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Study on the Efficacy and Safety of VA Regimen Compared to "3+7" Regimen in Newly Diagnosed AML With NPM1 or IDH1/IDH2 Mutations

23 juin 2026 mis à jour par: Shen yang

A Prospective, Multicenter, Randomized Controlled, Open Label, Non-Inferiority Study Comparing the Efficacy and Safety of the VA Regimen (Venetoclax Combined With Azacitidine) With the "3+7" Regimen in the Treatment of Newly Diagnosed AML Patients With NPM1 or IDH1/IDH2 Mutations

This prospective, multicenter, randomized, open-label, non-inferiority clinical study aims to compare the efficacy and safety of VA regimen (venetoclax combined with azacitidine) versus conventional "3+7" chemotherapy regimen in adult patients aged 18 to 65 years with newly diagnosed acute myeloid leukemia (AML) carrying NPM1, IDH1 or IDH2 gene mutations.

The primary goal of this trial is to check whether the VA treatment can reach a non-inferior composite complete remission rate at the end of the induction treatment cycle, which is the key primary endpoint of this research. Several secondary clinical outcomes will also be evaluated in this study, including the rate of minimal residual disease (MRD) negativity after remission, duration of remission, 1-year event-free survival rate and 1-year overall survival rate of enrolled patients. In addition, the safety and treatment-related side effects occurring during the whole induction treatment phase will be systematically collected and compared between two groups as another important secondary assessment.

Eligible enrolled participants will be randomly split into two study groups: patients in experimental group will receive venetoclax plus azacitidine (VA regimen), while patients in control group will receive standard "3+7" induction chemotherapy following conventional clinical protocol. All subjects will complete regular disease assessment, laboratory examinations and scheduled follow-up visits as required by trial design during treatment and post-treatment observation period.

Researchers will collect and analyze all above clinical outcome data from all participants, to verify the non-inferior efficacy and relative safety of VA regimen for this specific subtype of newly diagnosed AML patients.

Aperçu de l'étude

Statut

Pas encore de recrutement

Les conditions

Intervention / Traitement

Type d'étude

Interventionnel

Inscription (Estimé)

148

Phase

  • Phase 2

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Coordonnées de l'étude

Lieux d'étude

  • Chine
    • Shanghai Municipality
      • Shanghai, Shanghai Municipality, Chine, 200025
        • Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
        • Contact:

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

  • Adulte
  • Adulte plus âgé

Accepte les volontaires sains

Non

La description

Inclusion Criteria:

  • Age ≥ 65 years old ≥ 18 years old;
  • Diagnosed as acute myeloid leukemia (non APL) (diagnostic criteria refer to the 2022 ELN classification system);
  • Initial diagnosis accompanied by NPM1 mutations (A, B, D types and rare types are all acceptable) and/or IDH1/IDH2 mutations;
  • Have not received any other induction therapy before (except hydroxyurea);
  • Physical fitness status score (ECOG PS) 0-3;

  • Having sufficient organ function, defined as follows:

    1. Liver function: serum total bilirubin ≤ 3 x upper limit of normal range (ULN), aspartate aminotransferase (AST), alanine aminotransferase (ALT), and alkaline phosphatase (ALP) ≤ 3 x ULN, unless considered to be caused by leukemia;
    2. Renal function: endogenous creatinine clearance rate ≥ 30ml/min;
    3. Heart function: NYHA classification ≤ 2 points;
  • Participants must have the ability to understand and be willing to participate in this study, and sign an informed consent form.

Exclusion Criteria:

  • Acute promyelocytic leukemia;
  • Merge extramedullary infiltration such as central nervous system leukemia;
  • Have a clear history of CMML or MDS, and later progress to AML; Or have a history of malignant tumors;
  • There is uncontrolled active infection (including bacterial, fungal, or viral infections);
  • Pregnant or lactating women;
  • Researchers determine that participants are not suitable to participate in this experiment

Plan d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

  • Objectif principal: Traitement
  • Répartition: Randomisé
  • Modèle interventionnel: Affectation parallèle
  • Masquage: Aucun (étiquette ouverte)

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Expérimental: Venetoclax plus Azacitidine (VA regimen)
  1. Venetoclax: oral administration, specified dosage and schedule for induction cycle;
  2. Azacitidine: subcutaneous/intravenous injection with standard induction dose and treatment cycle per clinical protocol.
Médicament: VEN combined with azacitidine
Venetoclax,oral targeted anti-BCL-2 agent and Azacitidine,hypomethylating agent, given via injection for experimental VA arm only
Comparateur actif: "3+7" induction chemotherapy
Standard 3+7 induction chemotherapy (Cytarabine continuous infusion for 7 days plus Anthracycline intravenous infusion for 3 days) following routine clinical induction regimen for AML.
Médicament: Cytarabine plus Daunorubicin
Cytarabine,continuous intravenous infusion for total 7 days and Daunorubicin,Intravenous anthracycline chemotherapy administered for 3 days,in standard 3+7 induction regimen

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Délai
Composite complete remission rate at the end of induction cycle
Délai: At the end of 1-2 induction treatment cycles (each cycle is 28 days)
At the end of 1-2 induction treatment cycles (each cycle is 28 days)

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
Composite Complete Remission
Délai: At the end of 1-2 induction treatment cycles (each cycle is 28 days)
At the end of 1-2 induction treatment cycles (each cycle is 28 days)
Minimal residual disease (MRD) negative rate after remission
Délai: At the end of induction cycle (each cycle is 28 days)
At the end of induction cycle (each cycle is 28 days)
Duration of Response (DoR)
Délai: From date of confirmed complete response (CR) until documented disease relapse or death from any cause, assessed up to 24 months after randomization
From date of confirmed complete response (CR) until documented disease relapse or death from any cause, assessed up to 24 months after randomization
1-year Event-Free Survival (EFS) rate
Délai: From date of randomization until first documented treatment failure, relapse, or death from any cause, assessed up to 24 months after randomization; 1-year rate calculated at 12 months post-randomization
From date of randomization until first documented treatment failure, relapse, or death from any cause, assessed up to 24 months after randomization; 1-year rate calculated at 12 months post-randomization
1-year Overall Survival (OS) rate
Délai: From date of randomization until death from any cause, assessed up to 24 months after randomization; 1-year rate calculated at 12 months post-randomization
From date of randomization until death from any cause, assessed up to 24 months after randomization; 1-year rate calculated at 12 months post-randomization
1-year recurrence free survival rate
Délai: From date of randomization until first confirmed disease recurrence or death from any cause, assessed up to 24 months after randomization; the 1-year rate will be calculated at the 12-month timepoint after randomization
From date of randomization until first confirmed disease recurrence or death from any cause, assessed up to 24 months after randomization; the 1-year rate will be calculated at the 12-month timepoint after randomization
Safety profile during induction therapy
Délai: From initiation of induction therapy through the end of the induction treatment period,assessed up to 8 weeks after randomization
Evaluate the incidence of grade 3 and grade 4 adverse events classified per CTCAE Version 5.0, the duration of severe adverse events, type, frequency and management of all treatment-emergent adverse events occurring during the induction treatment phase.
From initiation of induction therapy through the end of the induction treatment period,assessed up to 8 weeks after randomization

Autres mesures de résultats

Mesure des résultats
Délai
Correlation between baseline AML gene mutation status detected by next-generation sequencing and anti-leukemic efficacy endpoints (complete remission rate, MRD-negative rate, 1-year recurrence-free survival rate) in VA regimen arm
Délai: From date of randomization up to 24 months after randomization, biomarker-efficacy correlation analysis will be performed using clinical data collected within the first 12 months post randomization
From date of randomization up to 24 months after randomization, biomarker-efficacy correlation analysis will be performed using clinical data collected within the first 12 months post randomization
Hospitalization duration during induction therapy
Délai: From the date of induction therapy initiation through completion of the first induction cycle (each cycle is 28 days)
From the date of induction therapy initiation through completion of the first induction cycle (each cycle is 28 days)
Transfusion volume during induction therapy
Délai: From the date of induction therapy initiation through completion of the first induction cycle (each cycle is 28 days)
From the date of induction therapy initiation through completion of the first induction cycle (each cycle is 28 days)
Medical cost during induction therapy
Délai: From the date of induction therapy initiation through completion of the first induction cycle (each cycle is 28 days)
From the date of induction therapy initiation through completion of the first induction cycle (each cycle is 28 days)

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Parrainer

Shen yang

Les enquêteurs

  • Chercheur principal: Yang Shen, MD, Ruijin Hospital

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Estimé)

1 juillet 2026

Achèvement primaire (Estimé)

31 mai 2028

Achèvement de l'étude (Estimé)

31 mai 2032

Dates d'inscription aux études

Première soumission

10 juin 2026

Première soumission répondant aux critères de contrôle qualité

23 juin 2026

Première publication (Réel)

24 juin 2026

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

24 juin 2026

Dernière mise à jour soumise répondant aux critères de contrôle qualité

23 juin 2026

Dernière vérification

1 juin 2026

Plus d'information

Termes liés à cette étude

Mots clés

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

  • NPM1/IDH-AML01

Plan pour les données individuelles des participants (IPD)

Prévoyez-vous de partager les données individuelles des participants (DPI) ?

NON

Informations sur les médicaments et les dispositifs, documents d'étude

Étudie un produit pharmaceutique réglementé par la FDA américaine

Non

Étudie un produit d'appareil réglementé par la FDA américaine

Non

Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .

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