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Palbociclib, All-Trans Retinoic Acid, and Venetoclax in Relapsed or Refractory Acute Myeloid Leukemia

2026年11月3日 更新者:Linhui Hu、Second Affiliated Hospital of Nanchang University

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

まだ募集していません

条件

介入・治療

研究の種類

介入

入学 (推定)

55

段階

  • フェーズ2
  • フェーズ 1

連絡先と場所

このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。

研究場所

    • Jiangxi
      • Nanchang、Jiangxi、中国、330000
        • Second Affiliated Hospital of Nanchang University
        • コンタクト:

参加基準

研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。

適格基準

就学可能な年齢

18年歳以上 (大人、高齢者)

健康ボランティアの受け入れ

いいえ

受講資格のある性別

全て

説明

**Inclusion Criteria:**

  1. 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.

  2. Adults aged 18 years or older.

  3. 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.

  4. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.

  5. Life expectancy greater than 3 months.

  6. 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.
  7. 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.

  8. 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:**

  1. Acute promyelocytic leukemia (APL), characterized by t(15;17) translocation or positivity for the PML-RARα fusion gene.
  2. Active or symptomatic central nervous system leukemia.
  3. 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.
  4. Previous treatment with venetoclax, another BCL-2 inhibitor, or a CDK4/6 inhibitor resulting in intolerable toxicity.
  5. 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.
  6. 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.
  7. 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.
  8. 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

このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。

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)

武器と介入

参加者グループ / アーム
介入・治療
実験的:Palbociclib, All-Trans Retinoic Acid, and Venetoclax

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

結果測定
メジャーの説明
時間枠
Incidence of Dose-Limiting Toxicities (DLTs) During Phase Ib
時間枠:During the first 28-day treatment cycle (Days 1-28)

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)
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

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

二次結果の測定

結果測定
メジャーの説明
時間枠
Maximum Tolerated Dose (MTD) During Phase Ib
時間枠:During the first 28-day treatment cycle (Days 1-28)

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)
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.

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.
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.

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.
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

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
Measurable Residual Disease (MRD) Negativity Rate
時間枠:At the end of each 28-day treatment cycle in participants achieving CR or CRi

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
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.

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.
Overall Survival (OS)
時間枠:From the date of study enrollment to death from any cause, assessed up to 24 months.

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.
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.

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.

協力者と研究者

ここでは、この調査に関係する人々や組織を見つけることができます。

スポンサー

研究記録日

これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。

主要日程の研究

研究開始 (推定)

2026年11月1日

一次修了 (推定)

2029年12月31日

研究の完了 (推定)

2029年12月31日

試験登録日

最初に提出

2026年10月28日

QC基準を満たした最初の提出物

2026年11月3日

最初の投稿 (実際)

2026年11月9日

学習記録の更新

投稿された最後の更新 (実際)

2026年11月9日

QC基準を満たした最後の更新が送信されました

2026年11月3日

最終確認日

2026年10月1日

詳しくは

本研究に関する用語

その他の研究ID番号

  • 2025efyC03

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?

いいえ

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米国で製造され、米国から輸出された製品。

いいえ

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