52 Participants Needed

CPX-351 + Quizartinib for Acute Myeloid Leukemia

MY
Overseen ByMusa Yilmaz
Age: 18+
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: M.D. Anderson Cancer Center
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

Trial Summary

Will I have to stop taking my current medications?

The trial protocol does not specify if you must stop taking your current medications. However, you cannot take certain drugs that affect heart rhythm or strong CYP3A4 inducers. It's best to discuss your current medications with the trial team.

What data supports the effectiveness of the drug CPX-351 + Quizartinib for Acute Myeloid Leukemia?

Research shows that CPX-351, a combination of two drugs in a special delivery system, significantly improved survival in older adults with a specific type of acute myeloid leukemia compared to traditional chemotherapy. This suggests it could be an effective option for treating this condition.12345

Is CPX-351 safe for humans?

CPX-351, also known as Vyxeos, has been shown to have a safety profile similar to traditional chemotherapy in studies with older adults with high-risk acute myeloid leukemia. It is generally considered to have an acceptable tolerability profile, meaning it is reasonably safe for use in humans.12567

What makes the drug CPX-351 + Quizartinib unique for treating acute myeloid leukemia?

CPX-351 is unique because it combines two chemotherapy drugs, daunorubicin and cytarabine, in a special liposome (a tiny bubble-like structure) that helps deliver the drugs more effectively to cancer cells. This combination has shown improved survival rates compared to the traditional 7+3 chemotherapy regimen, especially in older adults with high-risk acute myeloid leukemia.568910

What is the purpose of this trial?

This phase I/II trial studies the side effects and best dose of CPX-351 in combination with quizartinib for the treatment of acute myeloid leukemia and high risk myelodysplastic syndrome. CPX-351, composed of chemotherapy drugs daunorubicin and cytarabine, works 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. Quizartinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. The goal of this study is to learn if the combination of CPX-351 and quizartinib can help to control acute myeloid leukemia and myelodysplastic syndrome.

Research Team

MY

Musa Yilmaz

Principal Investigator

M.D. Anderson Cancer Center

Eligibility Criteria

This trial is for adults with Acute Myeloid Leukemia (AML) or high-risk Myelodysplastic Syndrome (MDS), who have either not received chemotherapy or have had up to four prior treatments. Participants must be over 60 years old for the frontline cohort, and at least 18 for the relapsed/refractory cohort. They should have adequate organ function, no severe concurrent illnesses, and women of childbearing potential must use contraception.

Inclusion Criteria

I can take care of myself but might not be able to do heavy physical work.
I have been diagnosed with AML or high-risk MDS, not including APL.
I have AML or MDS and have had 1-4 treatments for it.
See 15 more

Exclusion Criteria

Sustained heart rate of < 50/minute on pre-entry ECG
I do not have major stomach or intestine problems that affect medication absorption.
Atrial fibrillation documented within 2 weeks prior to first dose of study drug
See 19 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Induction

Patients receive CPX-351 IV on days 1, 3, and 5 and quizartinib PO on days 6-19. Treatment repeats every 28 days for up to 2 cycles.

Up to 8 weeks
Multiple visits for IV administration and monitoring

Consolidation

Patients receive CPX-351 IV on days 1 and 3 and quizartinib PO on days 4-28. Treatment repeats every 28 days for 2 cycles.

8 weeks
Multiple visits for IV administration and monitoring

Maintenance

Patients receive quizartinib PO on days 1-28 in the absence of disease progression or unacceptable toxicity.

Variable, based on patient response

Follow-up

Participants are monitored for safety and effectiveness after treatment completion.

Up to 5 years
Follow-up at 30 days, then every 3-6 months

Treatment Details

Interventions

  • Liposome-encapsulated Daunorubicin-Cytarabine
  • Quizartinib
Trial Overview The trial is testing CPX-351 (a combination of daunorubicin and cytarabine encapsulated in liposomes) alongside quizartinib to see if they can effectively control AML and MDS. It's a phase I/II study that aims to determine the best dose while monitoring side effects when these drugs are used together.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: Treatment (CPX-351, quizartinib)Experimental Treatment2 Interventions
INDUCTION: Patients receive CPX-351 IV over 90 minutes on days 1, 3 and 5 and quizartinib PO on days 6-19. Patients who do not respond to treatment during cycle 1 receive CPX-351 IV on days 1 and 3 and quixartinib PO on days 6-19 during cycle 2. Treatment repeats every 28 days for up 2 cycles in the absence of disease progression or unacceptable toxicity. CONSOLIDATION: Patients receive CPX-351 over 90 minutes on days 1 and 3 and quizartinib PO on days 4-28 of cycle 1. Treatment with CPX-351 repeats every 28 days for 2 cycles in the absence of disease progression or unacceptable toxicity. MAINTENANCE: Patients receive quizartinib PO on days 1-28 in the absence of disease progression or unacceptable toxicity.

Liposome-encapsulated Daunorubicin-Cytarabine is already approved in United States for the following indications:

🇺🇸
Approved in United States as Vyxeos for:
  • Acute Myeloid Leukemia with myelodysplasia-related changes (AML-MRC)
  • Therapy-related AML (t-AML)

Find a Clinic Near You

Who Is Running the Clinical Trial?

M.D. Anderson Cancer Center

Lead Sponsor

Trials
3,107
Recruited
1,813,000+

National Cancer Institute (NCI)

Collaborator

Trials
14,080
Recruited
41,180,000+

Findings from Research

VYXEOS™, a liposomal formulation of daunorubicin and cytarabine, has been shown to significantly improve overall survival and event-free survival in older patients with newly diagnosed high-risk acute myeloid leukaemia (AML) compared to conventional chemotherapy (7+3).
The treatment also resulted in higher rates of complete remission and was well-tolerated, with a safety profile similar to that of the standard chemotherapy regimen, making it a valuable option for patients with therapy-related AML or AML with myelodysplasia-related changes.
Daunorubicin/Cytarabine Liposome: A Review in Acute Myeloid Leukaemia.Blair, HA.[2020]
CPX-351 (Vyxeos®) is a dual-drug liposomal formulation of cytarabine and daunorubicin designed to improve efficacy in treating acute myeloid leukemia (AML) by maintaining a synergistic 5:1 drug ratio, which enhances its effectiveness compared to traditional chemotherapy regimens.
The liposomal encapsulation allows for controlled release and preferential uptake by malignant cells, which helps to protect the drugs from metabolism and reduces exposure to off-target tissues, contributing to a favorable safety profile while effectively targeting leukemia cells.
CPX-351: a nanoscale liposomal co-formulation of daunorubicin and cytarabine with unique biodistribution and tumor cell uptake properties.Mayer, LD., Tardi, P., Louie, AC.[2020]
CPX-351, a liposomal formulation of cytarabine and daunorubicin, showed high efficacy in treating childhood acute lymphoblastic leukemia (ALL) xenograft models, achieving complete responses in four B-lineage models and a partial response in one T-lineage model.
The drug was administered at a dose that resulted in plasma drug exposures similar to those seen in patients with acute myeloid leukemia (AML), indicating its potential effectiveness and safety for use in pediatric leukemia treatment.
Efficacy of CPX-351, (cytarabine:daunorubicin) liposome injection, against acute lymphoblastic leukemia (ALL) xenograft models of the Pediatric Preclinical Testing Program.Carol, H., Fan, MM., Harasym, TO., et al.[2021]

References

Daunorubicin/Cytarabine Liposome: A Review in Acute Myeloid Leukaemia. [2020]
CPX-351: a nanoscale liposomal co-formulation of daunorubicin and cytarabine with unique biodistribution and tumor cell uptake properties. [2020]
Efficacy of CPX-351, (cytarabine:daunorubicin) liposome injection, against acute lymphoblastic leukemia (ALL) xenograft models of the Pediatric Preclinical Testing Program. [2021]
Daunorubicin-cytarabine liposome (CPX-351) in the management of newly diagnosed secondary AML: A new twist on an old cocktail. [2020]
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. [2021]
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. [2023]
CPX-351 exhibits hENT-independent uptake and can be potentiated by fludarabine in leukaemic cells lines and primary refractory AML. [2022]
CPX-351 in FLT3-mutated acute myeloid leukemia. [2023]
Comparison of Hospital Length of Stay and Supportive Care Utilization Between Patients Treated with CPX-351 and 7+3 for Therapy-Related Acute Myeloid Leukemia or Acute Myeloid Leukemia with Myelodysplasia-Related Changes. [2022]
Real-world experience with CPX-351 in high-risk acute myeloid leukemia. [2023]
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