VAM (liposomal mitoxantrone) for secondary AML and myeloid sarcoma

Prospective, Multicenter, Single-Arm Clinical Study of Mitoxantrone Liposome Combined With Azacitidine and Venetoclax in the Treatment of Secondary AML, AML With Extramedullary Involvement, and Myeloid Sarcoma

This trial will test whether VAM (a liposomal form of mitoxantrone) is effective and safe for adults with secondary AML, therapy-related AML, AML with extramedullary disease, or myeloid sarcoma.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 48 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Ruijin Hospital (Academic / other)
• Locations: 1 site (Shanghai)
• Trial ID: NCT07028086 on ClinicalTrials.gov

What this trial studies

This is a phase 2, interventional trial of a liposomal mitoxantrone formulation designed to enhance tissue penetration and reduce circulating free drug, with the goal of lowering cardiotoxicity compared with conventional mitoxantrone. The study enrolls adults with newly diagnosed, previously untreated non-APL AML who have secondary or therapy-related disease, extramedullary involvement, or myeloid sarcoma, and who meet cardiac and organ-function criteria. Participants receive VAM as the investigational chemotherapy regimen and are followed for treatment response, adverse events, and cardiac safety. The trial is conducted at Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, with structured clinical and laboratory monitoring throughout therapy.

Who should consider this trial

Good fit: Adults aged 18–65 with newly diagnosed, previously untreated non-APL AML who have secondary or therapy-related AML, AML with extramedullary involvement, or myeloid sarcoma, with ECOG 0–2 and adequate cardiac, liver, and kidney function are the intended participants.

Not a fit: Patients who are frail/unfit for intensive therapy, have LVEF <50%, significant organ dysfunction, prior treated AML, or other active malignancies requiring treatment are unlikely to benefit or be eligible for this trial.

Why it matters

Potential benefit: If successful, VAM could provide a chemotherapy option with better tissue delivery and lower risk of heart damage for these high-risk AML subtypes.

How similar studies have performed: Liposomal formulations of anthracycline-like drugs have shown reduced cardiotoxicity in other settings, but liposomal mitoxantrone in AML is relatively novel and has limited published clinical data.

Eligibility criteria

Inclusion Criteria:

1. The patient fully understands this study, voluntarily participates, and signs the informed consent form (ICF)
2. Age 18-65 years
3. Clinically diagnosed, previously untreated acute myeloid leukemia (non-APL), meeting any one of the following criteria: a. Secondary acute myeloid leukemia; b. Therapy-related acute myeloid leukemia; c. AML with extramedullary/myeloid sarcoma; d. Age ≥60 years, assessed as fit-AML;
4. Normal cardiac function, with left ventricular ejection fraction (LVEF) ≥50%
5. Liver and kidney function: Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤2.5 times the upper limit of normal (ULN) (≤5 times ULN for patients with liver involvement); total bilirubin ≤1.5 times ULN; serum creatinine ≤1.5 times ULN
6. Eastern Cooperative Oncology Group (ECOG) performance status score: 0-2

Exclusion Criteria:

1. Assessed as unfit- or frail-AML;
2. Patients with a history or concurrent diagnosis of other malignancies requiring treatment
3. Uncontrolled systemic diseases (e.g., advanced active infections, uncontrolled hypertension, etc.)
4. Known history of immediate or delayed hypersensitivity reactions to the same class of study drugs or excipients
5. Pregnant or breastfeeding women, or patients who refuse to use effective contraception during the study period
6. Patients with a history of severe neurological or psychiatric disorders
7. Other severe medical conditions, such as myocardial infarction, severe or unstable angina, severe arrhythmias
8. Cerebrovascular events (including transient ischemic attacks), etc.
9. Known infection with human immunodeficiency virus (HIV); active hepatitis B or C infection; inactive hepatitis carriers or subjects with low viral titers after receiving non-prohibited antiviral therapy are not excluded
10. Subjects who have received strong or moderate CYP3A inducers/inhibitors or strong P-gp inhibitors or related foods within 7 days before starting the study treatment
11. Patients unable to take oral medications or with malabsorption syndrome
12. Patients deemed by the investigator to be unsuitable for participation in the study

Where this trial is running

Shanghai

• Ruijin Hospital, Shanghai Jiaotong University School of Medicine — Shanghai, China (Recruiting)

Conditions Acute Myeloid Leukemia; Myeloid Sarcoma; Secondary AML; AML with Extramedullary Involvement