Mitoxantrone Hydrochloride Liposome, Cytarabine, G-CSF Plus Venetoclax vs. Azacitidine Plus Venetoclax for MDS-IB2 and Secondary/Elderly AML
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
調査の概要
状態
詳細な説明
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.
研究の種類
入学 (推定)
段階
- フェーズ 3
連絡先と場所
研究連絡先
- 名前:Xiaoqian Xu
- 電話番号:13816205940
- メール:Ellenxxq@qq.com
参加基準
適格基準
就学可能な年齢
- 大人
- 高齢者
健康ボランティアの受け入れ
説明
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:
- Therapy-related AML
- Prior history of MDS
- Presence of MDS-related genetic/chromosomal abnormalities
- Prior history of CMML
- Age ≥ 60 years
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:
- Prior treatment with mitoxantrone or mitoxantrone liposome.
- Prior treatment with venetoclax or hypomethylating agents.
- 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).
- 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.
Cardiac function or disease meeting any of the following:
- Long QTc syndrome or QTc interval > 480 ms.
- Complete left bundle branch block, second-degree or third-degree atrioventricular block.
- Severe, uncontrolled arrhythmia requiring medication.
- New York Heart Association (NYHA) Class ≥ II.
- Left ventricular ejection fraction (LVEF) < 50%.
- 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.
- 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).
- Uncontrolled systemic disease (e.g., progressive infection, uncontrolled hypertension, diabetes mellitus).
- Central nervous system (CNS) leukemia.
- Secondary AML with bone marrow fibrosis ≥ grade 3.
- Blast crisis of chronic myeloid leukemia (CML).
- AML with favorable-risk karyotypes: t(8;21)(q22;q22.1) RUNX1::RUNX1T1, inv(16)(p13.1q22) CBFB::MYH11, or acute promyelocytic leukemia (APL).
- Human immunodeficiency virus (HIV) infection (HIV antibody positive).
- 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).
- Known immediate or delayed hypersensitivity reaction to the study drug's class or excipients.
研究計画
研究はどのように設計されていますか?
デザインの詳細
- 主な目的:処理
- 割り当て:ランダム化
- 介入モデル:並列代入
- マスキング:なし(オープンラベル)
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
|
実験的: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)
|
|
アクティブコンパレータ: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.
|
この研究は何を測定していますか?
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Proportion of Participants With Composite Complete Remission (CRc = CR + CRh + CRI) in Induction Therapy (Assessed via European LeukemiaNet [ELN] 2022 Criteria)
時間枠: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
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
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)
時間枠: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)
時間枠: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)
時間枠: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)
時間枠: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])
時間枠: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)
時間枠: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)
時間枠: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)
|
協力者と研究者
スポンサー
捜査官
- 主任研究者:Sujiang Zhang、Ruijin Hospital
研究記録日
主要日程の研究
研究開始 (推定)
一次修了 (推定)
研究の完了 (推定)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
- CSPC-DED-AML-K24
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
医薬品およびデバイス情報、研究文書
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