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

3 novembre 2026 aggiornato da: 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.

Panoramica dello studio

Stato

Non ancora reclutamento

Condizioni

Intervento / Trattamento

Tipo di studio

Interventistico

Iscrizione (Stimato)

55

Fase

  • Fase 2
  • Fase 1

Contatti e Sedi

Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.

Luoghi di studio

    • Jiangxi
      • Nanchang, Jiangxi, Cina, 330000
        • Second Affiliated Hospital of Nanchang University
        • Contatto:

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

18 anni e precedenti (Adulto, Adulto più anziano)

Accetta volontari sani

No

Sessi ammissibili allo studio

Tutto

Descrizione

**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.

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Dettagli di progettazione

  • Scopo principale: Trattamento
  • Assegnazione: N / A
  • Modello interventistico: Assegnazione di gruppo singolo
  • Mascheramento: Nessuno (etichetta aperta)

Armi e interventi

Gruppo di partecipanti / Arm
Intervento / Trattamento
Sperimentale: 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.

Cosa sta misurando lo studio?

Misure di risultato primarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Incidence of Dose-Limiting Toxicities (DLTs) During Phase Ib
Lasso di tempo: 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
Lasso di tempo: 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

Misure di risultato secondarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Maximum Tolerated Dose (MTD) During Phase Ib
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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.

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Sponsor

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Studia le date principali

Inizio studio (Stimato)

1 novembre 2026

Completamento primario (Stimato)

31 dicembre 2029

Completamento dello studio (Stimato)

31 dicembre 2029

Date di iscrizione allo studio

Primo inviato

28 ottobre 2026

Primo inviato che soddisfa i criteri di controllo qualità

3 novembre 2026

Primo Inserito (Effettivo)

9 novembre 2026

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

9 novembre 2026

Ultimo aggiornamento inviato che soddisfa i criteri QC

3 novembre 2026

Ultimo verificato

1 ottobre 2026

Maggiori informazioni

Termini relativi a questo studio

Altri numeri di identificazione dello studio

  • 2025efyC03

Piano per i dati dei singoli partecipanti (IPD)

Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?

NO

Informazioni su farmaci e dispositivi, documenti di studio

Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti

No

Studia un dispositivo regolamentato dalla FDA degli Stati Uniti

No

prodotto fabbricato ed esportato dagli Stati Uniti

No

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .