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Mitoxantrone Hydrochloride Liposome, Cytarabine, G-CSF Plus Venetoclax vs. Azacitidine Plus Venetoclax for MDS-IB2 and Secondary/Elderly AML

26 juli 2026 uppdaterad av: Ruijin Hospital

A Prospective, Multicenter, Randomized Controlled Clinical Study of Mitoxantrone Hydrochloride Liposome, Subcutaneous Cytarabine and G-CSF Combined With Venetoclax Versus Azacitidine Combined With Venetoclax in the Treatment of MDS-IB2 and Newly Diagnosed Adult Secondary or Elderly AML

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.

Studieöversikt

Detaljerad beskrivning

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.

Studietyp

Interventionell

Inskrivning (Beräknad)

168

Fas

  • Fas 3

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studiekontakt

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

  • Vuxen
  • Äldre vuxen

Tar emot friska volontärer

Nej

Beskrivning

Inclusion Criteria:

  • 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:

    1. Therapy-related AML
    2. Prior history of MDS
    3. Presence of MDS-related genetic/chromosomal abnormalities
    4. Prior history of CMML
    5. Age ≥ 60 years
    6. 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:

    1. Prior anti-cancer treatment history meeting any of the following:

      1. Prior treatment with mitoxantrone or mitoxantrone liposome.
      2. Prior treatment with venetoclax or hypomethylating agents.
      3. Prior treatment with doxorubicin or other anthracyclines, with a cumulative doxorubicin dose > 360 mg/m^2 (for other anthracyclines, 1 mg doxorubicin is equivalent to 2 mg daunorubicin or 0.5 mg idarubicin).
      4. Received anti-cancer treatment including surgery, chemotherapy, targeted therapy, etc., or participated in another clinical trial with investigational drug within 4 weeks or 5 half-lives before the first dose of study drug.
    2. Cardiac function or disease meeting any of the following:

      1. Long QTc syndrome or QTc interval > 480 ms.
      2. Complete left bundle branch block, second-degree or third-degree atrioventricular block.
      3. Severe, uncontrolled arrhythmia requiring medication.
      4. New York Heart Association (NYHA) Class ≥ II.
      5. Left ventricular ejection fraction (LVEF) < 50%.
      6. History of myocardial infarction, unstable angina, severe unstable ventricular arrhythmia or any other arrhythmia requiring treatment, clinically significant pericardial disease within 6 months before enrollment, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities.
    3. Concurrent uncontrolled malignancy other than adequately controlled non-melanoma skin basal cell carcinoma, carcinoma in situ of breast/cervix, or other malignancies that have been effectively controlled without treatment for > 6 months and patients receiving long-term non-chemotherapy treatment (e.g., hormone therapy).
    4. Uncontrolled systemic disease (e.g., progressive infection, uncontrolled hypertension, diabetes mellitus).
    5. Central nervous system (CNS) leukemia.
    6. Secondary AML with bone marrow fibrosis ≥ grade 3.
    7. Blast crisis of chronic myeloid leukemia (CML).
    8. AML with favorable-risk karyotypes: t(8;21)(q22;q22.1) RUNX1::RUNX1T1, inv(16)(p13.1q22) CBFB::MYH11, or acute promyelocytic leukemia (APL).
    9. Human immunodeficiency virus (HIV) infection (HIV antibody positive).
    10. Active hepatitis B or hepatitis C infection (HBsAg or HBcAb positive with HBV-DNA > 1×10^3 copies/mL; HCV antibody positive with HCV-RNA > 1×10^3 copies/mL).
    11. Known immediate or delayed hypersensitivity reaction to the study drug's class or excipients.

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Primärt syfte: Behandling
  • Tilldelning: Randomiserad
  • Interventionsmodell: Parallellt uppdrag
  • Maskning: Ingen (Open Label)

Vapen och interventioner

Deltagargrupp / Arm
Intervention / Behandling
Experimentell: CMG+VEN

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.

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)
Aktiv komparator: VA

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.

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.

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Proportion of Participants With Composite Complete Remission (CRc = CR + CRh + CRI) in Induction Therapy (Assessed via European LeukemiaNet [ELN] 2022 Criteria)
Tidsram: At the end of each cycle (each cycle is 28 days), up to 2 cycles
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.
At the end of each cycle (each cycle is 28 days), up to 2 cycles

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
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)
Tidsram: At the end of each cycle (each cycle is 28 days), up to 2 cycles
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.
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)
Tidsram: At the end of each cycle (each cycle is 28 days), up to 2 cycles
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.
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)
Tidsram: Up to 1 years after the date of the last enrolled participants
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.
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)
Tidsram: Up to 1 years after the date of the last enrolled participants
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.
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])
Tidsram: Up to 1 years after the date of the last enrolled participants
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.
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)
Tidsram: From day 1 of treatment to 28 days after the last dose
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.
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)
Tidsram: Baseline, end of each cycle (up to 2 cycles)
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).
Baseline, end of each cycle (up to 2 cycles)

Samarbetspartners och utredare

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Sponsor

Utredare

  • Huvudutredare: Sujiang Zhang, Ruijin Hospital

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Beräknad)

30 juli 2026

Primärt slutförande (Beräknad)

20 juli 2028

Avslutad studie (Beräknad)

31 juli 2029

Studieregistreringsdatum

Först inskickad

8 juni 2026

Först inskickad som uppfyllde QC-kriterierna

26 juli 2026

Första postat (Faktisk)

29 juli 2026

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

29 juli 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

26 juli 2026

Senast verifierad

1 april 2026

Mer information

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