The Efficacy of Allogeneic Hematopoietic Stem Cell Transplantation in Newly Diagnosed High-relapse-risk CEBPA Mutant Acute Myeloid Leukemia

NCT06458257 | Recruiting DMC FDA Drug

• 1 other identifier: CEBPA-AML01
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Acute Myeloid Leukemia

Keywords

newly diagnosed; high-relapse-risk; CEBPA mutant

Outcome Measures

Primary Outcomes (2)

• disease-free-survival

  disease-free-survival

  from data of AML diagnosis until the data of AML relapse, assessed up to 3 years

• The relation of CEBPA Mutant AML genetics subgroup with MRD negativity rate after chemotherapy and DFS after transplantation

  The relation of CEBPA Mutant AML genetics subgroup with MRD negativity rate after chemotherapy and DFS after transplantation

  from enrollment to study completion, a maximum of 3 years

Secondary Outcomes (3)

• non-relapse-mortality

  from enrollment to study completion, a maximum of 3 years

• overall survival

  from enrollment to study completion, a maximum of 3 years

• adverse events

  from enrollment to study completion, a maximum of 3 years

Study Arms (2)

HSCT-group

patients received allogeneic hematopoietic stem cell transplantation after consolidation treatment

Procedure: Allogeneic Hematopoietic Stem Cell Transplantation

Chemo-group

patients received chemotherapy after consolidation treatment

Drug: Chemotherapy

Interventions

Allogeneic Hematopoietic Stem Cell Transplantation PROCEDURE

Allogeneic Hematopoietic Stem Cell Transplantation

HSCT-group

Chemotherapy DRUG

Chemotherapy

Chemo-group

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Patients Diagnosed in Ruijin Hospital

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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;
• Allergies or contraindications to any of the drugs involved in the protocol;
• Liver and kidney function are obviously abnormal, exceeding the enrollment criteria;
• 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;
• 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;
• Concomitant other hematologic diseases (including coagulation abnormalities unrelated to leukemia);
• Inability to understand or follow the study protocol;
• Those who participate in other clinical studies at the same time; Presence of any other condition that would preclude the conduct of the study

Sponsors & Collaborators

• Ruijin Hospital: lead

Study Sites (1)

Ruijin Hospital Affiliated to Shanghai Jiaotong University School Of Medicine

Shanghai, Shanghai Municipality, 200025, China

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

bone marrow; peripheral blood

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

Drug Therapy

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Therapeutics

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief physician

Study Record Dates

• First Submitted: January 9, 2024
• First Posted: June 13, 2024
• Study Start: October 19, 2024
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2028
• Last Updated: July 29, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will share

Locations

CN (1)