AMLPhase 3NCT06744504
Standard-dose vs Intermediate-dose Cytarabine Induction in the Treatment of Acute Myeloid Leukemia With RUNX1-RUNX1T1
A Phase 3 study testing cytarabine, daunorubicin and idarubicin in people with AML and RUNX1-RUNX1T1 Fusion Protein Expression, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
Leukemia is one of the common malignant tumors that threaten human health. Although the efficacy of AML treatment has improved significantly in recent years, it remains one of the major diseases threatening human health. Current research on AML treatment mainly has two directions. Read more about this study
- Who is it for?
- Ages 14 to 60 years
- Conditions the study is for
- AML, RUNX1-RUNX1T1 Fusion Protein Expression
- What is being tested?
- cytarabine, daunorubicin and idarubicin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 284 participants
- Where
- 1 siteChina
- Study dates
- 10 Jan 2025to 1 Dec 2029
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- AML conforming to WHO (2022) or ICC standards
- Possessing the RUNX1::RUNX1T1 fusion gene
- Age ranging from 14 to 60 years old, regardless of gender.
- The performance status assessment of the Eastern Cooperative Oncology Group (ECOG-PS) being 0 - 2.
- Meeting the requirements of the following laboratory examination indicators (conducted within 7 days before treatment):
- 1) Total bilirubin ≤ 1.5 times the upper limit of the normal value for the same age group; 2) AST and ALT ≤ 2.5 times the upper limit of the normal value for the same age group; 3) Serum creatinine < 2 times the upper limit of the normal value for the same age group; 4) Cardiac enzymes < 2 times the upper limit of the normal value for the same age group; 5) The cardiac ejection fraction determined by echocardiography (ECHO) > 50%. An informed consent form must be signed before the commencement of all specific research procedures, either by the patient themselves or their immediate relatives. Considering the patient's condition, if the patient's signature is not conducive to the treatment of the disease, the informed consent form shall be signed by the legal guardian or the immediate relatives of the patient.
Who cannot join
- Acute promyelocytic leukemia accompanied by PML-RARA fusion gene.
- Acute myeloid leukemia featuring BCR-ABL fusion gene.
- Patients undergoing retreatment (but can receive cytoreductive therapy with hydroxyurea and cytarabine).
- Individuals concurrently having malignant tumors in other organs (requiring treatment).
- Active cardiac disorders, defined as one or more of the following:
- 1) A history of uncontrolled or symptomatic angina pectoris; 2) Myocardial infarction less than 6 months from study enrollment; 3) A history of significant arrhythmia requiring medication or presenting with severe clinical symptoms; 4) Uncontrolled or symptomatic congestive heart failure (> NYHA Class 2)
- 6. Severe infectious diseases (untreated tuberculosis, pulmonary aspergillosis).
- 7. Individuals deemed ineligible for enrollment by the investigator.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Blood Diseases Hospital
Tianjin, Tianjin Municipality, 300020
hui wei, MD, 86-13132507161, weihui@ihcams.ac.cn
About this study
Official title: Anthracycline-based Standard-dose vs Intermediate-dose Cytarabine Induction in the Treatment of Acute Myeloid Leukemia With RUNX1-RUNX1T1: a Prospective, Randomized, Controlled Phase III Clinical Trial
Leukemia is one of the common malignant tumors that threaten human health. Although the efficacy of AML treatment has improved significantly in recent years, it remains one of the major diseases threatening human health. Current research on AML treatment mainly has two directions. One is the addition of new targeted therapy drugs, and the other research direction is to enhance the intensity of AML chemotherapy, including the use of large doses of anthracycline drugs or the use of high-dose cytarabine treatment.
Since the 1990s, induction remission has been achieved by using anthracyclines in combination with high-dose cytarabine. The ECOG (Eastern Cooperative Oncology Group) contends that high-dose induction chemotherapy fails to enhance the bone marrow remission rate but elevates the chemotherapy-related mortality rate. Bradstock and the Australian Group also noted that although it does not increase the bone marrow remission rate, it can result in longer survival time and disease-free survival time. The clinical study from EORTC-GIMEMA AML-12 discovered that AML patients under the age of 45 could benefit from induction therapy incorporating high-dose cytarabine. In our previous randomized controlled clinical trials, it was found that the HAD and DA regimens containing intermediate-dose cytarabine could enhance the complete remission rate and improve the overall survival of adult AML. However, the degree of benefit varies among different AML subgroups.
The abnormalities of RUNX1-RUNX1T1 and CBFβ-MYH11 respectively involve a subunit of CBF (core binding factor), thus the two are collectively called CBF leukemia. Previous retrospective studies show that this type of leukemia benefits from intensified treatment regimens such as FLAG. However, at present, there is a lack of prospective randomized controlled clinical studies to confirm this. Therefore, in this study, we intend to further verify through a prospective randomized controlled clinical trial whether the induction treatment regimen containing intermediate-dose cytarabine can improve the long-term efficacy of adult RUNX1-RUNX1T1 acute myeloid leukemia.
Read the detailed description
This study is a prospective, randomized, controlled phase III clinical trial that plans to enroll adult patients with AML who meet the WHO (2022) or ICC criteria for eligibility for intensive chemotherapy with RUNX1-RUNX1 fusion. For patients who meet the inclusion criteria and do not meet any exclusion criteria, they will be randomly assigned to either the A group, which receives standard-dose DA induction therapy, or the B group, which receives intermediate-dose DA induction therapy. Patients who do not achieve complete remission after one cycle of induction therapy will receive IAC reinduction therapy. Patients who achieve complete remission after one or two cycles of induction therapy will proceed to receive three cycles of high-dose cytarabine consolidation therapy. Patients who do not achieve complete remission after two cycles of induction therapy will be withdrawn from the treatment protocol. For patients whose fusion gene levels do not meet the criteria, allogeneic hematopoietic stem cell transplantation is recommended.
- Conditions
- AML, RUNX1-RUNX1T1 Fusion Protein Expression
- Being tested
- cytarabine, daunorubicin, idarubicin, cyclophosphamide
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
cytarabineDrug
Cytarabine combined with daunorubicin was used for induction therapy. According to the different doses of cytarabine, it was divided into standard dose group and intermediate dose group.In addition, high doses of cytarabine are also used for post-remission treatment.
daunorubicinDrug
combined with cytarabine and used for induction therapy
idarubicinDrug
combined with cytarabine and cyclophosphamide and used for post-remission treatment
cyclophosphamideDrug
combined with cytarabine and idarubicin and used for post-remission treatment
| Study group | Role | What they receive |
|---|---|---|
| standard-dose Cytarabine | Active comparator | Induction therapy:
Cytarabine (Ara-c) 100mg/ m2/ day, day 1-7; Daunorubicin (DNR) 60mg/m2/ day, day 1-3; Those who did not meet CR were treated with IAC regimen.
IAC:
IDA 8 mg/m2/d, D1-3; Ara-c 100 mg/m2, D1-7; CTX 350mg/m2, D2,5;
Post-remission treatment options:
High dose Ara-C(HDAC) : 3 cycles Ara-C 3g/㎡/q12h, days 1, 3 and 5 For patietns RUNX1-RUNX1 MRD reduction < 3 logs after 2 courses, allo-transplantation is recommended if donor is available. Receives: Drug: cytarabine; Drug: daunorubicin; Drug: idarubicin; Drug: cyclophosphamide |
| Intermediate-dose Cytarabine | Experimental | Induction therapy:
Cytarabine (Ara-c) 100mg/ m2/ day, day 1-4; 1g/㎡/q12h, day 5-7; Daunorubicin (DNR) 60mg/m2/ day, day 1-3; Those who did not meet CR were treated with IAC regimen.
IAC:
IDA 8 mg/m2/d, D1-3; Ara-c 100 mg/m2, D1-7; CTX 350mg/m2, D2,5;
Post-remission treatment options:
High dose Ara-C(HDAC) : 3 cycles; Ara-C 3g/㎡/q12h, days 1, 3 and 5. For patietns RUNX1-RUNX1 MRD reduction < 3 logs after 2 courses, allo-transplantation is recommended if donor is available. Receives: Drug: cytarabine; Drug: daunorubicin; Drug: idarubicin; Drug: cyclophosphamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
overall survival
Used to evaluate all patients who enter clinical trials. From the date of entry into the trial until the date of patient death (including any cause) or last survival follow-up.
Measured: up to 2 years after the date of the last enrolled participants
Secondary outcomes (8)
Complete remission (CR)/CR with partial hematologic recovery (CRh)/CR with incomplete hematologic recovery (CRi) rate after induction therapy
The ratio of patients achieved CR/CRh/CRi after induction therapy.
Measured: up to 3 months after the date of the last enrolled participants
RUNX1::RUNX1T1 minimal residual disease (MRD) reduction >3 logs after 2 courses
RUNX1::RUNX1T1 MRD is measured by real-time PCR.
Measured: up to 2 years after the date of the last enrolled participants
RUNX1::RUNX1T1 molecular MRD undectable rate
MRD levels monitored by polymerase chain reaction are associated with outcomes in acute myeloid leukemia with RUNX1-RUNX1T1.
Measured: up to 2 years after the date of the last enrolled participants
Relapse-free survival (RFS)
Defined only for patients achieving CRc; measured from the date of achievement of remission until the date of hematologic relapse or death from any cause; patients not known to have relapsed or died at last follow-up are censored on the date they were last known to be alive
Measured: up to 2 years after the date of the last enrolled participants
Event-free survival (EFS)
All patients definitions for the trial; From the date of enrollment to the time of treatment failure after two courses of induction therapy, recurrence after CRc, date of all-cause death, or the date of last survival follow-up.
Measured: up to 2 years after the date of the last enrolled participants
30-day mortality
Percentage of patients who died within 30 days from enrollment.
Measured: within 30 days of the date of the last enrolled participants
60-day mortality
Percentage of patients who died within 60 days from enrollment
Measured: within 60 days of the date of the last enrolled participants
Complete remission (CR)/CR with partial hematologic recovery (CRh)/CR with incomplete hematologic recovery (CRi) rate
the ratio of patients achieved CR/CRh/CRi
Measured: up to12 months after the date of the last enrolled participants
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Dec 2024
- Study start10 Jan 2025
- Primary completion1 Dec 2027 (estimated)
- Study completion1 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AMLStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe sponsor's other registered studies.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 284 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06744504
- Sponsor's protocol ID
- IIT2024094
- Status last verified
- Aug 2026
- First submitted
- 17 Dec 2024
- Last updated
- 7 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Anthracycline-based Standard-dose vs Intermediate-dose Cytarabine Induction in the Treatment of Acute Myeloid Leukemia With RUNX1-RUNX1T1: a Prospective, Randomized, Controlled Phase III Clinical Trial
- ClinicalTrials.gov ID
- NCT06744504
- Acronym / sponsor ID
- IIT2024094
- Conditions
- AML, RUNX1-RUNX1T1 Fusion Protein Expression
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 284 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 10 Jan 2025 (actual)
- Primary completion
- 1 Dec 2027 (estimated)
- Study completion
- 1 Dec 2029 (estimated)
- First posted
- 20 Dec 2024 (actual)
- 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.
Leukemia is one of the common malignant tumors that threaten human health. Although the efficacy of AML treatment has improved significantly in recent years, it remains one of the major diseases threatening human health. Current research on AML treatment mainly has two directions. One is the addition of new targeted therapy drugs, and the other research direction is to enhance the intensity of AML chemotherapy, including the use of large doses of anthracycline drugs or the use of high-dose cytarabine treatment.
Since the 1990s, induction remission has been achieved by using anthracyclines in combination with high-dose cytarabine. The ECOG (Eastern Cooperative Oncology Group) contends that high-dose induction chemotherapy fails to enhance the bone marrow remission rate but elevates the chemotherapy-related mortality rate. Bradstock and the Australian Group also noted that although it does not increase the bone marrow remission rate, it can result in longer survival time and disease-free survival time. The clinical study from EORTC-GIMEMA AML-12 discovered that AML patients under the age of 45 could benefit from induction therapy incorporating high-dose cytarabine. In our previous randomized controlled clinical trials, it was found that the HAD and DA regimens containing intermediate-dose cytarabine could enhance the complete remission rate and improve the overall survival of adult AML. However, the degree of benefit varies among different AML subgroups.
The abnormalities of RUNX1-RUNX1T1 and CBFβ-MYH11 respectively involve a subunit of CBF (core binding factor), thus the two are collectively called CBF leukemia. Previous retrospective studies show that this type of leukemia benefits from intensified treatment regimens such as FLAG. However, at present, there is a lack of prospective randomized controlled clinical studies to confirm this. Therefore, in this study, we intend to further verify through a prospective randomized controlled clinical trial whether the induction treatment regimen containing intermediate-dose cytarabine can improve the long-term efficacy of adult RUNX1-RUNX1T1 acute myeloid leukemia.
Detailed description
This study is a prospective, randomized, controlled phase III clinical trial that plans to enroll adult patients with AML who meet the WHO (2022) or ICC criteria for eligibility for intensive chemotherapy with RUNX1-RUNX1 fusion. For patients who meet the inclusion criteria and do not meet any exclusion criteria, they will be randomly assigned to either the A group, which receives standard-dose DA induction therapy, or the B group, which receives intermediate-dose DA induction therapy. Patients who do not achieve complete remission after one cycle of induction therapy will receive IAC reinduction therapy. Patients who achieve complete remission after one or two cycles of induction therapy will proceed to receive three cycles of high-dose cytarabine consolidation therapy. Patients who do not achieve complete remission after two cycles of induction therapy will be withdrawn from the treatment protocol. For patients whose fusion gene levels do not meet the criteria, allogeneic hematopoietic stem cell transplantation is recommended.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| standard-dose Cytarabine | Active comparator | Drug: cytarabine, Drug: daunorubicin, Drug: idarubicin, Drug: cyclophosphamideInduction therapy: Cytarabine (Ara-c) 100mg/ m2/ day, day 1-7; Daunorubicin (DNR) 60mg/m2/ day, day 1-3; Those who did not meet CR were treated with IAC regimen. IAC: IDA 8 mg/m2/d, D1-3; Ara-c 100 mg/m2, D1-7; CTX 350mg/m2, D2,5; Post-remission treatment options: High dose Ara-C(HDAC) : 3 cycles Ara-C 3g/㎡/q12h, days 1, 3 and 5 For patietns RUNX1-RUNX1 MRD reduction < 3 logs after 2 courses, allo-transplantation is recommended if donor is available. |
| Intermediate-dose Cytarabine | Experimental | Drug: cytarabine, Drug: daunorubicin, Drug: idarubicin, Drug: cyclophosphamideInduction therapy: Cytarabine (Ara-c) 100mg/ m2/ day, day 1-4; 1g/㎡/q12h, day 5-7; Daunorubicin (DNR) 60mg/m2/ day, day 1-3; Those who did not meet CR were treated with IAC regimen. IAC: IDA 8 mg/m2/d, D1-3; Ara-c 100 mg/m2, D1-7; CTX 350mg/m2, D2,5; Post-remission treatment options: High dose Ara-C(HDAC) : 3 cycles; Ara-C 3g/㎡/q12h, days 1, 3 and 5. For patietns RUNX1-RUNX1 MRD reduction < 3 logs after 2 courses, allo-transplantation is recommended if donor is available. |
Who may qualify
- Ages
- 14 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- AML conforming to WHO (2022) or ICC standards
- Possessing the RUNX1::RUNX1T1 fusion gene
- Age ranging from 14 to 60 years old, regardless of gender.
- The performance status assessment of the Eastern Cooperative Oncology Group (ECOG-PS) being 0 - 2.
- Meeting the requirements of the following laboratory examination indicators (conducted within 7 days before treatment):
Key exclusion criteria (8 listed)
- Acute promyelocytic leukemia accompanied by PML-RARA fusion gene.
- Acute myeloid leukemia featuring BCR-ABL fusion gene.
- Patients undergoing retreatment (but can receive cytoreductive therapy with hydroxyurea and cytarabine).
- Individuals concurrently having malignant tumors in other organs (requiring treatment).
- Active cardiac disorders, defined as one or more of the following:
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 |
|---|---|---|
| cytarabineCytarabine combined with daunorubicin was used for induction therapy. According to the different doses of cytarabine, it was divided into standard dose group and intermediate dose group.In addition, high doses of cytarabine are also used for post-remission treatment. | Drug | Intermediate-dose Cytarabine, standard-dose Cytarabine |
| daunorubicincombined with cytarabine and used for induction therapy | Drug | Intermediate-dose Cytarabine, standard-dose Cytarabine |
| idarubicincombined with cytarabine and cyclophosphamide and used for post-remission treatment | Drug | Intermediate-dose Cytarabine, standard-dose Cytarabine |
| cyclophosphamidecombined with cytarabine and idarubicin and used for post-remission treatment | Drug | Intermediate-dose Cytarabine, standard-dose Cytarabine |
Primary outcome
overall survival
Time frame: up to 2 years after the date of the last enrolled participants
Used to evaluate all patients who enter clinical trials. From the date of entry into the trial until the date of patient death (including any cause) or last survival follow-up.
8 secondary outcomes
Complete remission (CR)/CR with partial hematologic recovery (CRh)/CR with incomplete hematologic recovery (CRi) rate after induction therapy
Time frame: up to 3 months after the date of the last enrolled participants
RUNX1::RUNX1T1 minimal residual disease (MRD) reduction >3 logs after 2 courses
Time frame: up to 2 years after the date of the last enrolled participants
RUNX1::RUNX1T1 molecular MRD undectable rate
Time frame: up to 2 years after the date of the last enrolled participants
Relapse-free survival (RFS)
Time frame: up to 2 years after the date of the last enrolled participants
Event-free survival (EFS)
Time frame: up to 2 years after the date of the last enrolled participants
30-day mortality
Time frame: within 30 days of the date of the last enrolled participants
60-day mortality
Time frame: within 60 days of the date of the last enrolled participants
Complete remission (CR)/CR with partial hematologic recovery (CRh)/CR with incomplete hematologic recovery (CRi) rate
Time frame: up to12 months after the date of the last enrolled participants
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2026.
- Individual participant dataSharing planned: No
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing cytarabine, daunorubicin and idarubicin in people with AML and RUNX1-RUNX1T1 Fusion Protein Expression, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
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 6 inclusion and 8 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: looking for participants now, as of the sponsor's last update on 7 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Hui Wei, MD (13132507161, weihui@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
overall survival (time frame: up to 2 years after the date of the last enrolled participants). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 7 Aug 2026.