Acute Myeloid LeukemiaNCT06458244
The Efficacy of Allo-HSCT in ND HR-CBF-AML
An observational study of Acute Myeloid Leukemia, run by Ruijin Hospital at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Allogeneic Hematopoietic Stem Cell Transplantation and Chemotherapy
- Type of treatment
- Surgery or procedure
- How many people
- 90 participants
- Where
- 1 siteChina
- Study dates
- 21 Sep 2024to Feb 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Newly Diagnosed High-relapse-risk Core-binding-factor Acute Myeloid Leukemia in Ruijin Hospital
Who may be able to join
- 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.
- 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);
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- 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.
Who cannot join
- 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);
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Ruijin Hospital Affiliated to Shanghai Jiaotong University School Of Medicine
Shanghai, Shanghai Municipality, 200025
Jian Li, PhD, 021-64370045, lj12116@rjh.com.cn
Yang Shen, PhD
About this study
Official title: The Efficacy of Allogeneic Hematopoietic Stem Cell Transplantation in Newly Diagnosed High-relapse-risk Core-binding-factor Acute Myeloid Leukemia
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.
Read the 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
- Being tested
- Allogeneic Hematopoietic Stem Cell Transplantation, Chemotherapy
- Keywords
- core binding factor; high-risk; newly diagnosed
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic Hematopoietic Stem Cell TransplantationProcedure or surgery
Allogeneic Hematopoietic Stem Cell Transplantation
ChemotherapyDrug
Also called High-dose cytarabine
Chemotherapy for AML consolidation treatment
| Study group | Role | What they receive |
|---|---|---|
| HSCT-group | The participants receive HSCT after two-cycle of consolidation treatment. Receives: Procedure: Allogeneic Hematopoietic Stem Cell Transplantation | |
| Chemo-group | The participants receive chemotherapy after two-cycle of consolidation treatment. Receives: Drug: Chemotherapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
disease-free-survival
disease-free-survival
Measured: 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
Measured: 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 ratio of non-relapse-mortality cases and all the mortality cases
Measured: the data of death from any cause, assessed up to 3 years
overall survival
overall survival
Measured: from data of AML diagnosed until the data of death from any cause, assessed up to 3 years
adverse events
adverse events related with the indicated regimen
Measured: from enrollment to study completion, a maximum of 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jun 2024
- Study start21 Sep 2024
- Primary completion20 Sep 2026 (estimated)
- Study completionFeb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by Ruijin HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ruijin Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Shen yang, Ruijin Hospital
- Investigators
- Yang Shen, MD, PhD, Study director, Ruijin Hospital, Shanghai, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06458244
- Sponsor's protocol ID
- CBF-AML01
- Status last verified
- Apr 2026
- First submitted
- 9 Jan 2024
- Last updated
- 16 Apr 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Efficacy of Allogeneic Hematopoietic Stem Cell Transplantation in Newly Diagnosed High-relapse-risk Core-binding-factor Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT06458244
- Acronym / sponsor ID
- CBF-AML01
- Conditions
- Acute Myeloid Leukemia
- Keywords
- core binding factor; high-risk; newly diagnosed
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- Ruijin HospitalUniversity, hospital or other
- Study start
- 21 Sep 2024 (actual)
- Primary completion
- 20 Sep 2026 (estimated)
- Study completion
- Feb 2028 (estimated)
- First posted
- 13 Jun 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| HSCT-group | – | Procedure: Allogeneic Hematopoietic Stem Cell TransplantationThe participants receive HSCT after two-cycle of consolidation treatment. |
| Chemo-group | – | Drug: ChemotherapyThe participants receive chemotherapy after two-cycle of consolidation treatment. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Newly Diagnosed High-relapse-risk Core-binding-factor Acute Myeloid Leukemia in Ruijin Hospital
Key inclusion criteria (12 listed)
- 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.
- 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;
Key exclusion criteria (11 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Allogeneic Hematopoietic Stem Cell TransplantationAllogeneic Hematopoietic Stem Cell Transplantation | Procedure or surgery | HSCT-group |
| ChemotherapyAlso: High-dose cytarabineChemotherapy for AML consolidation treatment | Drug | Chemo-group |
Primary outcome
disease-free-survival
Time frame: from data of AML diagnosis until the data of AML relapse, assessed up to 3 years
disease-free-survival
The relation of CBF-AML genetics subgroup with MRD negativity rate after chemotherapy and DFS after transplantation
Time frame: from data of AML diagnosis until the data of CR-achieved status, assessed up to 3 years
The relation of CBF-AML genetics subgroup with MRD negativity rate after chemotherapy and DFS after transplantation
3 secondary outcomes
Rate of non-relapse-mortality
Time frame: the data of death from any cause, assessed up to 3 years
overall survival
Time frame: from data of AML diagnosed until the data of death from any cause, assessed up to 3 years
adverse events
Time frame: from enrollment to study completion, a maximum of 3 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Apr 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Yang Shen, MD, PhD study director
Ruijin Hospital, Shanghai, China
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Acute Myeloid Leukemia, run by Ruijin Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yang Shen, MD, PhD (02164370045, shen_yang@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
disease-free-survival (time frame: from data of AML diagnosis until the data of AML relapse, assessed up to 3 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Apr 2026.