VYxeoS Liposomal Italian Observational Study iN the Real Practice

Study Overview
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Brief Summary
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The study is a prospective, single-arm, non-experimental, observational study in patients in Italy with Acute Myeloid Leukaemia (AML) with myelodysplastic-related changes or therapy related AML initiating treatment with JZP351 (Vyxeos liposomal) in their normal clinical practice.

Official Title
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VYxeoS Liposomal Italian Observational Study iN the Real Practice

Conditions
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Acute Myeloid Leukemia (AML), Acute Myeloid Leukemia With Myelodysplasia-Related Changes, Therapy-Related Acute Myeloid Leukemia

Intervention / Treatment
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* Drug: Vyxeos liposomal


Participation Criteria
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Eligibility Criteria
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Inclusion criteria: Patients newly diagnosed with AML-MRC (Acute myeloid leukemia with myelodysplasia-related changes) or t-related AML (Therapy related Acute myeloid leukemia) following 2016 World Health Organization (WHO) classification. Age ≥ 18 years old. Patients considered eligible for intensive chemotherapy in the opinion of the treating physician. Patients who will initiate the treatment with commercially JZP351 treatment after the Informed Consent Form (ICF) signature. The decision to prescribe JZP351 treatment must have been made prior and regardless of the enrollment of the patient in the study. Cardiac ejection fraction ≥ 50% by echocardiography or MUGA (Multi-Gated Acquisition). Exclusion criteria: Prior treatment intended for induction therapy of AML (Acute Myeloid Leukemia). Patients with prior cumulative anthracycline exposure of greater than 368 mg/m2 daunorubicin (or equivalent) Clinical evidence of active CNS (Central Nervous System) leukemia. Patients with active (uncontrolled, metastatic) second malignancies.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Acute Myeloid Leukemia (AML)<br>Participants with newly diagnosed AML-MRC (Acute myeloid leukemia with myelodysplasia-related changes) or t-related AML (Therapy related Acute myeloid leukemia) receiving Vyxeos liposomal as part of their standard of care treatment. | Drug: Vyxeos liposomal<br>* Standard of care JZP351 administered intravenously over 90 minutes.<br>* Other names: CPX-351;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of participants showing CR/CRi/CRh without MRD at the end of treatment | Percentage of participants showing CR (Complete Remission) / CRi (Complete Response with incomplete platelet or neutrophil recovery) /CRh (Complete Response with partial hematologic recovery) without MRD (measurable residual disease) at the end of treatment. MRD will be assessed via multiparameter flow cytometry. | Up to 24 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall Response Rate (ORR) after induction phase | ORR is the rate of participants achieving CR, CRi and CRh after induction phase | Up to 24 months | 
| Percentage of participants showing CR/CRi/CRh without MRD after induction phase | MRD will be assessed via multiparameter flow cytometry. | Up to 24 months | 
| Percentage of participants showing CR/CRi/CRh without MRD before transplantation | MRD will be assessed via multiparameter flow cytometry. | Up to 24 months | 
| Overall survival (OS) | Overall survival is date from the start of taking Vyxeos liposomal (JZP351) to the date of reported death due to any cause. | Up to 24 months | 
| Percentage of participants receiving an Hematopoietic stem-cell transplantation (HSCT) |  | Up to 24 months | 
| Landmark overall survival (OS) from the time of HSCT |  | Up to 24 months | 
| Percentage of participants with Adverse Events (AE) | Percentage of participants who experience Grades 1-5 and grade 3-5 adverse events, including AEs of special interest and serious AEs (SAEs), as evaluated by treating physician. | Up to 24 months | 
| Percentage of participants with a change in fitness status as assessed by Ferrara criteria before HSCT | Ferrara criteria is a list of assessments evaluating whether a participant is eligible for intensive chemotherapy or not. | Up to 24 months | 
| Percentage of participants with a change in fitness status as assessed by Ferrara criteria at the final treatment visit for patients not eligible for HSCT | Ferrara criteria is a list of assessments evaluating whether a participant is eligible for intensive chemotherapy or not. | Up to 24 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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JZP351, CPX-351, Vyxeos liposomal, AML, t-related AML, AML-MRC