AML, AdultPhase 3NCT06297772
Compare the Efficacy and Safety of Dec-FB4 and FB4 as Conditioning Regimen for AML-MR
A Phase 3 study testing Decitabine, Fludarabine Injection and Busulfan Injection in people with AML, Adult, run by Ruijin Hospital at 1 site in China.
Trial summary
A multicenter, randomized, controlled clinical study comparing the efficacy and safety of allogeneic hematopoietic stem cell transplantation with decitabine-Fludarabine- busulfan (Dec-FB4) and Fludarabine-busulfan (FB4) as pretreatment regimens for the treatment of acute myeloid leukemia in adults with MR gene… Read more about this study
- Who is it for?
- Ages 16 to 65 years
- Conditions the study is for
- AML, Adult
- What is being tested?
- Decitabine, Fludarabine Injection and Busulfan Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 220 participants
- Where
- 1 siteChina
- Study dates
- 1 Jun 2024to 1 May 2028
- Phase
- Phase 3
- 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 16 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age 16-65 years and with an ECOG physical fitness score of 0-2 and an HCT-CI of less than 3;
- Diagnosis: All enrolled cases were diagnosed with acute myeloid leukemia (AML-MR) after bone marrow cytomorphology, cytochemistry, immunophenotyping, chromosomal examination, and gene mutation, and achieved hematological remission (bone marrow smear primitive cells <5%) and negative MRD before transplantation.
- Have a medically qualified and suitable hematopoietic stem cell donor, including HLA-allogamous sibling donors, unrelated donors (HLA high-resolution 9-10/10 compatible) or related haploidentical donors;
- No dysfunction of the heart, liver, lungs, kidneys, or other important organs, as defined as follows: ALT and AST ≤3 times the upper limit of normal; total bilirubin ≤2 times the upper limit of normal); BUN and Cr ≤1.25 times the upper limit of normal; electrocardiograms not suggestive of acute myocardial infarction or serious arrhythmias; cardiac echocardiography left ventricular ejection fraction ≥50%, no significant cardiac enlargement, valvular disease, or congenital heart disease; pulmonary function examination FEV1, FVC, DLCO ≥ 60% of the predicted value.
- The patient and his/her legal representative have the desire and requirement to undergo hematopoietic stem cell transplantation and sign an informed consent, willingness and compliance with the treatment plan, follow-up schedule, laboratory tests, etc.
Who cannot join
- AML in unremitting stage; or BM in remission but with concurrent CNS leukemia or presence of extramedullary lesions;
- Active hepatitis B (HBV-DNA ≥1×10^3 copies/ml);
- HIV-infected patients;
- Active infections requiring intravenous antibiotic therapy;
- There is severe impairment of vital organ function: respiratory failure, heart failure, decompensated hepatic insufficiency, renal insufficiency, etc;
- Patients who use drugs or chronic alcohol abuse to the extent that it interferes with the evaluation of test results;
- Mentally challenged/unable to obtain informed consent;
- Those judged by the investigator to be unsuitable for participation in this trial.
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
Ruijin Hospital
Shanghai, 200025
Xiaoxia Hu, 862164370045, hxx12276@rjh.com.cn
About this study
Official title: Comparing the Efficacy and Safety of Decitabine-Fludarabine-busulfan (Dec-FB4) and Fludarabine-busulfan (FB4) as Conditioning Regimens for AML-MR
A multicenter, randomized, controlled clinical study comparing the efficacy and safety of allogeneic hematopoietic stem cell transplantation with decitabine-Fludarabine- busulfan (Dec-FB4) and Fludarabine-busulfan (FB4) as pretreatment regimens for the treatment of acute myeloid leukemia in adults with MR gene abnormalities
Read the detailed description
AML-MR is one of high-risk AML with aggressive disease progression and poor prognosis. The median survival of AML-MR is only 10.5 months. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective post-remission therapy for high-risk acute myeloid leukemia (AML)/myelodysplastic syndrome (MDS). Selection of an appropriate pretreatment regimen that balances the risk of relapse and reduces the risk of non-relapse mortality is a key component of successful HSCT. FB4 regimen consisting of Fludarabine (FLU) combined with busulfan (BU) has become the most commonly used myeloablative preconditioning regimen for allo-HSCT in patients with AML/MDS, and prospective studies have demonstrated a similar relapse rate and lower treatment-related mortality than the Bu/cy regimen.
Decitabine is also approved for the treatment of AML and MDS as an inhibitor of DNA methyltransferase I, which allows for the expression of silenced oncogenes and terminal differentiation of leukemia cells, and as a single agent is superior to supportive therapy for patients with MDS. In addition, decitabine has been shown to attenuate GVHD by enhancing the function of regulatory T cells, and in myeloid tumors, several prospective single-arm and retrospective clinical studies have demonstrated that modified pretreatment regimens containing decitabine have a lower relapse rate, as well as a reduction in the incidence and severity of GVHD, with satisfactory survival data in allogeneic hematopoietic stem cell transplantation. The investigators have not yet obtained direct evidence in high-risk patients to confirm the superiority of decitabine-containing pretreatment regimens over standard pretreatment regimens (FB4). There is a lack of prospective, controlled studies to clarify the efficacy and safety of decitabine-containing transplantation preconditioning in high-risk patients, especially those with specific AML-MR.Therefore, this multicenter, randomized controlled study was designed to compare the efficacy and safety of Dec-FB4 and FB4 regimens as pretreatment regimens for allogeneic HSCT in adults with AML-MR and to assess whether Dec-FB4 is superior or noninferior to FB4, in order to provide a reliable evidence-based medical basis for guiding the therapeutic choices for AML-MR/MDS patients.
- Conditions
- AML, Adult
- Being tested
- Decitabine, Fludarabine Injection, Busulfan Injection
- Keywords
- AML-MR, adults, conditioning regimen
- 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.
DecitabineDrug
decitabine 20mg/m2 per day for consecutive 5 days
Fludarabine InjectionDrug
fludarabine 30mg/m2 per day for consecutive 5 days
Busulfan InjectionDrug
Busulfan 3.2mg/kg per day for consecutive 4 days
| Study group | Role | What they receive |
|---|---|---|
| Flu-Bu4 | Active comparator | Preconditioning with Flu-Bu4 without decitabine for patients with AML-MR Receives: Drug: Fludarabine Injection; Drug: Busulfan Injection |
| Dec-Flu-Bu4 | Experimental | Preconditioning with Flu-Bu4 and decitabine for patients with AML-MR Receives: Drug: Decitabine; Drug: Fludarabine Injection; Drug: Busulfan Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
disease-free survival
disease progression or death as a result of any causes
Measured: through study completion, an average of 2 year
Secondary outcomes (2)
incidence of relapse rate
disease relapse
Measured: through study completion, an average of 2 year
Non-recurrent mortality
death without disease progression or relapse
Measured: through study completion, an average of 2 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2024
- Study start1 Jun 2024 (estimated)
- Primary completion1 Mar 2026 (estimated)
- Study completion1 May 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AML, AdultStudies 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 220 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ruijin Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Hu Xiaoxia, Ruijin Hospital
- Investigators
- Xiaoxia Hu, Principal investigator, Ruijin Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06297772
- Sponsor's protocol ID
- RJBMT-003
- Status last verified
- Jun 2024
- First submitted
- 1 Mar 2024
- Last updated
- 4 Jun 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Comparing the Efficacy and Safety of Decitabine-Fludarabine-busulfan (Dec-FB4) and Fludarabine-busulfan (FB4) as Conditioning Regimens for AML-MR
- ClinicalTrials.gov ID
- NCT06297772
- Acronym / sponsor ID
- RJBMT-003
- Conditions
- AML, Adult
- Keywords
- AML-MR, adults, conditioning regimen
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 220 participants (estimated)
- Lead sponsor
- Ruijin HospitalUniversity, hospital or other
- Study start
- 1 Jun 2024 (estimated)
- Primary completion
- 1 Mar 2026 (estimated)
- Study completion
- 1 May 2028 (estimated)
- First posted
- 7 Mar 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.
A multicenter, randomized, controlled clinical study comparing the efficacy and safety of allogeneic hematopoietic stem cell transplantation with decitabine-Fludarabine- busulfan (Dec-FB4) and Fludarabine-busulfan (FB4) as pretreatment regimens for the treatment of acute myeloid leukemia in adults with MR gene abnormalities
Detailed description
AML-MR is one of high-risk AML with aggressive disease progression and poor prognosis. The median survival of AML-MR is only 10.5 months. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective post-remission therapy for high-risk acute myeloid leukemia (AML)/myelodysplastic syndrome (MDS). Selection of an appropriate pretreatment regimen that balances the risk of relapse and reduces the risk of non-relapse mortality is a key component of successful HSCT. FB4 regimen consisting of Fludarabine (FLU) combined with busulfan (BU) has become the most commonly used myeloablative preconditioning regimen for allo-HSCT in patients with AML/MDS, and prospective studies have demonstrated a similar relapse rate and lower treatment-related mortality than the Bu/cy regimen.
Decitabine is also approved for the treatment of AML and MDS as an inhibitor of DNA methyltransferase I, which allows for the expression of silenced oncogenes and terminal differentiation of leukemia cells, and as a single agent is superior to supportive therapy for patients with MDS. In addition, decitabine has been shown to attenuate GVHD by enhancing the function of regulatory T cells, and in myeloid tumors, several prospective single-arm and retrospective clinical studies have demonstrated that modified pretreatment regimens containing decitabine have a lower relapse rate, as well as a reduction in the incidence and severity of GVHD, with satisfactory survival data in allogeneic hematopoietic stem cell transplantation. The investigators have not yet obtained direct evidence in high-risk patients to confirm the superiority of decitabine-containing pretreatment regimens over standard pretreatment regimens (FB4). There is a lack of prospective, controlled studies to clarify the efficacy and safety of decitabine-containing transplantation preconditioning in high-risk patients, especially those with specific AML-MR.Therefore, this multicenter, randomized controlled study was designed to compare the efficacy and safety of Dec-FB4 and FB4 regimens as pretreatment regimens for allogeneic HSCT in adults with AML-MR and to assess whether Dec-FB4 is superior or noninferior to FB4, in order to provide a reliable evidence-based medical basis for guiding the therapeutic choices for AML-MR/MDS patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Flu-Bu4 | Active comparator | Drug: Fludarabine Injection, Drug: Busulfan InjectionPreconditioning with Flu-Bu4 without decitabine for patients with AML-MR |
| Dec-Flu-Bu4 | Experimental | Drug: Decitabine, Drug: Fludarabine Injection, Drug: Busulfan InjectionPreconditioning with Flu-Bu4 and decitabine for patients with AML-MR |
Who may qualify
- Ages
- 16 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Age 16-65 years and with an ECOG physical fitness score of 0-2 and an HCT-CI of less than 3;
- Diagnosis: All enrolled cases were diagnosed with acute myeloid leukemia (AML-MR) after bone marrow cytomorphology, cytochemistry, immunophenotyping, chromosomal examination, and gene mutation, and achieved hematological remission (bone marrow smear primitive cells <5%) and negative MRD before transplantation.
- Have a medically qualified and suitable hematopoietic stem cell donor, including HLA-allogamous sibling donors, unrelated donors (HLA high-resolution 9-10/10 compatible) or related haploidentical donors;
- No dysfunction of the heart, liver, lungs, kidneys, or other important organs, as defined as follows: ALT and AST ≤3 times the upper limit of normal; total bilirubin ≤2 times the upper limit of normal); BUN and Cr ≤1.25 times the upper limit of normal; electrocardiograms not suggestive of acute myocardial infarction or serious arrhythmias; cardiac echocardiography left ventricular ejection fraction ≥50%, no significant cardiac enlargement, valvular disease, or congenital heart disease; pulmonary function examination FEV1, FVC, DLCO ≥ 60% of the predicted value.
- The patient and his/her legal representative have the desire and requirement to undergo hematopoietic stem cell transplantation and sign an informed consent, willingness and compliance with the treatment plan, follow-up schedule, laboratory tests, etc.
Key exclusion criteria (8 listed)
- AML in unremitting stage; or BM in remission but with concurrent CNS leukemia or presence of extramedullary lesions;
- Active hepatitis B (HBV-DNA ≥1×10^3 copies/ml);
- HIV-infected patients;
- Active infections requiring intravenous antibiotic therapy;
- There is severe impairment of vital organ function: respiratory failure, heart failure, decompensated hepatic insufficiency, renal insufficiency, etc;
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 |
|---|---|---|
| Decitabinedecitabine 20mg/m2 per day for consecutive 5 days | Drug | Dec-Flu-Bu4 |
| Fludarabine Injectionfludarabine 30mg/m2 per day for consecutive 5 days | Drug | Dec-Flu-Bu4, Flu-Bu4 |
| Busulfan InjectionBusulfan 3.2mg/kg per day for consecutive 4 days | Drug | Dec-Flu-Bu4, Flu-Bu4 |
Primary outcome
disease-free survival
Time frame: through study completion, an average of 2 year
disease progression or death as a result of any causes
2 secondary outcomes
incidence of relapse rate
Time frame: through study completion, an average of 2 year
Non-recurrent mortality
Time frame: through study completion, an average of 2 year
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 4 Jun 2024.
Related trials for AML, Adult
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
RecruitingPhase 2
Comparing Different Treatment Lengths for Venetoclax in Older People With Newly Diagnosed Acute Myeloid Leukemia (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia
RecruitingPhase 3
Bleximenib in Combination With Standard Induction and Consolidation Therapy Followed by Maintenance for Treatment of Patients With Acute Myeloid Leukemia (AML)
Acute Myeloid Leukemia
RecruitingPhase 2
Testing the Addition of Venetoclax or Gemtuzumab Ozogamicin (GO) to Usual Treatment Regimen (Cytarabine and Daunorubicin, "7+3") for Core Binding Factor Acute Myeloid Leukemia (CBF-AML) to Improve Response (A MYELOMATCH Treatment Trial)
Core Binding Factor Acute Myeloid Leukemia
RecruitingPhase 2
Venetoclax and HMA Treatment of Older and Unfit Adults With FLT3 Mutated Acute Myeloid Leukemia (AML) (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia
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.
Xiaoxia Hu principal investigator
Ruijin Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Decitabine, Fludarabine Injection and Busulfan Injection in people with AML, Adult, 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 5 inclusion and 8 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: opening soon, as of the sponsor's last update on 4 Jun 2024. 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 Xiaoxia Hu (+862164370045, hxx12276@rjh.com.cn), 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: through study completion, an average of 2 year). 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 4 Jun 2024.