The Efficacy of Allo-HSCT in ND HR-CBF-AML

NCT ID: NCT06458244

Last Updated: 2024-11-07

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 90 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2024-09-21
• Study Completion Date: 2028-02-29

Brief Summary

For newly diagnosed high-relapse-risk core-binding-factor acute myeloid leukemia participants, the investigators aim to perform allogeneic hematopoietic stem cell transplantation after participants finished one cycle of induction and two cycles of consolidation. To access whether the therapeutic regimen is effective for high-relapse-risk core-binding-factor acute myeloid leukemia, the disease-free-survival (DFS), overall survival (OS), non-relapse-mortality of participants is evaluated.

Detailed Description

High-relapse-risk definition:

Participants with high-risk gene mutations or complex karyotypes for disease recurrence, or flow cytometry/gene MRD positivity after two chemotherapy treatments; High-risk gene mutations include: TP53, RTK/RAS signaling (FLT3, NRAS, KRAS, KIT, JAK2, CSF3R), chromatin modification (ASXL1, ASXL2, KMD6A, EZH2, SETD2) or mutations listed as intermediate-risk or high-risk in the 2022NCCN guidelines; 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.

Conditions

Acute Myeloid Leukemia

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

HSCT-group

The participants receive HSCT after two-cycle of consolidation treatment.

Allogeneic Hematopoietic Stem Cell Transplantation

Intervention Type: PROCEDURE

Allogeneic Hematopoietic Stem Cell Transplantation

Chemo-group

The participants receive chemotherapy after two-cycle of consolidation treatment.

Chemotherapy

Intervention Type: DRUG

Chemotherapy for AML consolidation treatment

Interventions

Allogeneic Hematopoietic Stem Cell Transplantation

Allogeneic Hematopoietic Stem Cell Transplantation

Intervention Type: PROCEDURE

Chemotherapy

Chemotherapy for AML consolidation treatment

Intervention Type: DRUG

Other Intervention Names

High-dose cytarabine

Eligibility Criteria

Inclusion Criteria

1. Participants with confirmed CBF-AML. Diagnostic criteria include the presence of t(8; 21)(q22; q22)/RUNX1-RUNX1T1 fusion gene detected at the molecular level; or chromosome presence of inv(16)(p13.1q22)/t(16; 16)(p13.1; q22) /Detection of CBFβ-MYH11 fusion gene at the molecular level;

2. Participants with high-risk gene mutations or complex karyotypes for disease recurrence, or flow cytometry/gene MRD positivity after two chemotherapy treatments; High-risk gene mutations include: TP53, RTK/RAS signaling (FLT3, NRAS, KRAS, KIT, JAK2, CSF3R), chromatin modification (ASXL1, ASXL2, KMD6A, EZH2, SETD2) or mutations listed as intermediate-risk or high-risk in the 2022NCCN guidelines; 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.

3. Medical history and diagnosis of MICM, exclusion of MDS, transformation and treatment-related AML;

4. Age 18-65 years old (18 years old ≤Age< 65 years old);

5. Liver and kidney function: blood bilirubin ≤ 35 μmol/L, AST/ALT below 2 times the upper limit of normal, serum creatinine ≤ 150 μmol/L;

6. Normal cardiac function (EF≥50%, New York Cardiac Function Classification NYHA I/II);

7. Physical condition score 0-2 (ECOG score);

8. For participants 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;

9. For participants 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;

10. Non-pregnant and lactating women;

11. For all women of childbearing age, a pregnancy test must be performed to measure hCG to rule out pregnancy;

12. Obtain informed consent signed by the patient or family member.

Exclusion Criteria

1. MDS-converted AML, treatment-related AML; mixed cell leukemia; AML with central nervous system infiltrates and extramedullary lesions at the time of onset;

2. Relapse AML;

3. Allergies or contraindications to any of the drugs involved in the protocol;

4. Liver and kidney function are obviously abnormal, exceeding the enrollment criteria;

5. 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;

6. Lung diseases: pulmonary edema, pleural effusion (CTCAE score >2);

7. Suffering from malignant tumors of other organs at the same time;

8. Active patients with HAV, HBV, HCV and tuberculosis, HIV-positive patients;

9. Concomitant other hematologic diseases (including coagulation abnormalities unrelated to leukemia);

10. Inability to understand or follow the study protocol;

11. Those who participate in other clinical studies at the same time; Presence of any other condition that would preclude the conduct of the study.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Ruijin Hospital

OTHER

Sponsor Role: lead

Responsible Party

Shen yang

Chief physician

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Yang Shen, MD, PhD

Role: STUDY_DIRECTOR

Ruijin Hospital, Shanghai, China

Locations

Ruijin Hospital Affiliated to Shanghai Jiaotong University School Of Medicine

Shanghai, Shanghai Municipality, China

Site Status: RECRUITING

Countries

China

Central Contacts

Yang Shen, MD, PhD

Role: CONTACT

shen_yang@126.com

02164370045

Facility Contacts

Jian Li, PhD

Role: primary

lj12116@rjh.com.cn

021-64370045

Yang Shen, PhD

Role: backup

Other Identifiers

CBF-AML01

Identifier Type: -

Identifier Source: org_study_id