Palbociclib, All-Trans Retinoic Acid, and Venetoclax in Relapsed or Refractory Acute Myeloid Leukemia
A Phase Ib/II, Multicenter, Open-Label, Single-Arm Study of Palbociclib, All-Trans Retinoic Acid, and Venetoclax in Patients With Relapsed or Refractory Acute Myeloid Leukemia
This is a prospective, multicenter, single-arm, open-label Phase Ib/II clinical trial evaluating the safety, tolerability, and preliminary efficacy of palbociclib, all-trans retinoic acid (ATRA), and venetoclax (PAV regimen) in adult patients with relapsed or refractory acute myeloid leukemia (AML).
The Phase Ib portion will use a standard 3+3 dose-escalation design to evaluate dose-limiting toxicities (DLTs), determine the maximum tolerated dose (MTD), and establish the recommended Phase II dose (RP2D). The Phase II portion will use Simon's two-stage design to further evaluate the antileukemic activity and safety of the combination regimen, with overall response rate (ORR) as the primary efficacy endpoint.
Additional assessments will include complete remission (CR), complete remission with incomplete hematologic recovery (CRi), measurable residual disease (MRD) negativity, event-free survival (EFS), overall survival (OS), and duration of response (DOR). Exploratory analyses will investigate treatment-associated changes in leukemic cell differentiation and apoptosis and identify potential biomarkers associated with treatment response.
調査の概要
状態
状態
条件
条件
介入・治療
介入・治療
研究の種類
研究の種類
入学 (推定)
入学
段階
段階
- フェーズ2
- フェーズ 1
連絡先と場所
研究場所
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Jiangxi
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Nanchang、Jiangxi、中国、330000
- Second Affiliated Hospital of Nanchang University
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コンタクト:
- linhu hu
- 電話番号:+86 13907984324
- メール:hulinhui1992@163.com
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参加基準
適格基準
適格基準
就学可能な年齢
健康ボランティアの受け入れ
受講資格のある性別
説明
**Inclusion Criteria:**
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The participant or their legally authorized representative must provide written informed consent and be willing and able to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures.
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Adults aged 18 years or older.
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Histologically, immunophenotypically, cytogenetically, or molecularly confirmed relapsed or refractory acute myeloid leukemia (R/R AML) according to WHO classification criteria. Relapsed or refractory AML is defined as failure to achieve remission after at least one cycle of standard induction therapy or recurrence of disease after achieving remission.
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Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
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Life expectancy greater than 3 months.
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Adequate organ function, defined as:
- Total bilirubin ≤1.5 × upper limit of normal (ULN).
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤2.5 × ULN, or ≤5 × ULN if the elevation is attributable to leukemic involvement.
- Creatinine clearance ≥30 mL/min.
- Left ventricular ejection fraction (LVEF) ≥50%.
- Corrected QT interval using Fridericia's formula (QTcF) ≤450 ms for males or ≤470 ms for females.
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White blood cell (WBC) count <25 × 10⁹/L before the first dose of study treatment. Cytoreduction with hydroxyurea or leukapheresis is permitted before treatment initiation.
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Women of childbearing potential must have a negative pregnancy test before enrollment. Male participants and women of childbearing potential must agree to use highly effective contraception during the study and for at least 6 months after the last dose of study treatment.
**Exclusion Criteria:**
- Acute promyelocytic leukemia (APL), characterized by t(15;17) translocation or positivity for the PML-RARα fusion gene.
- Active or symptomatic central nervous system leukemia.
- Use of strong CYP3A4 inhibitors (e.g., voriconazole or posaconazole) or strong CYP3A4 inducers (e.g., rifampin or carbamazepine) within 7 days before the first dose of study treatment when these medications cannot be replaced. Participants requiring strong CYP3A4 inhibitors for fungal infection prophylaxis or treatment may be enrolled if no suitable alternative is available, provided that palbociclib and venetoclax doses are adjusted according to the study protocol. However, strong CYP3A4 inhibitors are prohibited during venetoclax dose ramp-up.
- Previous treatment with venetoclax, another BCL-2 inhibitor, or a CDK4/6 inhibitor resulting in intolerable toxicity.
- Severe gastrointestinal disorders that may significantly impair oral drug absorption, such as Crohn's disease or severe ulcerative colitis, or inability to swallow oral tablets or capsules.
- Severe, active, and uncontrolled systemic infection, including bacterial, fungal, or viral infection; positive testing for human immunodeficiency virus (HIV); or active hepatitis B or hepatitis C infection.
- History of congenital or acquired long QT syndrome; current use of medications known to prolong the QT interval that cannot be discontinued (e.g., amiodarone, sotalol, or methadone); or myocardial infarction, unstable angina, severe arrhythmia, or New York Heart Association (NYHA) Class III-IV heart failure within the preceding 6 months.
- History of another malignancy, except for adequately treated nonmelanoma skin cancer, cervical carcinoma in situ, or similar conditions, with a disease-free interval of less than 2 years.
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:なし
- 介入モデル:単一グループの割り当て
- マスキング:なし(オープンラベル)
アーム数
武器と介入
参加者グループ / アーム参加者グループ / アーム |
介入・治療介入・治療 |
|---|---|
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実験的:Palbociclib, All-Trans Retinoic Acid, and Venetoclax
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Palbociclib will be administered orally on Days 1-21 of each 28-day treatment cycle. During Phase Ib, three dose levels will be evaluated using a standard 3+3 dose-escalation design to determine the maximum tolerated dose (MTD) and recommended Phase II dose (RP2D). During Phase II, palbociclib will be administered at the established RP2D in combination with all-trans retinoic acid and venetoclax. All-trans retinoic acid (ATRA) will be administered orally at a dose of 25 mg/m² on Days 1-28 of each 28-day treatment cycle, in combination with palbociclib and venetoclax. Venetoclax will be administered orally with a dose ramp-up of 100 mg on Day 1, 200 mg on Day 2, and 400 mg daily from Day 3 onward. Treatment duration will range from 14 to 28 days per 28-day cycle, depending on hematologic recovery and myelosuppression, with dose adjustments according to the study protocol. |
この研究は何を測定していますか?
主要な結果の測定
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Incidence of Dose-Limiting Toxicities (DLTs) During Phase Ib
時間枠:During the first 28-day treatment cycle (Days 1-28)
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The proportion of participants experiencing dose-limiting toxicities (DLTs) during the first 28-day treatment cycle in Phase Ib. DLTs will be assessed according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), version 5.0, and include Grade 4 or higher hematologic toxicity lasting more than 7 days with bone marrow hypoplasia or Grade 3 or higher nonhematologic toxicity that does not resolve with optimal supportive care. |
During the first 28-day treatment cycle (Days 1-28)
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Overall Response Rate (ORR) During Phase II
時間枠:At the end of each 28-day treatment cycle, until disease progression or treatment discontinuation
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The proportion of participants in Phase II who achieve complete remission (CR), complete remission with incomplete hematologic recovery (CRi), or partial remission (PR). Treatment response will be assessed by bone marrow examination according to the response criteria specified in the study protocol. |
At the end of each 28-day treatment cycle, until disease progression or treatment discontinuation
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二次結果の測定
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Maximum Tolerated Dose (MTD) During Phase Ib
時間枠:During the first 28-day treatment cycle (Days 1-28)
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The highest evaluated dose level of palbociclib in combination with all-trans retinoic acid and venetoclax that is considered tolerable based on dose-limiting toxicities observed during the Phase Ib 3+3 dose-escalation stage. |
During the first 28-day treatment cycle (Days 1-28)
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Recommended Phase II Dose (RP2D)
時間枠:From initiation of study treatment through completion of the Phase Ib dose-escalation stage, assessed up to 6 months.
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The recommended Phase II dose of palbociclib in combination with all-trans retinoic acid and venetoclax, determined based on the dose-limiting toxicity findings and overall safety and tolerability during Phase Ib. |
From initiation of study treatment through completion of the Phase Ib dose-escalation stage, assessed up to 6 months.
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Incidence and Severity of Adverse Events (AEs)
時間枠:From the first dose of study treatment through 30 days after the last dose, assessed up to 12 months.
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The number and percentage of participants experiencing adverse events (AEs) and serious adverse events (SAEs), including their severity and relationship to study treatment. Adverse events will be graded according to NCI CTCAE version 5.0. |
From the first dose of study treatment through 30 days after the last dose, assessed up to 12 months.
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Complete Remission (CR) and Complete Remission With Incomplete Hematologic Recovery (CRi) Rate
時間枠:At the end of each 28-day treatment cycle, until disease progression or treatment discontinuation
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The proportion of participants achieving CR or CRi. CR is defined as bone marrow blasts below 5%, absence of peripheral blood blasts and extramedullary disease, absolute neutrophil count above 1.0 × 10^9/L, and platelet count above 100 × 10^9/L. CRi meets the bone marrow criteria for CR but with incomplete neutrophil or platelet recovery. |
At the end of each 28-day treatment cycle, until disease progression or treatment discontinuation
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Measurable Residual Disease (MRD) Negativity Rate
時間枠:At the end of each 28-day treatment cycle in participants achieving CR or CRi
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The proportion of participants achieving MRD negativity among those with CR or CRi. MRD will be assessed by multiparameter flow cytometry, with MRD negativity defined as a level below 0.1%. |
At the end of each 28-day treatment cycle in participants achieving CR or CRi
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Event-Free Survival (EFS)
時間枠:From the date of study enrollment to the first occurrence of disease progression, relapse, or death from any cause, whichever occurs first, assessed up to 24 months.
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Time from study enrollment to the first occurrence of disease progression, relapse, or death from any cause. Participants without an event will be censored at the date of their last disease assessment. |
From the date of study enrollment to the first occurrence of disease progression, relapse, or death from any cause, whichever occurs first, assessed up to 24 months.
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Overall Survival (OS)
時間枠:From the date of study enrollment to death from any cause, assessed up to 24 months.
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Time from study enrollment to death from any cause. Participants who are alive at the time of analysis will be censored at the date they were last known to be alive. |
From the date of study enrollment to death from any cause, assessed up to 24 months.
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Duration of Response (DOR)
時間枠:From the date of first documented CR or CRi to disease relapse or death from any cause, whichever occurs first, assessed up to 24 months.
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Time from the first documented CR or CRi to disease relapse or death from any cause, whichever occurs first, among participants achieving CR or CRi. |
From the date of first documented CR or CRi to disease relapse or death from any cause, whichever occurs first, assessed up to 24 months.
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協力者と研究者
研究記録日
主要日程の研究
研究開始 (推定)
研究開始
一次修了 (推定)
一次修了
研究の完了 (推定)
研究の完了
試験登録日
最初に提出
最初に提出
QC基準を満たした最初の提出物
QC基準を満たした最初の提出物
最初の投稿 (実際)
最初の投稿
学習記録の更新
投稿された最後の更新 (実際)
投稿された最後の更新
QC基準を満たした最後の更新が送信されました
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
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
その他の研究ID番号
- 2025efyC03
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