Intermediate-dose treatment for CEBPA double-mutated AML

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

This study is testing a new intermediate-dose treatment for people with CEBPA double-mutated acute myeloid leukemia to see if it works better than the standard treatment.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 148 (estimated)
• Ages: 14 Years to 54 Years
• Sex: All
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China (Academic / other)
• Drugs / interventions: chemotherapy, cyclophosphamide
• Locations: 1 site (Tianjin, Tianjin Municipality)
• Trial ID: NCT06529250 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to evaluate the efficacy of an intermediate-dose HAD regimen in patients diagnosed with CEBPA double-mutated acute myeloid leukemia (AML). Participants will be randomly assigned to receive either the intermediate-dose HAD regimen or the conventional '3+7' induction regimen. The study will assess treatment outcomes, including complete remission rates and overall survival, to determine if the new regimen offers superior results compared to standard therapies. The trial will also include reinduction therapy for patients who do not achieve complete remission after initial treatment.

Who should consider this trial

Good fit: Ideal candidates for this study are patients aged 14 to 55 years with a confirmed diagnosis of CEBPA double-mutated AML.

Not a fit: Patients who have previously received induction chemotherapy or have other concurrent malignancies may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a more effective option for patients with CEBPA double-mutated AML, potentially improving survival rates.

How similar studies have performed: While the approach is based on previous retrospective findings, this specific regimen's efficacy in a randomized controlled setting is novel and untested.

Eligibility criteria

Inclusion Criteria:

1. AML diagnosed according to WHO-2022 classification with recurrent CEBPA mutations and containing mutation in the bZIP domain.
2. Older than 14 years old and younger than 55 years old
3. Male or female.
4. The Eastern Cooperative Oncology Group Performance Status (ECOG-PS) of AML patients were 0-2 points.
5. 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.

Exclusion Criteria:

1. Patients who have previously received induction chemotherapy, regardless of efficacy.
2. Simultaneously suffering from malignant tumors of other organs and requiring treatment).
3. Pregnant or lactating women. Male or female patients participating in the trial must take contraceptive measures during the trial treatment period.
4. 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.
5. Serious infectious diseases (uncured tuberculosis, pulmonary aspergillosis).
6. Those who were not considered suitable for inclusion by the researchers.

Where this trial is running

Tianjin, Tianjin Municipality

• Blood Hospital — Tianjin, Tianjin Municipality, China (Recruiting)

Study contacts

• Principal investigator: Hui Wei, MD — Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
• Study coordinator: Hui Wei, MD

Conditions AML; CEBPA double-mutated; treatment