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
About this trial
This is an interventional treatment trial for Acute Myeloid Leukemia
Eligibility Criteria
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
NCT ID
NCT02936089
First Posted
October 11, 2016
Last Updated
August 6, 2023
Sponsor
Nanfang Hospital, Southern Medical University
Collaborators
Zhujiang Hospital
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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