 (cytarabine and daunorubicin)
- Keywords
- AML, Acute Myeloid Leukemia, high risk AML, secondary AML, AML in elderly
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CPX-351Drug
First induction: 100 units/m2 by 90-minute IV infusion on Days 1, 3, 5. Second induction: 100 units/m2 by 90-minute IV infusion on Days 1 and 3. Consolidation therapy: 65 units/m2 by 90-minute IV infusion on Days 1 and 3.
7+3 (cytarabine and daunorubicin)Drug
Also called cytarabine and daunorubicin
First induction: 7+3 was administered as: cytarabine at a dose of 100 mg/m2/day on Days 1 through 7 by continuous infusion, and daunorubicin at a dose of 60 mg/m2/day on Days 1, 2, and 3.
Second induction: 5+2 was administered as: cytarabine at a dose of 100 mg/m2/day on Days 1 through 5 by continuous infusion and daunorubicin at a dose of 60 mg/m2/day on Days 1 and 2.
Consolidation therapy: 5+2 was administered as: cytarabine at a dose of 100 mg/m2/day on Days 1 through 5 by continuous infusion, and daunorubicin at a dose of 60 mg/m2/day on Days 1 and 2.
| Study group | Role | What they receive |
|---|---|---|
| Arm A (CPX-351) | Experimental | Subjects are eligible to receive up to 2 inductions and up to 2 consolidations with CPX-351. The number of inductions and consolidations a subject received depended on response. Receives: Drug: CPX-351 |
| Arm B (7+3) | Active comparator | Subjects are eligible to receive up to 2 inductions and up to 2 consolidations with cytarabine and daunorubicin given as a 7 + 3, or 5 days of continuous infusion of cytarabine and 2 days of daunorubicin (5+2, second induction, consolidation courses) therapy. The number of inductions and consolidations a subject received depended on response. Receives: Drug: 7+3 (cytarabine and daunorubicin) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Survival
Overall survival was measured from the date of randomization to death from any cause, subjects not known to have died by the last follow-up were censored on the date they were last known to be alive.
Measured: From the date of randomization to death from any cause
Secondary outcomes (5)
Proportion of Subjects With a Response
Complete Remission (CR)
Measured: Post Induction
Event-free Survival
All randomized subjects were assessed for event-free survival (EFS). EFS was defined as the time from study randomization to the date of induction treatment failure (persistent disease), relapse from CR or CRi or death from any cause, whichever came first. Subjects alive and not known to have any of these events were censored on thee date they were last examined on study.
Measured: From the date of randomization to the date that persistent disease was documented or the date of relapse after CR or death, whichever came first
Remission Duration
Only subjects achieving CR or CRi were assessed for remission duration.
Measured: From the date of achievement of a remission until the date of relapse or death from any cause
Rate of Achieving Morphologic Leukemia-free State
All randomized subjects with at least 1 evaluable postrandomization bone marrow assessment performed on or after Day 14 after the last induction were assessed for MLFS.
Measured: Day 14
Proportion of Subjects Receiving a Stem Cell Transplant
The number and percentage of subjects transferred for HSCT after induction treatment was recorded.
Measured: Post Induction
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2012 (estimated)
- Study start13 Dec 2012
- Primary completion31 Dec 2015
- Study completion31 Dec 2015
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.
- Publications (9)Papers the sponsor has linked to this study.
- Other trials for High Risk Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by Jazz PharmaceuticalsThe 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 309 participants (actual)
Sponsor and investigators
- Lead sponsor
- Jazz Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- The Leukemia and Lymphoma Society
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01696084
- Sponsor's protocol ID
- CLTR0310-301
- Acronym
- 301
- Status last verified
- Jul 2020
- First submitted
- 26 Sep 2012
- Last updated
- 10 Aug 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Shimony S, Murdock HM, Keating J, Tsai HK, Sasi A, Gibson CJ, Faderl S, Wagner A, Dronamraju N, Lin TL, Prebet T, Cortes JE, Uy GL, Lancet JE, Reilly CR, Neuberg D, Stone RM, Lindsley RC. CPX-351 selectively benefits patients with AML and myelodysplasia-related mutations in the pivotal randomized trial. Blood Adv. 2026 Apr 28;10(8):2854-2864. doi: 10.1182/bloodadvances.2025019378.
PubMed 41628350Guolo F, Fianchi L, Martelli MP, Lussana F, Grimaldi F, Pilo F, Rondoni M, Fili C, Minetto P, Capelli D, Chiusolo P, Breccia M, Mastaglio S, Bernardi M, Bocchia M, Fumagalli M, Galimberti S, Mancini V, Piccioni AL, Maurillo L, Fracchiolla NS, Palmieri R, Vetro C, Papayannidis C, Brunetti L, Sperotto A, Gigli F, Zappasodi P, Mule A, Patti C, Borlenghi E, Dargenio M, Lessi F, Cerrano M, Cilloni D, Isidori A, Lunghi M, Alati C, Gurrieri C, Riva C, Marconi G, Lotesoriere I, Gatani S, Scattolin AM, Caizzi M, Perrone S, Billio A, Gherlinzoni F, Mannelli F, Gottardi M, Cairoli R, Candoni A, Ferrara F, Pagano L, Lemoli RM, Venditti A, Todisco E. Real World Study on the Best CPX-351 Treatment Duration and Timing for Allogeneic Stem Cell Transplantation. Am J Hematol. 2025 Dec;100(12):2293-2304. doi: 10.1002/ajh.70083. Epub 2025 Sep 24.
PubMed 40990091Cortes JE, Lin TL, Asubonteng K, Faderl S, Lancet JE, Prebet T. Efficacy and safety of CPX-351 versus 7 + 3 chemotherapy by European LeukemiaNet 2017 risk subgroups in older adults with newly diagnosed, high-risk/secondary AML: post hoc analysis of a randomized, phase 3 trial. J Hematol Oncol. 2022 Oct 26;15(1):155. doi: 10.1186/s13045-022-01361-w.
PubMed 36289532Cortes JE, Lin TL, Uy GL, Ryan RJ, Faderl S, Lancet JE. Quality-adjusted Time Without Symptoms of disease or Toxicity (Q-TWiST) analysis of CPX-351 versus 7 + 3 in older adults with newly diagnosed high-risk/secondary AML. J Hematol Oncol. 2021 Jul 13;14(1):110. doi: 10.1186/s13045-021-01119-w.
PubMed 34256819Lancet JE, Uy GL, Newell LF, Lin TL, Ritchie EK, Stuart RK, Strickland SA, Hogge D, Solomon SR, Bixby DL, Kolitz JE, Schiller GJ, Wieduwilt MJ, Ryan DH, Faderl S, Cortes JE. CPX-351 versus 7+3 cytarabine and daunorubicin chemotherapy in older adults with newly diagnosed high-risk or secondary acute myeloid leukaemia: 5-year results of a randomised, open-label, multicentre, phase 3 trial. Lancet Haematol. 2021 Jul;8(7):e481-e491. doi: 10.1016/S2352-3026(21)00134-4.
PubMed 34171279Lin TL, Rizzieri DA, Ryan DH, Schiller GJ, Kolitz JE, Uy GL, Hogge DE, Solomon SR, Wieduwilt MJ, Ryan RJ, Faderl S, Cortes JE, Lancet JE. Older adults with newly diagnosed high-risk/secondary AML who achieved remission with CPX-351: phase 3 post hoc analyses. Blood Adv. 2021 Mar 23;5(6):1719-1728. doi: 10.1182/bloodadvances.2020003510.
PubMed 33724305Kolitz JE, Strickland SA, Cortes JE, Hogge D, Lancet JE, Goldberg SL, Villa KF, Ryan RJ, Chiarella M, Louie AC, Ritchie EK, Stuart RK. Consolidation outcomes in CPX-351 versus cytarabine/daunorubicin-treated older patients with high-risk/secondary acute myeloid leukemia. Leuk Lymphoma. 2020 Mar;61(3):631-640. doi: 10.1080/10428194.2019.1688320. Epub 2019 Nov 25.
PubMed 31760835Lancet JE, Uy GL, Cortes JE, Newell LF, Lin TL, Ritchie EK, Stuart RK, Strickland SA, Hogge D, Solomon SR, Stone RM, Bixby DL, Kolitz JE, Schiller GJ, Wieduwilt MJ, Ryan DH, Hoering A, Banerjee K, Chiarella M, Louie AC, Medeiros BC. CPX-351 (cytarabine and daunorubicin) Liposome for Injection Versus Conventional Cytarabine Plus Daunorubicin in Older Patients With Newly Diagnosed Secondary Acute Myeloid Leukemia. J Clin Oncol. 2018 Sep 10;36(26):2684-2692. doi: 10.1200/JCO.2017.77.6112. Epub 2018 Jul 19.
PubMed 30024784Stein EM, Tallman MS. Emerging therapeutic drugs for AML. Blood. 2016 Jan 7;127(1):71-8. doi: 10.1182/blood-2015-07-604538. Epub 2015 Dec 10.
PubMed 26660428
Study at a glance
From the registry record, last updated by the sponsor on 10 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase III, Multicenter, Randomized, Trial of CPX-351 (Cytarabine:Daunorubicin) Liposome Injection Versus Cytarabine and Daunorubicin in Patients 60-75 Years of Age With Untreated High Risk (Secondary) AML
- ClinicalTrials.gov ID
- NCT01696084
- Acronym / sponsor ID
- 301 / CLTR0310-301
- Conditions
- High Risk Acute Myeloid Leukemia
- Keywords
- AML, Acute Myeloid Leukemia, high risk AML, secondary AML, AML in elderly
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 309 participants (actual)
- Lead sponsor
- Jazz PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- The Leukemia and Lymphoma Society
- Study start
- 13 Dec 2012 (actual)
- Primary completion
- 31 Dec 2015 (actual)
- Study completion
- 31 Dec 2015 (actual)
- First posted
- 28 Sep 2012 (estimated)
- Results
- Posted 2 Oct 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To confirm the efficacy of CPX-351 compared to 7+3 as first line therapy in elderly patients (60-75 yrs) with high risk (secondary) Acute Myeloid Leukemia. The primary efficacy endpoint will be overall survival.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm A (CPX-351) | Experimental | Drug: CPX-351Subjects are eligible to receive up to 2 inductions and up to 2 consolidations with CPX-351. The number of inductions and consolidations a subject received depended on response. |
| Arm B (7+3) | Active comparator | Drug: 7+3 (cytarabine and daunorubicin)Subjects are eligible to receive up to 2 inductions and up to 2 consolidations with cytarabine and daunorubicin given as a 7 + 3, or 5 days of continuous infusion of cytarabine and 2 days of daunorubicin (5+2, second induction, consolidation courses) therapy. The number of inductions and consolidations a subject received depended on response. |
Who may qualify
- Ages
- 60 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- Ability to understand and voluntarily give informed consent
- Age 60-75 years at the time of diagnosis of AML
- Pathological diagnosis of AML according to WHO criteria (with at least 20% blasts in the peripheral blood or bone marrow)
- Confirmation of:
- Eastern Cooperative Oncology Group (ECOG) performance status 0-2
Key exclusion criteria (14 listed)
- Except for CMMoL, patients with history of myeloproliferative neoplasms (MPN) (defined as a history of essential thrombocytosis or polycythemia vera, or idiopathic myelofibrosis prior to the diagnosis of AML) or combined MDS/MPN are not eligible.
- Acute promyelocytic leukemia [t(15;17)] or favorable cytogenetics, including t(8;21) or inv16 if known at the time of randomization.
- Clinical evidence of active CNS leukemia
- Patients with active (uncontrolled, metastatic) second malignancies are excluded.
- Prior treatment intended for induction therapy of AML; only hydroxyurea is permitted for control of blood counts. For example, a patient with MDS that changes HMA dose and schedule after the diagnosis of AML is excluded. AML-type therapy, such as cytarabine alone (>1g/m2/day) or cytarabine plus an anthracycline as well as prior HSCT are also excluded.
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-351First induction: 100 units/m2 by 90-minute IV infusion on Days 1, 3, 5. Second induction: 100 units/m2 by 90-minute IV infusion on Days 1 and 3. Consolidation therapy: 65 units/m2 by 90-minute IV infusion on Days 1 and 3. | Drug | Arm A (CPX-351) |
| 7+3 (cytarabine and daunorubicin)Also: cytarabine and daunorubicinFirst induction: 7+3 was administered as: cytarabine at a dose of 100 mg/m2/day on Days 1 through 7 by continuous infusion, and daunorubicin at a dose of 60 mg/m2/day on Days 1, 2, and 3. Second induction: 5+2 was administered as: cytarabine at a dose of 100 mg/m2/day on Days 1 through 5 by continuous infusion and daunorubicin at a dose of 60 mg/m2/day on Days 1 and 2. Consolidation therapy: 5+2 was administered as: cytarabine at a dose of 100 mg/m2/day on Days 1 through 5 by continuous infusion, and daunorubicin at a dose of 60 mg/m2/day on Days 1 and 2. | Drug | Arm B (7+3) |
Primary outcome
Overall Survival
Time frame: From the date of randomization to death from any cause
Overall survival was measured from the date of randomization to death from any cause, subjects not known to have died by the last follow-up were censored on the date they were last known to be alive.
5 secondary outcomes
Proportion of Subjects With a Response
Time frame: Post Induction
Event-free Survival
Time frame: From the date of randomization to the date that persistent disease was documented or the date of relapse after CR or death, whichever came first
Remission Duration
Time frame: From the date of achievement of a remission until the date of relapse or death from any cause
Rate of Achieving Morphologic Leukemia-free State
Time frame: Day 14
Proportion of Subjects Receiving a Stem Cell Transplant
Time frame: Post Induction
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Aug 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41628350Shimony S, Murdock HM, Keating J, Tsai HK, Sasi A, Gibson CJ, Faderl S, Wagner A, Dronamraju N, Lin TL, Prebet T, Cortes JE, Uy GL, Lancet JE, Reilly CR, Neuberg D, Stone RM, Lindsley RC. CPX-351 selectively benefits patients with AML and myelodysplasia-related mutations in the pivotal randomized trial. Blood Adv. 2026 Apr 28;10(8):2854-2864. doi: 10.1182/bloodadvances.2025019378.
- Derived from this study: PubMed 40990091Guolo F, Fianchi L, Martelli MP, Lussana F, Grimaldi F, Pilo F, Rondoni M, Fili C, Minetto P, Capelli D, Chiusolo P, Breccia M, Mastaglio S, Bernardi M, Bocchia M, Fumagalli M, Galimberti S, Mancini V, Piccioni AL, Maurillo L, Fracchiolla NS, Palmieri R, Vetro C, Papayannidis C, Brunetti L, Sperotto A, Gigli F, Zappasodi P, Mule A, Patti C, Borlenghi E, Dargenio M, Lessi F, Cerrano M, Cilloni D, Isidori A, Lunghi M, Alati C, Gurrieri C, Riva C, Marconi G, Lotesoriere I, Gatani S, Scattolin AM, Caizzi M, Perrone S, Billio A, Gherlinzoni F, Mannelli F, Gottardi M, Cairoli R, Candoni A, Ferrara F, Pagano L, Lemoli RM, Venditti A, Todisco E. Real World Study on the Best CPX-351 Treatment Duration and Timing for Allogeneic Stem Cell Transplantation. Am J Hematol. 2025 Dec;100(12):2293-2304. doi: 10.1002/ajh.70083. Epub 2025 Sep 24.
- Derived from this study: PubMed 36289532Cortes JE, Lin TL, Asubonteng K, Faderl S, Lancet JE, Prebet T. Efficacy and safety of CPX-351 versus 7 + 3 chemotherapy by European LeukemiaNet 2017 risk subgroups in older adults with newly diagnosed, high-risk/secondary AML: post hoc analysis of a randomized, phase 3 trial. J Hematol Oncol. 2022 Oct 26;15(1):155. doi: 10.1186/s13045-022-01361-w.
- Derived from this study: PubMed 34256819Cortes JE, Lin TL, Uy GL, Ryan RJ, Faderl S, Lancet JE. Quality-adjusted Time Without Symptoms of disease or Toxicity (Q-TWiST) analysis of CPX-351 versus 7 + 3 in older adults with newly diagnosed high-risk/secondary AML. J Hematol Oncol. 2021 Jul 13;14(1):110. doi: 10.1186/s13045-021-01119-w.
- Derived from this study: PubMed 34171279Lancet JE, Uy GL, Newell LF, Lin TL, Ritchie EK, Stuart RK, Strickland SA, Hogge D, Solomon SR, Bixby DL, Kolitz JE, Schiller GJ, Wieduwilt MJ, Ryan DH, Faderl S, Cortes JE. CPX-351 versus 7+3 cytarabine and daunorubicin chemotherapy in older adults with newly diagnosed high-risk or secondary acute myeloid leukaemia: 5-year results of a randomised, open-label, multicentre, phase 3 trial. Lancet Haematol. 2021 Jul;8(7):e481-e491. doi: 10.1016/S2352-3026(21)00134-4.
- Derived from this study: PubMed 33724305Lin TL, Rizzieri DA, Ryan DH, Schiller GJ, Kolitz JE, Uy GL, Hogge DE, Solomon SR, Wieduwilt MJ, Ryan RJ, Faderl S, Cortes JE, Lancet JE. Older adults with newly diagnosed high-risk/secondary AML who achieved remission with CPX-351: phase 3 post hoc analyses. Blood Adv. 2021 Mar 23;5(6):1719-1728. doi: 10.1182/bloodadvances.2020003510.
- Derived from this study: PubMed 31760835Kolitz JE, Strickland SA, Cortes JE, Hogge D, Lancet JE, Goldberg SL, Villa KF, Ryan RJ, Chiarella M, Louie AC, Ritchie EK, Stuart RK. Consolidation outcomes in CPX-351 versus cytarabine/daunorubicin-treated older patients with high-risk/secondary acute myeloid leukemia. Leuk Lymphoma. 2020 Mar;61(3):631-640. doi: 10.1080/10428194.2019.1688320. Epub 2019 Nov 25.
- Derived from this study: PubMed 30024784Lancet JE, Uy GL, Cortes JE, Newell LF, Lin TL, Ritchie EK, Stuart RK, Strickland SA, Hogge D, Solomon SR, Stone RM, Bixby DL, Kolitz JE, Schiller GJ, Wieduwilt MJ, Ryan DH, Hoering A, Banerjee K, Chiarella M, Louie AC, Medeiros BC. CPX-351 (cytarabine and daunorubicin) Liposome for Injection Versus Conventional Cytarabine Plus Daunorubicin in Older Patients With Newly Diagnosed Secondary Acute Myeloid Leukemia. J Clin Oncol. 2018 Sep 10;36(26):2684-2692. doi: 10.1200/JCO.2017.77.6112. Epub 2018 Jul 19.
- Derived from this study: PubMed 26660428Stein EM, Tallman MS. Emerging therapeutic drugs for AML. Blood. 2016 Jan 7;127(1):71-8. doi: 10.1182/blood-2015-07-604538. Epub 2015 Dec 10.
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Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
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Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
RecruitingPhase 1/2
Venetoclax and CLAG-M for the Treatment of Acute Myeloid Leukemia and High-Grade Myeloid Neoplasms
Acute Biphenotypic Leukemia, Acute Myeloid Leukemia
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome
RecruitingPhase 1
First-in-human (FIH) Trial of GEN3018 in Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML) or Higher-risk Myelodysplastic Syndrome (HR-MDS)
R/R AML, R/R HR-MDS
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.
The record lists no contact. Use the official record to reach the sponsor.
43 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing CPX-351 and 7+3 (cytarabine and daunorubicin) in people with High Risk Acute Myeloid Leukemia, run by Jazz Pharmaceuticals at 43 sites in 2 countries.
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 16 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: finished, as of the sponsor's last update on 10 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 43 sites in United States, Canada. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 2 Oct 2017). 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?
Overall Survival (time frame: From the date of randomization to death from any cause). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01696084, which also lists 9 linked publications.
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 10 Aug 2020.