Venetoclax Combined With Azacitidine and CAG in Refractory/Relapse Acute Myeloid Leukemia

Hematology department of the 920th hospital (Other)
Overall Status
Recruiting ID

Study Details

Study Description

Brief Summary

This study aims to assess the therapeutic efficacy and safety of venetoclax in combination with azacitidine and CAG as induction regimen in Patients with Refreactory/Relapse Acute Myeloid Leukemia.

Condition or Disease Intervention/Treatment Phase
Phase 2

Detailed Description

This is an open-label, multicenter, phase II clinical trial to assess the therapeutic efficacy and safety of venetoclax in combination with azacitidine (VA) and CAG (G-CSF priming, low dose cytarabine, and aclarubicin) as induction regimen in Patients with Refreactory/Relapse Acute Myeloid Leukemia(AML).

Previous studies have shown that venetoclax plus intense chemotherapy represent promising efficacy in de novo AML patients with high complete remission rates and good tolerance. Our preliminary results suggest that venetoclax in combination with azacitidine and CAG are well tolerated and effective for patients who were diagnosed with refreactory/relapse AML. Thus, this phase II clinical trial is going to further explore its efficacy and safety. It is expected that about 42 patients will take part in this trial.

Study Design

Study Type:
Anticipated Enrollment :
42 participants
Intervention Model:
Single Group Assignment
None (Open Label)
Masking Description:
Open lable
Primary Purpose:
Official Title:
Phase II Trial of Venetoclax in Combination With Azacitidine and CAG as Induction Therapy in Patients With Refractory/Relapse Acute Myeloid Leukemia
Actual Study Start Date :
Feb 1, 2023
Anticipated Primary Completion Date :
Feb 1, 2024
Anticipated Study Completion Date :
Feb 1, 2026

Arms and Interventions

Arm Intervention/Treatment
Experimental: VACAG(Venetoclax Combined with Azacitidine and CAG)regimen

Participants will receive induction as azacitidine on days 1-7, venetoclax aily on days 1-28, cytarabine q12h on days 1-7, aclacinomycin on days 1,3,5,7, and granulocyte colony-stimulating factor on days 0-8. Participants will receive second induction if not reach complete remission.

Drug: Venetoclax
VA regimen Drug: Venetoclax orally once daily (100 mg d1, 200 mg d2, 400 mg d3-21); Drug: Azacitidine 75 mg/m2 subcutaneously once daily on days 1-7. CAG regimen Drug: Cytarabine 10mg/m2 subcutaneously q12h on days 1-7 Drug: Aclacinomycin 12-14mg/m2 on days 1,3,5,7 Drug: Granulocyte colony-stimulating factor 5ug/kg on days 0-8, discontinue if WBC >20×109/L
Other Names:
  • Azacitidine
  • Cytarabine
  • Aclacinomycin
  • Granulocyte colony-stimulating factor
  • Outcome Measures

    Primary Outcome Measures

    1. Overall response rate (ORR) [up to 2 cycles from the start of VACAG regimen (each cycle is 28 days)]

      The overall remission rate (ORR) was defined as the percentage of patients who achieved complete remission (CR), complete remission with incomplete count recovery (CRi), or morphologic leukemia free state (MLFS) per the International Working Group criteria for AML.

    Secondary Outcome Measures

    1. Rate of Minimal Residual Disease (MRD) negativity [up to 2 years]

      Percentage of participants who converted to MRD < 10^-3 by flow cytometry before initiation of consolidation therapy.

    2. Incidence of Treatment-Emergent Adverse Events [up to 2 years]

      Safety and tolerability analysis will be assessed by the Common Toxicity Criteria for Adverse Events (CTCAE) version 5.0.

    3. Duration of myelosuppression [up to 2 years]

      The duration of absolute value of peripheral blood neutrophils <0.5×10^9/L and platelet count <50×10^9/L during myelosuppression.

    4. Leukaemia-free survival [up to 2 years]

      Leukaemia-free survival will be defined as the time since date of CR until either relapse or death in remission.

    5. Overall survival [up to 2 years]

      Overall Survival be defined as the time from administration of the initial doses until death from any cause.

    Eligibility Criteria


    Ages Eligible for Study:
    18 Years to 65 Years
    Sexes Eligible for Study:
    Accepts Healthy Volunteers:
    Inclusion Criteria:
    1. Patients ≥ 18 years old and ≤ 65 years old

    2. Refractory/Relapse AML patients according to 2016 World Health Organization (WHO) classification;

    3. Eastern Cooperative Oncology Group (ECOG) Performance status score less than 3;

    4. Liver function: Total bilirubin ≦2 upper limit of normal (ULN); aspartate aminotransferase (AST) ≦3 ULN; alanine aminotransferase (ALT)≦3 ULN

    5. Renal function:Ccr(Creatinine Clearance Rate) ≧30 ml/min; Scr (serum creatinine) ≦2 ULN

    6. Heart function: left ventricular ejection fraction ≧45%

    7. Patients must participate in this clinical trial voluntarily and sign an informed consent form.

    Exclusion Criteria:
    1. Other diseases;

    2. AML with central nervous system (CNS) infiltration;

    3. Patients have received prior CAG or VA regimen before;

    4. Patients with a life expectancy <3 months

    5. Patients with uncontrolled active infection;

    6. HIV infection;

    7. Evidence of other clinically significant uncontrolled condition(s) including, but not limited to: a) Chronic hepatitis B virus (HBV) or hepatitis C (HCV) requiring treatment. b) An active second cancer that requires treatment within 6 months of study entry

    8. Female who are pregnant, breast feeding or childbearing potential.

    9. Patients deemed unsuitable for enrollment by the investigator;

    Contacts and Locations


    Site City State Country Postal Code
    1 Department of Hematology,920th Hospital of Joint Logistic Support Force of People's Liberation Kunming Yunnan China

    Sponsors and Collaborators

    • Hematology department of the 920th hospital


    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information


    None provided.
    Responsible Party:
    Hematology department of the 920th hospital Identifier:
    Other Study ID Numbers:
    • KM02
    First Posted:
    Apr 11, 2023
    Last Update Posted:
    Apr 11, 2023
    Last Verified:
    Feb 1, 2023
    Individual Participant Data (IPD) Sharing Statement:
    Plan to Share IPD:
    Studies a U.S. FDA-regulated Drug Product:
    Studies a U.S. FDA-regulated Device Product:
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Apr 11, 2023