Donor stem cell transplant for high-risk CEBPA-mutated acute myeloid leukemia
Studies donor stem cell transplants in CEBPA-mutated acute myeloid leukemia.
Plain-language title generated from the registry record
Registry title: The Efficacy of Allogeneic Hematopoietic Stem Cell Transplantation in Newly Diagnosed High-relapse-risk CEBPA Mutant Acute Myeloid Leukemia
Key facts
- Where
- Shanghai, ChinaSee the site
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults aged 18 to 65 with newly diagnosed CEBPA-mutant acute myeloid leukemia (AML) at high risk of coming back. They get a stem cell transplant from a donor after chemotherapy.
- Age
- 18 to 65 years old (18 years or older and younger than 65).
- Sex
- All sexes can join.
- Disease stage
- Newly diagnosed CEBPA-mutant AML at high risk of relapse. This means a high-risk marker or gene change (CD7 positive, WT1 mutation, non-bzip-inframe CEBPA mutation), a complex karyotype, or MRD still positive after two chemotherapy treatments.
- Earlier treatment
- Before induction therapy, only hydroxyurea is allowed if white blood cells are under 50*109/L. If white blood cells are 50*109/L or higher, cytarabine and hydroxyurea are allowed. The plan is one cycle of induction and two cycles of consolidation before the transplant.
- Biomarker or test result
- A CEBPA gene mutation must be found by molecular testing. High-risk markers are CD7 positive, WT1 mutation or non-bzip-inframe CEBPA mutation. MRD positive means 0.0001% or more by flow cytometry.
Main reasons for not taking part
- AML that came from MDS or from earlier cancer treatment, or mixed cell leukemia
- AML in the brain or spinal fluid, or outside the bone marrow, at the start
- Relapsed leukemia
- Serious heart, lung, liver or kidney problems beyond the trial limits
- Active hepatitis A, B or C, tuberculosis or HIV, or another cancer at the same time
Read by machine on 12 November 2026.
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 65 years old?
Source: NCT06458257, who can take part
Where in China
- Ruijin Hospital Affiliated to Shanghai Jiaotong University School of MedicineShanghai, ChinaContact: Jian Li, PhDTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- Ruijin Hospital
- Trial code
- NCT06458257
- Record updated
- 29 July 2025
- Original description
For newly diagnosed high-relapse-risk CEBPA mutant acute myeloid leukemia patients, we aim to perform allogeneic hematopoietic stem cell transplantation after patients finished one cycle of induction and two cycles of consolidation. To access whether the therapeutic regimen is effective for high-relapse-risk CEBPA mutant acute myeloid leukemia, the disease-free-survival (DFS), overall survival (OS), non-relapse-mortality of patients is evaluated.Read full original descriptionShow less
The Efficacy of Allogeneic Hematopoietic Stem Cell Transplantation in Newly Diagnosed High-relapse-risk CEBPA Mutant Acute Myeloid Leukemia
For newly diagnosed high-relapse-risk CEBPA mutant acute myeloid leukemia patients, we aim to perform allogeneic hematopoietic stem cell transplantation after patients finished one cycle of induction and two cycles of consolidation. To access whether the therapeutic regimen is effective for high-relapse-risk CEBPA mutant acute myeloid leukemia, the disease-free-survival (DFS), overall survival (OS), non-relapse-mortality of patients is evaluated.
Inclusion: Patients with confirmed CEBPA mutant AML. Diagnostic criteria include the presence of CEBPA mutant gene detected at the molecular level; Patients with high-risk molecular markers or gene mutations, or complex karyotypes for disease recurrence, or flow cytometry/gene MRD positivity after two chemotherapy treatments; high-risk molecular markers or gene mutations include: CD7 positive, WT1 mutation, non-bzip-inframe CEBPA mutation; The positive threshold for flow cytometry MRD was 0.0001%; The MRD threshold of molecular biology is the lowest value of the detection protocol of the center; Medical history and diagnosis of MICM, exclusion of MDS, transformation and treatment-related AML; Age 18-65 years old (18 years old ≤Age< 65 years old); Liver and kidney function: blood bilirubin ≤ 35 μmol/L, AST/ALT below 2 times the upper limit of normal, serum creatinine ≤ 150 μmol/L; Normal cardiac function (EF≥50%, New York Cardiac Function Classification NYHA I/II); Physical condition score 0-2 (ECOG score); For patients with peripheral blood leukocytes < 50*109/L at the initial onset, no chemotherapy has been given except for hydroxyurea before the start of induction therapy; For patients with peripheral blood leukocytes ≥ 50*109/L at the initial onset, cytarabine and hydroxyurea are allowed to be treated before the start of induction therapy; Non-pregnant and lactating women; For all women of childbearing age, a pregnancy test must be performed to measure hCG to rule out pregnancy; Obtain informed consent signed by the patient or family member.
Exclusion: Concomitant other hematologic diseases (including coagulation abnormalities unrelated to leukemia); Allergies or contraindications to any of the drugs involved in the protocol; Lung diseases: pulmonary edema, pleural effusion (CTCAE score >2); Suffering from malignant tumors of other organs at the same time; Active patients with HAV, HBV, HCV and tuberculosis, HIV-positive patients; Those who participate in other clinical studies at the same time; Presence of any other condition that would preclude the conduct of the study; Cardiac disease: including echocardiogram EF <50%, cardiac insufficiency (New York cardiac function classification NYHA: III/IV), pericardial effusion (CTCAE score >2) within six months after acute myocardial infarction, ECG QTc >470ms; MDS-converted AML, treatment-related AML; mixed cell leukemia; AML patients with central nervous system infiltrates and extramedullary lesions at the time of onset; Relapse patients; Liver and kidney function are obviously abnormal, exceeding the enrollment criteria; Inability to understand or follow the study protocol.