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AMLHADCEBPA2023NCT06529250

Intermediate-dose HAD Regimen for CEBPA Double-mutated AML

A study testing HAD and Daunorubicin+Cytarabine in people with AML, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

AML is highly heterogeneous in pathogenesis, and CEBPA double-mutated (CEBPAdm) AML is a common type of leukemia in China. Currently, no targeted therapies for CEBPAdm, and chemotherapy and transplantation are still the treatment options for CEBPA double-mutated AML. Read more about this study

Who is it for?
Ages 14 to 54 years
Conditions the study is for
AML
What is being tested?
HAD and Daunorubicin+Cytarabine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
148 participants
Where
1 siteChina
Study dates
13 Aug 2024to 1 Sep 2029
Phase
Interventional
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 54 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • AML diagnosed according to WHO-2022 classification with recurrent CEBPA mutations and containing mutation in the bZIP domain.
  • Older than 14 years old and younger than 55 years old
  • Male or female.
  • The Eastern Cooperative Oncology Group Performance Status (ECOG-PS) of AML patients were 0-2 points.
  • Meet the following laboratory tests (performed within 7 days prior to treatment) 1) Total bilirubin ≤ 1.5 times of the upper limit of normal value (same age); 2) AST and ALT≤ 2.5 times of the upper limit of normal value (same age); 3) Blood creatinine < 2 times of the upper limit of normal value (same age); 4) Myocardial enzymes < 2 times of the upper limit of normal value (same age); 5) Echocardiography (ECHO) was performed to determine the ejection fraction of the heart within the normal range.

Who cannot join

  • Patients who have previously received induction chemotherapy, regardless of efficacy.
  • Simultaneously suffering from malignant tumors of other organs and requiring treatment).
  • Pregnant or lactating women. Male or female patients participating in the trial must take contraceptive measures during the trial treatment period.
  • Active heart disease, defined as one or more of the following:1) Have a history of uncontrolled or symptomatic angina pectoris;2) Myocardial infarction less than 6 months prior to enrollment in the study;3) A history of arrhythmia requiring medication treatment or severe clinical symptoms;4) Uncontrolled or symptomatic congestive heart failure (> NYHA grade 2);5) The ejection fraction is below the lower limit of the normal range.
  • Serious infectious diseases (uncured tuberculosis, pulmonary aspergillosis).
  • Those who were not considered suitable for inclusion by the researchers.

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
  • Blood Hospital

    Tianjin, Tianjin Municipality, 300020

    hui wei, MD, 86-13132507161, weihui@ihcams.ac.cn

    Hui Wei, MD

    Recruiting

Other trials in China

About this study

Official title: A Multicenter, Randomized, Controlled Clinical Trial of Intermediate-dose HAD Regimen for CEBPA Double-mutated Acute Myeloid Leukemia

AML is highly heterogeneous in pathogenesis, and CEBPA double-mutated (CEBPAdm) AML is a common type of leukemia in China. Currently, no targeted therapies for CEBPAdm, and chemotherapy and transplantation are still the treatment options for CEBPA double-mutated AML. At present, the "3+7" treatment induction regimen of cytarabine combined with anthracyclines is still the first-line recommended regimen. In our retrospective study, the intermediate dose HAD regimen produced a 3-year RFS of 84.7% and a 3-year OS of 92.8% in CEBPAdm AML. Therefore, this project intends to confirm the efficacy of intermediate-dose HAD in the treatment of CEBPA double-mutated AML is superior to the conventional treatment regimen through the multi-center RCT study.

Read the detailed description

This is a prospective, randomized, controlled clinical trial of patients diagnosed with CEBPA double-mutated AML. Patients who meet the inclusion criteria are randomly assigned to receive the intermediate-dose HAD regimen or the conventional 3+7 induction regimen (IA or DA), respectively. When patients reach complete remission (CR) after the induction therapy, 3 courses of the high-dose cytarabine regimen (3g/m2 q12h, 3 days) are used. Hematopoietic stem cell transplantation is recommended for patients with persistent MRD positive after treatment. When patients do not achieve CR after the induction therapy, reinduction therapy with IAC (IDA 10mg/ m2 for 3 days, cytarabine 100mg/m2 for 7 days, cyclophosphamide 350mg/m2 d2, d5) regimen is used. Patients who still do not achieve CR after reinduction therapy will be removed from the group.

Conditions
AML
Being tested
HAD, Daunorubicin+Cytarabine
Keywords
AML, CEBPA double-mutated, treatment

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • HADDrug

    Also called Homoharringtonine, Cytarabine, Daunorubicin

    Induction therapy:Homoharringtonine: 2mg/㎡/d, days 1-7 Cytarabine: (Ara-c 100mg/㎡/d, day 1-4; 1g/㎡ /q12h, day 5-7), Daunorubicin: (DNR 40mg/㎡/d, day 1-3).

    Reinduction therapy:

    Idarubicin (IDA) 10mg/㎡ for d1-3 , Ara-c 100mg/㎡ d1-7 , Cyclophosphamide (CTX350mg/㎡ d2, d5) . Patients who did not achieve CR after reinduction therapy were removed from the group.

    After achieving CR, they received high-dose cytarabine (3g/m2 q12h, 3 days) regimen for consolidation for 3 courses. Hematopoietic stem cell transplantation is recommended for patients with persistent MRD positive after treatment.

  • Daunorubicin+CytarabineDrug

    Also called Cytarabine, Daunorubicin

    Cytarabine: (Ara-c 100mg/㎡/d, day 1-7), Daunorubicin: (DNR 60mg/㎡/d, day 1-3) or idarubicin (IDA 12mg/㎡/d, day 1-3).

    Treatment did not achieve CR, and reinduction of IAC regimen was given.

    Reinduction therapy:

    Idarubicin (IDA) 10mg/㎡ ,d1-3, Ara-c 100mg/㎡ d1-7 , Cyclophosphamide (CTX350mg/㎡ d2, d5). Patients who did not achieve CR after reinduction therapy were removed from the group.

    After achieving CR, they received high-dose cytarabine (3g/m2 q12h, 3 days) regimen for consolidation for 3 courses. Hematopoietic stem cell transplantation is recommended for patients with persistent MRD positive after treatment.

Study groups in NCT06529250
Study groupRoleWhat they receive
intermediate-dose HAD regimenExperimentalPatients received intermediate-dose HAD regimen. When patients reach complete remission (CR) after the induction therapy, 3 courses of the high-dose cytarabine regimen (3g/m2 q12h, 3 days) are used. Hematopoietic stem cell transplantation is recommended for patients with persistent MRD positive after treatment. When patients do not achieve CR after the induction therapy, reinduction therapy with IAC (IDA 10mg/m2 for 3 days, cytarabine 100mg/m2 for 7 days, cyclophosphamide 350mg/m2 d2, d5) regimen is used. Patients who still do not achieve CR after reinduction therapy will be removed from the group.
Receives: Drug: HAD
conventional treatmentActive comparatorPatients were treated with 3+7 induction regimen (IA or DA). When patients reach complete remission (CR) after the induction therapy, 3 courses of the high-dose cytarabine regimen (3g/m2 q12h, 3 days) are used. Hematopoietic stem cell transplantation is recommended for patients with persistent MRD positive after treatment. When patients do not achieve CR after the induction therapy, reinduction therapy with IAC (IDA 10mg/m2 for 3 days, cytarabine 100mg/m2 for 7 days, cyclophosphamide 350mg/ m2 d2, d5) regimen is used. Patients who still do not achieve CR after reinduction therapy will be removed from the group.
Receives: Drug: Daunorubicin+Cytarabine

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Event-free survival (EFS)

    The interval from randomization to assessment of response after the second course of chemotherapy treatment if patients failed to achieve CR after two courses of induction therapy, the date of relapse, or the date of death, whichever occurred first.

    Measured: up to 2 years after the date of the last enrolled participants

Secondary outcomes (8)
  1. Complete response rate (CR)

    The proportion that reaches CR status after two courses of induction therapy. Patients should be morphologically free of leukemia, and free of extramedullary leukemia. Absolute neutrophil counts was greater than 1.0*10^9/L, and platelet counts was greater than 100*10^9/L.

    Measured: Six weeks after induction therapy

  2. 30-day mortality

    Percentage of patients who died within 30 days from randomization

    Measured: Within 30 days of randomization

  3. overall survival

    The interval from the date of randomization to the date of death or the date of last follow-up for surviving patients.

    Measured: up to 2 years after the date of the last enrolled participants

  4. Event-free survival censored at hematopoietic stem cell transplantation

    The interval from randomization to assessment of response after the second course of chemotherapy treatment if patients failed to achieve CR after two courses of induction therapy, the date of relapse, or the date of death, or the date of last follow-up, or the date of hematopoietic stem cell transplantation, whichever occurred first.

    Measured: up to 2 years after the date of the last enrolled participants

  5. Relapse free survival censored at hematopoietic stem cell transplantation

    The interval from CR to the date of relapse, or the date of death, or the date of last follow-up, or the date of hematopoietic stem cell transplantation, whichever occurred first. This outcome analyze patients achieved CR in two courses induction therapy.

    Measured: up to 2 years after the date of the last enrolled participants

  6. overall survival censored at hematopoietic stem cell transplantation

    It is defined as the time from randomization to the date of death or the date of last follow-up, or the date of hematopoietic stem cell transplantation, whichever occurred first.

    Measured: up to 2 years after the date of the last enrolled participants

  7. 60-day mortality

    Percentage of patients who died within 60 days from randomization

    Measured: Within 60 days of randomization

  8. Relapse free survival(RFS)

    The interval from CR to the date of relapse, or the date of death, or the date of last follow-up, whichever occurred first. This outcome analyze patients achieved CR in two courses induction therapy.

    Measured: up to 2 years 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.

  1. First posted31 Jul 2024
  2. Study start13 Aug 2024
  3. Primary completion30 Aug 2027 (estimated)
  4. Study completion1 Sep 2029 (estimated)

Resources

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
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
148 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06529250
Sponsor's protocol ID
IIT2023005
Acronym
HADCEBPA2023
Status last verified
Aug 2026
First submitted
17 Jul 2024
Last updated
10 Aug 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Wei H, Wang Y, Zhou C, Lin D, Liu B, Liu K, Qiu S, Gong B, Li Y, Zhang G, Wei S, Gong X, Liu Y, Zhao X, Gu R, Mi Y, Wang J. Distinct genetic alteration profiles of acute myeloid leukemia between Caucasian and Eastern Asian population. J Hematol Oncol. 2018 Feb 10;11(1):18. doi: 10.1186/s13045-018-0566-8.

    PubMed 29427994
  • Wei H, Zhou C, Liu B, Lin D, Li Y, Wei S, Gong B, Zhang G, Liu K, Gong X, Fang Q, Liu Y, Qiu S, Gu R, Song Z, Chen J, Yang M, Zhang J, Jin J, Wang Y, Mi Y, Wang J. The prognostic factors in acute myeloid leukaemia with double-mutated CCAAT/enhancer-binding protein alpha (CEBPAdm). Br J Haematol. 2022 May;197(4):442-451. doi: 10.1111/bjh.18113. Epub 2022 Mar 10.

    PubMed 35274287
  • Wei H, Wang Y, Gale RP, Lin D, Zhou C, Liu B, Qiu S, Gu R, Li Y, Zhao X, Wei S, Gong B, Liu K, Gong X, Liu Y, Zhang G, Song Z, Wang Y, Li W, Mi Y, Wang J. Randomized Trial of Intermediate-dose Cytarabine in Induction and Consolidation Therapy in Adults with Acute Myeloid Leukemia. Clin Cancer Res. 2020 Jul 1;26(13):3154-3161. doi: 10.1158/1078-0432.CCR-19-3433. Epub 2020 Feb 6.

    PubMed 32029439

Questions about this trial

What is this clinical trial studying?

A study testing HAD and Daunorubicin+Cytarabine in people with AML, 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 5 inclusion and 6 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 10 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.

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?

Event-free survival (EFS) (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?

On the ClinicalTrials.gov record for NCT06529250, which also lists 3 linked publications.

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 10 Aug 2026.

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