Liposome-encapsulated Daunorubicin-Cytarabine and Venetoclax in Treating Participants With Relapsed, Refractory or Untreated Acute Myeloid Leukemia
Phase II Study of CPX-351 in Combination With Venetoclax in Patients With Acute Myeloid Leukemia (AML)
2 other identifiers
interventional
52
1 country
1
Brief Summary
This phase II trial studies how well liposome-encapsulated daunorubicin-cytarabine and venetoclax work in treating participants with acute myeloid leukemia that has come back (relapsed), does not respond to treatment (refractory), or has not been treated (untreated). Drugs used in chemotherapy, such as liposome-encapsulated daunorubicin-cytarabine and venetoclax, 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Oct 2018
Longer than P75 for phase_2
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 9, 2018
CompletedFirst Posted
Study publicly available on registry
August 14, 2018
CompletedStudy Start
First participant enrolled
October 29, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 31, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 31, 2026
May 20, 2026
May 1, 2026
8.2 years
August 9, 2018
May 18, 2026
Conditions
Outcome Measures
Primary Outcomes (2)
Achievement of composite complete remission
Defined as complete remission/complete remission without blood count recovery/complete remission without platelet recovery. Will be monitored simultaneously using the Bayesian approach of Thall, Simon, Estey. Will be estimated along with the 95% credible interval.
Up to 3 cycles (84 days)
Incidence of adverse events
Will be monitored simultaneously using the Bayesian approach of Thall, Simon, Estey. Will be summarized using descriptive statistics such as mean, standard deviation, median and range. Safety data will be summarized by category, severity and frequency.
Up to 28 days
Secondary Outcomes (3)
Event-free survival
Number of days from the date of treatment initiation up to 1 year
Overall survival
Time from treatment start till death or last follow-up, assessed up to 1 year
Biomarker changes
Baseline up to 1 year
Study Arms (1)
Treatment (CPX-351, venetoclax)
EXPERIMENTALINDUCTION: Participants receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5 of cycle 1 and on days 1 and 3 of cycle 2. Participants also receive venetoclax PO QD on days 2-21. Treatment repeats every 28 days for up to 2 cycles in the absence of disease progression or unacceptable toxicity. CONSOLIDATION: Participants receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3 and venetoclax PO QD on days 2-21. Treatment repeats every 28 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.
Interventions
Given IV
Given PO
Eligibility Criteria
You may qualify if:
- For the lead in phase: Patients \>= 18 years of age with a diagnosis of relapsed and/or refractory AML will be eligible. Patients who have had prior treatment with venetoclax will be allowed to participate in the lead in phase and cohort A
- For the dose expansion cohort A (relapsed/refractory \[R/R\] AML): Patients \>= 18 years of age with a diagnosis of relapsed and/or refractory AML will be eligible
- For the dose expansion cohort B (de novo AML): Patients \>= 18 years to 69 years of age; patients in this cohort must have received no prior therapy for AML
- Prior therapy with hydroxyurea, hematopoietic growth factors, or tretinoin (ATRA) (for emergency use for stabilization) is allowed with no washout. A cumulative dose of ara-C of up to 3 g for emergency stabilization in patients with rapidly proliferating disease is also allowed provided it was administered \> 48 hrs prior to enrollment
- Bilirubin =\< 2 mg/dL
- Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) =\< 3 x upper limit of normal (ULN) or \< 5 x ULN if related to leukemic involvement
- Creatinine =\< 1.5 x ULN
- Known cardiac ejection fraction of \> or = 45% within the past 3 months
- Eastern Cooperative Oncology Group (ECOG) performance status of =\< 2
- A negative urine or serum pregnancy test is required within 1 week for all women of childbearing potential prior to enrolling on this trial. A woman of childbearing potential is defined as a woman who has not been naturally postmenopausal for at least 12 consecutive months, or who had no previous surgical sterilization
- Patient must have the ability to understand the requirements of the study and signed informed consent. A signed informed consent by the patient or his legally authorized representative is required prior to their enrollment on the protocol
You may not qualify if:
- Pregnant women are excluded from this study because the agents used in this study have the potential for teratogenic or abortifacient effects. Because there is a potential risk for adverse events in nursing infants secondary to treatment of the mother with the chemotherapy agents, breastfeeding should also be avoided
- Uncontrolled intercurrent illness including, but not limited to active uncontrolled infection, symptomatic congestive heart failure (New York Heart Association \[NYHA\] class III or IV), unstable angina pectoris, clinically significant cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
- Patient with documented hypersensitivity to any of the components of the chemotherapy program
- Patients with acute promyelocytic leukemia (M3) or core-binding factor AML
- Patients with active central nervous system (CNS) leukemia are excluded since the antileukemia activity of the treatment components against CNS leukemia are not known
- Men and women of childbearing potential who do not practice contraception. Women of childbearing potential and men must agree to use contraception prior to study entry and for the duration of study participation
- Prior treatment with CPX-351 or venetoclax. Patients with prior treatment with venetoclax will be allowed in patients with relapsed/refractory (R/R) disease including those in the lead-in phase as well as those in cohort A
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
M D Anderson Cancer Center
Houston, Texas, 77030, United States
Related Publications (1)
Kadia TM, Jen WY, Bataller A, Bazinet A, Borthakur G, Jabbour E, Qiao W, Short NJ, Takahashi K, Issa GC, DiNardo CD, Montalban-Bravo G, Pemmaraju N, Tran A, Bharathi V, Loghavi S, Alousi AM, Popat U, Daver NG, Ravandi F, Kantarjian HM. A Phase 2 Trial of CPX-351 Combined With Venetoclax in Relapsed or Refractory Acute Myeloid Leukemia. Am J Hematol. 2025 Aug;100(8):1365-1373. doi: 10.1002/ajh.27723. Epub 2025 May 22.
PMID: 40401707DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Tapan M Kadia
M.D. Anderson Cancer Center
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 9, 2018
First Posted
August 14, 2018
Study Start
October 29, 2018
Primary Completion (Estimated)
December 31, 2026
Study Completion (Estimated)
December 31, 2026
Last Updated
May 20, 2026
Record last verified: 2026-05