A Phase 3 interventional study of Mitoxantrone Hydrochloride Liposome and Cytarabine in Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS), sponsored by Ruijin Hospital. Not yet recruiting. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-07-29.
Sponsored by Ruijin Hospital · Phase 3, Interventional, and Treatment
This study aims to evaluate the efficacy and safety of mitoxantrone hydrochloride liposome, subcutaneous cytarabine and G-CSF combined with venetoclax (CMG+Ven) versus azacitidine combined with venetoclax (VA) in the treatment of adult myelodysplastic syndrome IB2 (MDS-IB2) and newly diagnosed secondary or elderly AML.
Patients with secondary AML (S-AML) and elderly AML have an extremely poor prognosis due to advanced age, multiple comorbidities, and unfavorable cytogenetic abnormalities. Traditional intensive chemotherapy is associated with low remission rates and substantial toxicity. Myelodysplastic syndrome with excess blasts-2 (MDS-IB2) carries a very high risk of transformation to AML, and its management is similar to that of AML. Although venetoclax, a BCL-2 targeted agent, combined with azacitidine (VA regimen) has revolutionized the treatment paradigm for AML patients unfit for intensive chemotherapy, the VA regimen provides insufficient depth of remission in patients eligible for chemotherapy and is difficult to administer at full dosage and full course, highlighting an urgent need for optimization.
Mitoxantrone hydrochloride liposome is an improved formulation of conventional mitoxantrone. Through liposomal encapsulation and polyethylene glycol modification, it exhibits a prolonged half-life and enhanced tumor targeting, while significantly reducing cardiac toxicity and other non-hematologic toxicities. Our center's previous exploratory study demonstrated that mitoxantrone hydrochloride liposome combined with cytarabine, G-CSF, and venetoclax (CMG+Ven) achieved a composite complete remission (CRc) rate of 72.6% and an MRD-negative rate of 75.6% in patients with newly diagnosed secondary or elderly AML. Furthermore, compared with the VA regimen, CMG+Ven significantly increased the MRD-negative rate and shortened hospital stay, showing promising clinical potential.
Therefore, the investigators designed a prospective, multicenter, randomized controlled trial. The study plans to enroll 168 adult patients with clinically confirmed MDS-IB2 and newly diagnosed secondary or elderly AML. Participants will be randomly assigned in a 1:1 ratio to receive one of the following induction treatments: 1) mitoxantrone hydrochloride liposome, subcutaneous cytarabine, and G-CSF combined with venetoclax (CMG+VEN), or 2) azacitidine combined with venetoclax (VA). The primary endpoint is the composite complete remission (CRc) rate following induction therapy.
2,970 studies on the registry are indexed under Leukemia, Myeloid, Acute; 744 are open to participants now.
This study's planned enrollment of 168 is above the median of 41 across 2,508 interventional studies indexed under Leukemia, Myeloid, Acute.
Browse Leukemia, Myeloid, Acute studies →Ruijin Hospital is the lead sponsor of 635 studies on the registry; 359 are open to participants now.
Counted across the registry records on this site, refreshed daily.
1. The patient fully understands the study, voluntarily participates, and signs the Informed Consent Form (ICF).
2. Age: 18-75 years inclusive. 3. Patients with clinically confirmed adult AML or MDS-IB2 (according to WHO 2022 criteria or ICC 2022 criteria). AML patients must meet any of the following:
Prior history of antecedent MPN (including ET, PV, and MF) with bone marrow fibrosis ≤ grade 2 (on a 0-3 grade scale) 4. For elderly AML patients, comprehensive assessment must show they belong to the Fit population: ECOG \< 3, CCI ≤ 0, and MMSE and SPPB assessment results meeting the Fit population criteria.
5. Liver and kidney function: ALT and AST ≤ 2.5 × ULN (≤ 5 × ULN for patients with liver infiltration); total bilirubin ≤ 1.5 × ULN (≤ 3 × ULN for patients with liver infiltration); serum creatinine ≤ 1.5 × ULN.
6. Expected survival ≥ 3 months. 7. Prior MDS-related therapy (excluding blood transfusions) must be completed at least 2 weeks before the start of study treatment. In cases of rapidly proliferative disease, hydroxyurea is permitted up to 24 hours before the start of study treatment. Toxicities from prior MDS therapy must have recovered to Grade 2 or lower before the start of study treatment.
Exclusion Criteria:
Patients who meet any of the following criteria will be excluded from the study:
Prior anti-cancer treatment history meeting any of the following:
Cardiac function or disease meeting any of the following:
Patients who achieve complete remission (CR), CR with partial hematologic recovery (CRh), CR with incomplete hematologic recovery (CRi), or morphologic leukemia-free state (MLFS) following Cycle 1 will proceed to consolidation therapy. Patients achieving a partial response (PR) or a ≥50% reduction in bone marrow blasts after Cycle 1 will receive one additional cycle of re-induction therapy with the same CMG+Ven regimen (venetoclax 400 mg daily on Days 1-7). Those who subsequently attain CR, CRh, CRi, or MLFS after Cycle 2 will proceed to consolidation therapy. Patients with no response (NR) after Cycle 1, or with NR or PR after Cycle 2, will discontinue study treatment.
Drug: Mitoxantrone Hydrochloride Liposome · Drug: Cytarabine · Drug: Granulocyte Colony-Stimulating Factor(G-CSF) · Drug: Venetoclax
Patients who achieve complete remission (CR), CR with partial hematologic recovery (CRh), CR with incomplete hematologic recovery (CRi), or morphologic leukemia-free state (MLFS) following Cycle 1 will proceed to consolidation therapy. Patients achieving a partial response (PR) or a ≥50% reduction in bone marrow blasts after Cycle 1 will receive one additional cycle of re-induction therapy with the same VA regimen. Those who subsequently attain CR, CRh, CRi, or MLFS after Cycle 2 will proceed to consolidation therapy. Patients with no response (NR) after Cycle 1, or with NR or PR after Cycle 2, will discontinue study treatment.
Drug: Venetoclax · Drug: azacitidine
Mitoxantrone Hydrochloride Liposome: 15 mg/m², administered by intravenous drip (ivgtt) on day 1
Cytarabine: 10 mg/m², administered subcutaneously (H) every 12 hours (q12h) on days 1-7
G-CSF: 5 μg/kg, administered subcutaneously (H) starting from day 0, and discontinued when WBC ≥ 20×10\^9/L
Venetoclax: 100 mg on day 2, 200 mg on day 3, and 400 mg on days 4-10, administered orally (po)
Venetoclax: 100 mg on day 1, 200 mg on day 2, and 400 mg on days 3-21 or 3-28, administered orally (po)
Azacitidine: 75 mg/m\^2, administered subcutaneously (H) on days 1-7.
Proportion of Participants With Composite Complete Remission (CRc = CR + CRh + CRI) in Induction Therapy (Assessed via European LeukemiaNet [ELN] 2022 Criteria)
Number of participants achieving composite complete remission (CRc), defined as complete remission (CR) + complete remission with partial hematologic recovery (CRh) + complete remission with incomplete hematologic recovery (CRI), assessed at the end of each 28-day cycle (up to 2 cycles) using European LeukemiaNet \[ELN\] 2022 criteria.
Time frame: At the end of each cycle (each cycle is 28 days), up to 2 cycles
Proportion of Participants With Objective Response (ORR = CRC + Morphologic Leukemia-Free State [MLFS] + Partial Remission [PR]) in Induction Therapy (Assessed via European LeukemiaNet [ELN] 2022 Criteria)
Number of participants achieving objective response (ORR), defined as composite complete remission (CRC) + morphologic leukemia-free state (MLFS) + partial remission (PR), assessed at the end of each 28-day cycle (up to 2 cycles) using European LeukemiaNet \[ELN\] 2022 criteria.
Time frame: At the end of each cycle (each cycle is 28 days), up to 2 cycles
Proportion of CRc-Achieving Participants With Measurable Residual Disease (MRD) Negativity (Assessed via Flow Cytometry Testing Per ELN 2022 Criteria)
Number of participants who achieved CRc and had MRD negativity, assessed at the end of each 28-day cycle (up to 2 cycles) using flow cytometry testing per European LeukemiaNet \[ELN\] 2022 criteria.
Time frame: At the end of each cycle (each cycle is 28 days), up to 2 cycles
Overall Survival (OS) Time (From Treatment Day 1 to Date of Death From Any Cause)
Time from day 1 of treatment to date of death from any cause, measured for all participants in the study, up to 1 year after the last enrolled participant's enrollment date.
Time frame: Up to 1 years after the date of the last enrolled participants
Relapsed-Free Survival (RFS) Time (From CRc Achievement to Hematologic Relapse or Death From Any Cause)
Time from date of achieving CRc to date of hematologic relapse or death from any cause, measured only for participants who achieved CRc, up to 1 year after the last enrolled participant's enrollment date.
Time frame: Up to 1 years after the date of the last enrolled participants
Event-Free Survival (EFS) Time (From Treatment Day 1 to Treatment Failure, Hematologic Relapse From CRc, or Death From Any Cause [Whichever Occurs First])
Time from day 1 of treatment to date of treatment failure, hematologic relapse from CRc, or death from any cause (whichever occurs first), measured for all participants in the study, up to 1 year after the last enrolled participant's enrollment date.
Time frame: Up to 1 years after the date of the last enrolled participants
Incidence of Treatment-Emergent Adverse Events (Assessed via Common Terminology Criteria for Adverse Events [CTCAE] v5.0)
Number of participants with treatment-emergent adverse events, assessed via Common Terminology Criteria for Adverse Events \[CTCAE\] v5.0, from day 1 of treatment to 28 days after the last dose.
Time frame: From day 1 of treatment to 28 days after the last dose
Exploratory Biomarker Profiling (Including Genetic Mutations, Gene Expression Profiles, and Molecular Markers in Blood/Bone Marrow)
Assessment of exploratory biomarkers (including genetic mutations, gene expression profiles, and molecular markers) in blood or bone marrow, to explore association with treatment response/resistance/prognosis in AML, at baseline and end of each 28-day cycle (up to 2 cycles).
Time frame: Baseline, end of each cycle (up to 2 cycles)
No study locations are listed for this record.
Plan to share: No
No publications or documents are linked to this record.
This study is not yet recruiting, as verified in Apr 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Ruijin Hospital