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

NCT06458244 | Recruiting FDA Drug

• 1 other identifier: CBF-AML01
• Study Type: observational
• Target: 90
• Locations: 1 country
• Sites: 1

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.

Conditions

Acute Myeloid Leukemia

Keywords

core binding factor; high-risk; newly diagnosed

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 CBF-AML genetics subgroup with MRD negativity rate after chemotherapy and DFS after transplantation

  The relation of CBF-AML genetics subgroup with MRD negativity rate after chemotherapy and DFS after transplantation

  from data of AML diagnosis until the data of CR-achieved status, assessed up to 3 years

Secondary Outcomes (3)

• Rate of non-relapse-mortality

  the data of death from any cause, assessed up to 3 years

• overall survival

  from data of AML diagnosed until the data of death from any cause, assessed up to 3 years

• adverse events

  from enrollment to study completion, a maximum of 3 years

Study Arms (2)

HSCT-group

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

Procedure: Allogeneic Hematopoietic Stem Cell Transplantation

Chemo-group

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

Drug: Chemotherapy

Interventions

Allogeneic Hematopoietic Stem Cell Transplantation PROCEDURE

Allogeneic Hematopoietic Stem Cell Transplantation

HSCT-group

Chemotherapy DRUG

Chemotherapy for AML consolidation treatment

Also known as: High-dose cytarabine

Chemo-group

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Newly Diagnosed High-relapse-risk Core-binding-factor Acute Myeloid Leukemia in Ruijin Hospital

You may qualify if:

• 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;
• 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.
• 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 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;
• 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;
• 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 with central nervous system infiltrates and extramedullary lesions at the time of onset;
• Relapse AML;
• 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

The samples including bone marrow cells, blood or DNA/RNA extracted from the cells will be reposited for 5 years.

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

Drug Therapy; Cytarabine

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Therapeutics; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Arabinonucleosides; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides

Study Officials

• Yang Shen, MD, PhD

  Ruijin Hospital, Shanghai, China

  STUDY DIRECTOR

Central Study Contacts

Yang Shen, MD, PhD

CONTACT

02164370045; shen_yang@126.com

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: September 21, 2024
• Primary Completion (Estimated): September 20, 2026
• Study Completion (Estimated): February 1, 2028
• Last Updated: April 16, 2026

Record last verified: 2026-04

Locations

CN (1)