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Risk Stratification-directed Therapy for AML With t(8;21) /AML1-ETO+

Primary Purpose

Acute Myeloid Leukemia, Risk Stratification

Status
Completed
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
Consolidation with chemotherapy (CT) or autologous hematopoietic stem cell transplantation (auto-HSCT)
Consolidation with auto-HSCT or HLA-matched HSCT
allogeneic HSCT
Sponsored by
Nanfang Hospital, Southern Medical University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Acute Myeloid Leukemia

Eligibility Criteria

14 Years - 70 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • AE AML aged 15-60
  • No abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
  • Expected survival time is more than 2 months

Exclusion Criteria:

  • Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
  • Patients with any conditions not suitable for the trial (investigators' decision)

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm 3

    Arm Type

    Experimental

    Experimental

    Experimental

    Arm Label

    Low risk group

    Intermediate risk group

    High risk group

    Arm Description

    Patients with KIT-ASXL1- (non-mutation, NM) and acquiring main molecular response (MMR) after two cycles of consolidation.

    Patients with KIT+/ASXL1+ (single mutation, 1M) and acquiring MMR after two cycles of consolidation.

    Patients with KIT+ASXL1+ (two mutations ,2M) or without acquiring MMR after two cycles of consolidation.

    Outcomes

    Primary Outcome Measures

    overall survival (OS)

    Secondary Outcome Measures

    leukemia relapse rate
    disease-free survival (DFS)
    event Free Survival (EFS)

    Full Information

    First Posted
    October 11, 2016
    Last Updated
    August 6, 2023
    Sponsor
    Nanfang Hospital, Southern Medical University
    Collaborators
    Zhujiang Hospital
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    1. Study Identification

    Unique Protocol Identification Number
    NCT02936089
    Brief Title
    Risk Stratification-directed Therapy for AML With t(8;21) /AML1-ETO+
    Official Title
    Risk Stratification-directed Therapy for Acute Myeloid Leukemia With t(8;21) /AML1-ETO-positive
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    August 2023
    Overall Recruitment Status
    Completed
    Study Start Date
    October 2016 (undefined)
    Primary Completion Date
    December 31, 2021 (Actual)
    Study Completion Date
    December 31, 2022 (Actual)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Nanfang Hospital, Southern Medical University
    Collaborators
    Zhujiang Hospital

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    No
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    Yes

    5. Study Description

    Brief Summary
    Acute myeloid leukemia with t(8;21) /AML1-ETO-positive (AE AML) is a heterogeneous disease entailing different prognoses. There were significant differences in the therapeutic effect between different subgroups of AE AML. Therefore, risk stratification-directed therapy is very necessary for AE AML.
    Detailed Description
    Acute myeloid leukemia with t(8;21) /AML1-ETO-positive (AE AML) is a heterogeneous disease entailing different prognoses.There were significant differences in the therapeutic effect between different subgroups of AE AML. For example, patients with c-kit mutation had higher relapse rate and lower overall survival, compared with those without c-kit mutation. Therefore, risk stratification-directed therapy is very necessary for AE AML. The purpose of this study is to establish risk stratification-directed therapy for AE AML.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Acute Myeloid Leukemia, Risk Stratification

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Not Applicable
    Interventional Study Model
    Parallel Assignment
    Model Description
    Patients with newly diagnosed AML1/ETO-positive AML took risk-directed stratification therapy based on c-KIT and ASXL1 mutations and measurable residual disease (MRD). low risk (LR) group (KIT-ASXL1- with main molecular response (MMR)) was recommended to chemotherapy (CT) or autologous hematopoietic stem cell transplantation (auto-HSCT). Intermediate risk (IR) group (KIT+/ASXL1+ with MMR) was suggested for auto-HSCT or HLA-matched HSCT. High risk (HR) group (KIT+ASXL1+ or without MMR) was treated with allogeneic (allo-) HSCT.
    Masking
    None (Open Label)
    Allocation
    Non-Randomized
    Enrollment
    207 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Low risk group
    Arm Type
    Experimental
    Arm Description
    Patients with KIT-ASXL1- (non-mutation, NM) and acquiring main molecular response (MMR) after two cycles of consolidation.
    Arm Title
    Intermediate risk group
    Arm Type
    Experimental
    Arm Description
    Patients with KIT+/ASXL1+ (single mutation, 1M) and acquiring MMR after two cycles of consolidation.
    Arm Title
    High risk group
    Arm Type
    Experimental
    Arm Description
    Patients with KIT+ASXL1+ (two mutations ,2M) or without acquiring MMR after two cycles of consolidation.
    Intervention Type
    Other
    Intervention Name(s)
    Consolidation with chemotherapy (CT) or autologous hematopoietic stem cell transplantation (auto-HSCT)
    Intervention Description
    For CT, patients were treated with high dose cytarabine (HDAC), cytarabine at a dosage of 1-3 g/m2 q12 h ×6 doses, for 4-6 cycles. For auto-HSCT, patients were treated with 3 cycles of HDAC and then bridged to auto-HSCT.
    Intervention Type
    Other
    Intervention Name(s)
    Consolidation with auto-HSCT or HLA-matched HSCT
    Intervention Description
    For auto-HSCT, patients were treated with 3 cycles of HDAC and then bridged to auto-HSCT. For HLA-matched HSCT, patients were treated with 1-2 cycles of HDAC and then bridged to HLA-matched HSCT. HLA-matched donors were available in these patients.
    Intervention Type
    Other
    Intervention Name(s)
    allogeneic HSCT
    Intervention Description
    For allogeneic HSCT, patients were treated with 1-2 cycles of HDAC and then bridged to allogeneic HSCT, including HLA-matched and haploidentical transplantation.
    Primary Outcome Measure Information:
    Title
    overall survival (OS)
    Time Frame
    3 year
    Secondary Outcome Measure Information:
    Title
    leukemia relapse rate
    Time Frame
    3 year
    Title
    disease-free survival (DFS)
    Time Frame
    3 year
    Title
    event Free Survival (EFS)
    Time Frame
    3 year

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    14 Years
    Maximum Age & Unit of Time
    70 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: AE AML aged 14-70 No abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure) Expected survival time is more than 2 months Exclusion Criteria: Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure) Patients with any conditions not suitable for the trial (investigators' decision)
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Dan Xu
    Organizational Affiliation
    Nanfang Hospital, Southern Medical University
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Plan to Share IPD
    Yes
    IPD Sharing Plan Description
    All collected IPD, all IPD that underlie results will be shared in a publication. For the detail, those who are interested in can contact the authors.
    IPD Sharing Time Frame
    The data will be available after being published, for at least five years.
    IPD Sharing Access Criteria
    All collected IPD, all IPD that underlie results will be shared in a publication. For the detail, those who are interested in can contact the authors.

    Learn more about this trial

    Risk Stratification-directed Therapy for AML With t(8;21) /AML1-ETO+

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