Vyxeos for Acute Myeloid Leukemia

Recruiting at 1 trial location

Overseen By The Ohio State University Comprehensive Cancer Center

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Ohio State University Comprehensive Cancer Center

Must be taking: Cytarabine, Daunorubicin

Must not be taking: Investigational agents

Disqualifiers: Acute promyelocytic leukemia, TP53 mutations, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 2

Duration: 1 week

28 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new combination chemotherapy treatment called Vyxeos for individuals with acute myeloid leukemia (AML) who did not respond well to standard treatment. Vyxeos combines two chemotherapy drugs, cytarabine and daunorubicin, using a special delivery method that might reduce side effects and improve effectiveness. The trial aims to assess the treatment's effectiveness and potential side effects. It seeks participants with AML who have recently tried standard chemotherapy and still have active disease. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants a chance to be among the first to receive this potentially groundbreaking therapy.

Do I have to stop taking my current medications for the Vyxeos trial?

The trial protocol does not specify if you need to stop taking your current medications. However, if you were taking a targeted therapy for AML during initial treatment, you will not continue it during Vyxeos treatment.

Is there any evidence suggesting that Vyxeos is likely to be safe for humans?

Research has shown that Vyxeos, a combination of the chemotherapy drugs cytarabine and daunorubicin within a liposome, is generally safe and well-tolerated. Studies have found that its side effects are similar to those of standard chemotherapy, indicating it does not cause more or worse side effects than usual treatments.

The FDA has already approved Vyxeos for treating a specific type of acute myeloid leukemia (AML), confirming its reasonable safety in past research. Reports indicate that it helps patients live longer compared to traditional chemotherapy. While side effects like nausea or low blood counts can occur, they are manageable.

Overall, extensive studies support Vyxeos's safety, backed by real-world data. This should reassure those considering joining a trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for acute myeloid leukemia?

Unlike standard treatments for acute myeloid leukemia, which often involve separate administration of chemotherapy drugs like daunorubicin and cytarabine, Vyxeos combines these two drugs into a single liposome-encapsulated formulation. This unique delivery method allows for more targeted and potentially more effective delivery of the chemotherapy agents directly to cancer cells while minimizing exposure to healthy cells. Researchers are excited about Vyxeos because it has shown promise in improving treatment outcomes and potentially reducing side effects compared to conventional chemotherapy regimens.

What evidence suggests that Vyxeos might be an effective treatment for acute myeloid leukemia?

Research has shown that Vyxeos, a combination of cytarabine and daunorubicin encapsulated in a bubble similar to a cell membrane, is more effective for treating acute myeloid leukemia (AML). Studies have found that Vyxeos significantly increases overall survival compared to the standard 7+3 chemotherapy. Specifically, one study noted that patients on Vyxeos had more than double the five-year survival rate compared to those on standard treatment. Additionally, real-world evidence suggests Vyxeos is effective for both younger and older AML patients. This treatment is already FDA-approved for newly diagnosed therapy-related AML, highlighting its potential effectiveness. Participants in this trial will receive Vyxeos as the investigational treatment. ^{3 4 5 6 7}

Who Is on the Research Team?

Gregory K Behbehani, MD, PhD

Principal Investigator

Ohio State University Comprehensive Cancer Center

Are You a Good Fit for This Trial?

This trial is for adults with acute myeloid leukemia who didn't respond to initial chemo. They must be fit enough for intensive treatment, have proper heart function and organ health, and not exceed a lifetime dose of certain chemo drugs. Participants need to use effective contraception and cannot be pregnant or breastfeeding.

Inclusion Criteria

Aspartate aminotransferase (AST) < 5 x upper limit of normal (ULN) for the local laboratory

I can take care of myself and am up and about more than half of my waking hours.

My doctor thinks I won't fully recover without more treatment.

Exclusion Criteria

I am not pregnant, can use effective birth control for 6 months, and won't breastfeed for 14 days after my last Vyxeos dose.

Patients with psychological, familial, social, or geographic factors that otherwise preclude them from giving informed consent, following the protocol, or potentially hamper compliance with study treatment and follow-up

I do not have any severe heart conditions or recent heart attacks.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Patients receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3

1 week

2 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

60 days

Weekly visits

Long-term Follow-up

Participants are monitored monthly for progression-free survival and overall survival

Up to 2 years

Monthly visits

What Are the Treatments Tested in This Trial?

Interventions

• Liposome-encapsulated Daunorubicin-Cytarabine

Trial Overview The study tests Vyxeos, a liposome-encapsulated combo of chemotherapy drugs cytarabine and daunorubicin, in patients whose leukemia persisted after standard treatment. It aims to see if this approach has fewer side effects while being more effective.

How Is the Trial Designed?

Group I: Treatment (liposome-encapsulated daunorubicin-cytarabine) Experimental Treatment 1 Intervention

Within 14-33 days after the start of previous cycle of chemotherapy, patients receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3 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)

Who Is Running the Clinical Trial?

Ohio State University Comprehensive Cancer Center

Lead Sponsor

Trials

350

Recruited

295,000+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7485353/

EMA Review of Daunorubicin and Cytarabine ... Vyxeos has demonstrated a clinically significant improvement in overall survival compared with the standard of care 7+3 in the proposed population of patients ...

2. vyxeospro.com vyxeospro.com/clinical-data/efficacy

The only choice for more than double the 5-year OS vs 7+3 1 VYXEOS is the only FDA-approved treatment for sAML demonstrating superior overall survival a in a Phase 3 study.

3. ascopubs.org ascopubs.org/doi/10.1200/JCO.2017.77.6112

CPX-351 (cytarabine and daunorubicin) Liposome for ... CPX-351 treatment is associated with significantly longer survival compared with conventional 7+3 in older adults with newly diagnosed sAML.

4. sciencedirect.com sciencedirect.com/science/article/pii/S2152265023002124

Real-World Experience With CPX-351 Treatment for Acute ... This study provides real-world survival outcomes data suggesting that CPX-351 is an effective treatment for both younger (<60 years) and older (≥60 years) ...

5. ashpublications.org ashpublications.org/blood/article/142/Supplement%201/5946/500672/A-Phase-1b-2-Trial-of-Liposomal-Cytarabine-and

A Phase 1b/2 Trial of Liposomal Cytarabine and Daunorubicin ... Liposomal Daunorubicin and Cytarabine (CPX-351) has been approved by the FDA for treatment of newly-diagnosed therapy-related acute myeloid leukemia or AML.

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40348597/

The Clinical Safety and Efficacy of Cytarabine ... This review presents strong evidence supporting CPX-351 as a therapeutic alternative with superior efficacy and comparable safety to standard chemotherapy ...

7. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/30024784/

CPX-351 (cytarabine and daunorubicin) Liposome for ... Prior clinical studies demonstrated a sustained drug ratio and exposure in vivo and prolonged survival versus standard-of-care cytarabine plus ...