CEBPA Double MutationPhase 2NCT04415008
Efficacy of HAD Induction With Intensified Cytarabine in Newly-diagnosed CEBPA Double Mutated Acute Myeloid Leukemia
A Phase 2 study testing HAD induction with intermediate dose cytarabine in people with CEBPA Double Mutation and Acute Myeloid Leukemia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
Acute myeloid leukemia is a heterogenous hematological malignancy, characterized by different cytogenetic or molecular features. CEBPA double mutation acute myeloid leukemia （CEBPAdm AML）has favourite prognosis, especially in younger adult patients. Read more about this study
- Who is it for?
- Ages 14 to 55 years
- Conditions the study is for
- CEBPA Double Mutation, Acute Myeloid Leukemia
- What is being tested?
- HAD induction with intermediate dose cytarabine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 61 participants
- Where
- 1 siteChina
- Study dates
- 1 Jun 2020to 1 Jun 2027
- Phase
- Phase 2
- 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 55 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- newly-diagnosed of acute myeloid leukemia except acute promyelocytic leukemia
- with CEBPA double mutation
- age≥ 14 years and<55 years，male or female
- ECOG-PS score 0-2
- laboratory tests（within 7 days before chemotherapy）
- serum total bilirubin≤1.5xULN；
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- serum AST and ALT≤2.5xULN
- serum creatinine≤2xULN；
- cardiac enzymes≤2xULN
- ejection fraction >50% by ECHO。
- written informed consent。
Who cannot join
- subject has received remission induction chemotherapy
- secondary AML
- with other hematological malignancy
- with other tumors(needing treatment)
- pregnant or lactating women
- active heart diseases
- severe active infection
- unfit for enrollment evaluated by investigator
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Prospective, Multicenter, Single Arm Clinical Study to Evaluate Efficacy of HAD Induction With Intensified Cytarabine in Newly-diagnosed CEBPA Double Mutated Acute Myeloid Leukemia
Acute myeloid leukemia is a heterogenous hematological malignancy, characterized by different cytogenetic or molecular features. CEBPA double mutation acute myeloid leukemia （CEBPAdm AML）has favourite prognosis, especially in younger adult patients. But cumulative incidence of relapse of this group patients is still high, so the treatment options need to be optimized urgently.HAD(homoharringtonine(HHT)+cytarabine+daunorubicin) with intermediate dose cytarabine improved the survival of AML, especially in patients with CEBPA double mutation.
Read the detailed description
In this phase 2 study, 40 patients will be enrolled and treated with HAD induction regimen. High dose cytarabine will be given after complete remission achieved. The primary endpoint was event-free and relapse-free survival. Genetic mutations and measurable residual disease (MRD) will be detected at diagnosis and after chemotherapy. The risk stratification according to genetic mutations and MRD will also be explored.
induction chemotherapy:HHT 2mg/m2/d,day 1-7 cytarabine 100mg/m2/d, day1-4；1g/m2 /q12h ，day5-7 DNR 40mg/m2/d，day 1-3 re-induction chemotherapy:IDA 10mg/m2，day 1-3 cytarabine 100mg/m2，day 1-7 CTX 350mg/m2，day 2，day 5 consolidation chemotherapy: high dose cytarabine 3g/m2/Q12h,day1-3,for three cycles
- Conditions
- CEBPA Double Mutation, Acute Myeloid Leukemia
- Being tested
- HAD induction with intermediate dose cytarabine
- 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.
HAD induction with intermediate dose cytarabineDrug
daunorubicin,cytarabine,homoharringtonine
| Study group | Role | What they receive |
|---|---|---|
| treatment arm | Experimental | prospective, open-label, multicenter,single arm Receives: Drug: HAD induction with intermediate dose cytarabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
event free survival
Measured from day 1 of randomization to the date of treatment failure, hematologic relapse from CR or death from any cause, whichever occurs first
Measured: 5 years
relapse free survival
survival from complete remission to relapse
Measured: 5 years
Secondary outcomes (4)
complete remission rate
incidence of complete remission after induction chemotherapy
Measured: 2.5 years
early mortality
death during 30 days from induction chemotherapy
Measured: 2.5 years
overall survival
duration from enrollment to death or loss of followup
Measured: 5 years
RFS cencored at stem cell transplantation
RFS cencored at the date of stem cell transplantation
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jun 2020
- Study start1 Jun 2020
- Primary completion3 Oct 2026 (estimated)
- Study completion1 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CEBPA Double MutationStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 61 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04415008
- Sponsor's protocol ID
- IIT2020008
- Status last verified
- May 2025
- First submitted
- 31 May 2020
- Last updated
- 3 Jun 2025
- 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 3 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Multicenter, Single Arm Clinical Study to Evaluate Efficacy of HAD Induction With Intensified Cytarabine in Newly-diagnosed CEBPA Double Mutated Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT04415008
- Acronym / sponsor ID
- IIT2020008
- Conditions
- CEBPA Double Mutation, Acute Myeloid Leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 61 participants (actual)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 1 Jun 2020 (actual)
- Primary completion
- 3 Oct 2026 (estimated)
- Study completion
- 1 Jun 2027 (estimated)
- First posted
- 4 Jun 2020 (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.
Acute myeloid leukemia is a heterogenous hematological malignancy, characterized by different cytogenetic or molecular features. CEBPA double mutation acute myeloid leukemia （CEBPAdm AML）has favourite prognosis, especially in younger adult patients. But cumulative incidence of relapse of this group patients is still high, so the treatment options need to be optimized urgently.HAD(homoharringtonine(HHT)+cytarabine+daunorubicin) with intermediate dose cytarabine improved the survival of AML, especially in patients with CEBPA double mutation.
Detailed description
In this phase 2 study, 40 patients will be enrolled and treated with HAD induction regimen. High dose cytarabine will be given after complete remission achieved. The primary endpoint was event-free and relapse-free survival. Genetic mutations and measurable residual disease (MRD) will be detected at diagnosis and after chemotherapy. The risk stratification according to genetic mutations and MRD will also be explored.
induction chemotherapy:HHT 2mg/m2/d,day 1-7 cytarabine 100mg/m2/d, day1-4；1g/m2 /q12h ，day5-7 DNR 40mg/m2/d，day 1-3 re-induction chemotherapy:IDA 10mg/m2，day 1-3 cytarabine 100mg/m2，day 1-7 CTX 350mg/m2，day 2，day 5 consolidation chemotherapy: high dose cytarabine 3g/m2/Q12h,day1-3,for three cycles
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| treatment arm | Experimental | Drug: HAD induction with intermediate dose cytarabineprospective, open-label, multicenter,single arm |
Who may qualify
- Ages
- 14 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- newly-diagnosed of acute myeloid leukemia except acute promyelocytic leukemia
- with CEBPA double mutation
- age≥ 14 years and<55 years，male or female
- ECOG-PS score 0-2
- laboratory tests（within 7 days before chemotherapy）
Key exclusion criteria (8 listed)
- subject has received remission induction chemotherapy
- secondary AML
- with other hematological malignancy
- with other tumors(needing treatment)
- pregnant or lactating women
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 |
|---|---|---|
| HAD induction with intermediate dose cytarabinedaunorubicin,cytarabine,homoharringtonine | Drug | treatment arm |
Primary outcome
event free survival
Time frame: 5 years
Measured from day 1 of randomization to the date of treatment failure, hematologic relapse from CR or death from any cause, whichever occurs first
relapse free survival
Time frame: 5 years
survival from complete remission to relapse
4 secondary outcomes
complete remission rate
Time frame: 2.5 years
early mortality
Time frame: 2.5 years
overall survival
Time frame: 5 years
RFS cencored at stem cell transplantation
Time frame: 5 years
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 3 Jun 2025.
- Individual participant dataSharing planned: No
Related trials for CEBPA Double Mutation
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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing HAD induction with intermediate dose cytarabine in people with CEBPA Double Mutation and Acute Myeloid Leukemia, 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 11 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: under way, not looking for participants, as of the sponsor's last update on 3 Jun 2025. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
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?
event free survival (time frame: 5 years). The study lists 2 primary outcomes in all. 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 3 Jun 2025.