Acute Myeloid LeukemiaNCT02936089
Risk Stratification-directed Therapy for AML With t(8;21) /AML1-ETO+
A study testing Consolidation with chemotherapy (CT) or autologous hematopoietic stem cell transplantation (auto-HSCT), Consolidation with auto-HSCT or HLA-matched HSCT and allogeneic HSCT in people with Acute Myeloid Leukemia and Risk Stratification, run by Nanfang Hospital, Southern Medical University.
Trial 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. Read more about this study
- Who is it for?
- Ages 14 to 70 years
- Conditions the study is for
- Acute Myeloid Leukemia, Risk Stratification
- What is being tested?
- Consolidation with chemotherapy (CT) or autologous hematopoietic stem cell transplantation (auto-HSCT), Consolidation with auto-HSCT or HLA-matched HSCT and allogeneic HSCT
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 207 participants
- Where
- No sites listed yet
- Study dates
- Oct 2016to 31 Dec 2022
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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)
About this study
Official title: Risk Stratification-directed Therapy for Acute Myeloid Leukemia With t(8;21) /AML1-ETO-positive
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.
Read the 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.
- Conditions
- Acute Myeloid Leukemia, Risk Stratification
- Being tested
- Consolidation with chemotherapy (CT) or autologous hematopoietic stem cell transplantation (auto-HSCT), Consolidation with auto-HSCT or HLA-matched HSCT, allogeneic HSCT
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Consolidation with chemotherapy (CT) or autologous hematopoietic stem cell transplantation (auto-HSCT)Other
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.
Consolidation with auto-HSCT or HLA-matched HSCTOther
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.
allogeneic HSCTOther
For allogeneic HSCT, patients were treated with 1-2 cycles of HDAC and then bridged to allogeneic HSCT, including HLA-matched and haploidentical transplantation.
| Study group | Role | What they receive |
|---|---|---|
| Low risk group | Experimental | Patients with KIT-ASXL1- (non-mutation, NM) and acquiring main molecular response (MMR) after two cycles of consolidation. Receives: Other: Consolidation with chemotherapy (CT) or autologous hematopoietic stem cell transplantation (auto-HSCT) |
| Intermediate risk group | Experimental | Patients with KIT+/ASXL1+ (single mutation, 1M) and acquiring MMR after two cycles of consolidation. Receives: Other: Consolidation with auto-HSCT or HLA-matched HSCT |
| High risk group | Experimental | Patients with KIT+ASXL1+ (two mutations ,2M) or without acquiring MMR after two cycles of consolidation. Receives: Other: allogeneic HSCT |
What is being measured
The primary outcome is the main question the study is designed to answer.
overall survival (OS)
Measured: 3 year
Secondary outcomes (3)
leukemia relapse rate
Measured: 3 year
disease-free survival (DFS)
Measured: 3 year
event Free Survival (EFS)
Measured: 3 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2016 (estimated)
- Study startOct 2016
- Primary completion31 Dec 2021
- Study completion31 Dec 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- 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)
- Enrolment
- 207 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nanfang Hospital, Southern Medical University
- Sponsor type
- University, hospital or other
- Collaborators
- Zhujiang Hospital
- Responsible party
- Sponsor
- Investigators
- Dan Xu, Principal investigator, Nanfang Hospital, Southern Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02936089
- Sponsor's protocol ID
- NFEC-2017-168
- Status last verified
- Aug 2023
- First submitted
- 11 Oct 2016
- Last updated
- 8 Aug 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Publications
Xu D, Yang Y, Yin Z, Tu S, Nie D, Li Y, Huang Z, Sun Q, Huang C, Nie X, Yao Z, Shi P, Zhang Y, Jiang X, Liu Q, Yu G. Risk-directed therapy based on genetics and MRD improves the outcomes of AML1-ETO-positive AML patients, a multi-center prospective cohort study. Blood Cancer J. 2023 Nov 13;13(1):168. doi: 10.1038/s41408-023-00941-4. No abstract available.
PubMed 37957175
Study at a glance
From the registry record, last updated by the sponsor on 8 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Risk Stratification-directed Therapy for Acute Myeloid Leukemia With t(8;21) /AML1-ETO-positive
- ClinicalTrials.gov ID
- NCT02936089
- Acronym / sponsor ID
- NFEC-2017-168
- Conditions
- Acute Myeloid Leukemia, Risk Stratification
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 207 participants (actual)
- Lead sponsor
- Nanfang Hospital, Southern Medical UniversityUniversity, hospital or other
- Collaborators
- Zhujiang Hospital
- Study start
- Oct 2016
- Primary completion
- 31 Dec 2021 (actual)
- Study completion
- 31 Dec 2022 (actual)
- First posted
- 18 Oct 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
- 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.
| Arm | Type | Receives |
|---|---|---|
| Low risk group | Experimental | Other: Consolidation with chemotherapy (CT) or autologous hematopoietic stem cell transplantation (auto-HSCT)Patients with KIT-ASXL1- (non-mutation, NM) and acquiring main molecular response (MMR) after two cycles of consolidation. |
| Intermediate risk group | Experimental | Other: Consolidation with auto-HSCT or HLA-matched HSCTPatients with KIT+/ASXL1+ (single mutation, 1M) and acquiring MMR after two cycles of consolidation. |
| High risk group | Experimental | Other: allogeneic HSCTPatients with KIT+ASXL1+ (two mutations ,2M) or without acquiring MMR after two cycles of consolidation. |
Who may qualify
- Ages
- 14 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (2 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Consolidation with chemotherapy (CT) or autologous hematopoietic stem cell transplantation (auto-HSCT)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. | Other | Low risk group |
| Consolidation with auto-HSCT or HLA-matched HSCTFor 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. | Other | Intermediate risk group |
| allogeneic HSCTFor allogeneic HSCT, patients were treated with 1-2 cycles of HDAC and then bridged to allogeneic HSCT, including HLA-matched and haploidentical transplantation. | Other | High risk group |
Primary outcome
overall survival (OS)
Time frame: 3 year
3 secondary outcomes
leukemia relapse rate
Time frame: 3 year
disease-free survival (DFS)
Time frame: 3 year
event Free Survival (EFS)
Time frame: 3 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Aug 2023.
- Derived from this study: PubMed 37957175Xu D, Yang Y, Yin Z, Tu S, Nie D, Li Y, Huang Z, Sun Q, Huang C, Nie X, Yao Z, Shi P, Zhang Y, Jiang X, Liu Q, Yu G. Risk-directed therapy based on genetics and MRD improves the outcomes of AML1-ETO-positive AML patients, a multi-center prospective cohort study. Blood Cancer J. 2023 Nov 13;13(1):168. doi: 10.1038/s41408-023-00941-4. No abstract available.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Dan Xu principal investigator
Nanfang Hospital, Southern Medical University
Questions about this trial
What is this clinical trial studying?
A study testing Consolidation with chemotherapy (CT) or autologous hematopoietic stem cell transplantation (auto-HSCT), Consolidation with auto-HSCT or HLA-matched HSCT and allogeneic HSCT in people with Acute Myeloid Leukemia and Risk Stratification, run by Nanfang Hospital, Southern Medical University.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
overall survival (OS) (time frame: 3 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02936089, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Aug 2023.