Acute Myeloid LeukemiaTBF-AMLNCT07563920
TBF Conditioning Regimen for Haploidentical Stem Cell Transplantation in Elderly AML Patients in First Complete Remission
A study testing Thiotepa, Busulfan (BU) and Fludarabine in people with Acute Myeloid Leukemia, run by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine.
Trial summary
Acute myeloid leukemia (AML) is a serious blood cancer that mainly affects older adults. For patients who achieve their first complete remission (CR1), allogeneic hematopoietic stem cell transplantation (HSCT) may provide a chance for long-term survival. Read more about this study
- Who is it for?
- Ages 55 to 75 years
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Thiotepa, Busulfan (BU) and Fludarabine
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 93 participants
- Where
- No sites listed yet
- Study dates
- Jun 2026to Jun 2030
- Phase
- Interventional
- 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 55 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 55 to 75 years
- Diagnosed with acute myeloid leukemia (AML) based on morphology, immunophenotyping, cytogenetics, or molecular testing
- First complete remission (CR1) or complete remission with incomplete hematologic recovery (CRi)
- Eligible for haploidentical hematopoietic stem cell transplantation
- Availability of a suitable haploidentical donor
- ECOG performance status 0-2
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- Adequate organ function:
- Left ventricular ejection fraction ≥50%
- Oxygen saturation >92% on room air
- Serum creatinine ≤1.5 × upper limit of normal (ULN)
- Total bilirubin ≤1.5 × ULN
- AST and ALT ≤2.0 × ULN
- DLCO ≥40% and FEV1 ≥50%
- Ability to understand and sign informed consent
Who cannot join
- Secondary AML (including AML evolving from myelodysplastic syndrome or therapy-related AML)
- Active, uncontrolled infection
- Severe uncontrolled systemic disease (e.g., unstable cardiovascular disease, recent stroke, or severe organ dysfunction)
- HIV infection
- Active hepatitis B or C requiring antiviral treatment
- Pregnant or breastfeeding women
- Known hypersensitivity to study drugs
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Single-Center, Prospective, Single-Arm Clinical Study Evaluating the Efficacy and Safety of Thiotepa, Busulfan, and Fludarabine (TBF) Conditioning Regimen in Haploidentical Peripheral Blood Stem Cell Transplantation for Elderly Acute Myeloid Leukemia Patients in First Complete Remission
Acute myeloid leukemia (AML) is a serious blood cancer that mainly affects older adults. For patients who achieve their first complete remission (CR1), allogeneic hematopoietic stem cell transplantation (HSCT) may provide a chance for long-term survival. However, relapse after transplantation remains a major challenge.
This study aims to evaluate the effectiveness and safety of a conditioning regimen that combines thiotepa, busulfan, and fludarabine (TBF) before haploidentical peripheral blood stem cell transplantation (haplo-PBSCT) in elderly patients with AML in first complete remission.
Read more
Eligible patients will receive the TBF conditioning regimen followed by stem cell transplantation from a partially matched donor. Participants will be followed to assess relapse-free survival, overall survival, transplant-related complications, and infections.
The results of this study may help improve treatment strategies and outcomes for elderly AML patients undergoing transplantation.
Read the detailed description
Acute myeloid leukemia (AML) is a heterogeneous hematologic malignancy with increasing incidence in older populations. Despite achieving first complete remission (CR1) after induction chemotherapy, elderly patients remain at high risk of relapse and have poor long-term outcomes.
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is considered a potentially curative treatment for AML. With the development of reduced-intensity conditioning regimens and haploidentical transplantation strategies, more elderly patients are now eligible for transplantation. However, relapse after transplantation remains a major limitation.
The conditioning regimen plays a critical role in determining transplant outcomes. The combination of thiotepa, busulfan, and fludarabine (TBF) has been proposed to enhance anti-leukemic activity while maintaining acceptable toxicity. Previous retrospective and registry-based studies suggest that TBF conditioning may reduce relapse risk compared with conventional regimens, but prospective data in elderly AML patients, especially in Asian populations, remain limited.
This study is a single-center, prospective, single-arm clinical trial designed to evaluate the efficacy and safety of the TBF conditioning regimen in elderly AML patients in first complete remission undergoing haploidentical peripheral blood stem cell transplantation (haplo-PBSCT).
Eligible patients aged 55-75 years with AML in CR1 or CRi will receive a conditioning regimen consisting of thiotepa (day -7), busulfan (days -4 and -3), and fludarabine (days -6 to -2), followed by infusion of donor stem cells on day 0.
The primary endpoint is 1-year relapse-free survival (RFS), defined as the time from transplantation to relapse or death from any cause. Secondary endpoints include overall survival, incidence of acute and chronic graft-versus-host disease (GVHD), non-relapse mortality, hematopoietic engraftment, donor chimerism, and infection rates.
Participants will be followed regularly after transplantation with clinical assessments, laboratory tests, and bone marrow evaluations according to protocol-defined schedules.
The findings from this study are expected to provide prospective evidence for the use of TBF conditioning in elderly AML patients and support optimization of transplantation strategies in this population.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Thiotepa, Busulfan (BU), Fludarabine, Haploidentical Peripheral Blood Stem Cell Transplantation
- Keywords
- Haploidentical Stem Cell Transplantation, TBF Conditioning Regimen, Thiotepa Busulfan Fludarabine, Elderly AML, Allogeneic Hematopoietic Stem Cell Transplantation, Relapse-Free Survival
- 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.
ThiotepaDrug
Thiotepa is administered intravenously at a dose of 5 mg/kg on day -7 as part of the TBF conditioning regimen prior to haploidentical peripheral blood stem cell transplantation.
Busulfan (BU)Drug
Busulfan is administered intravenously at a dose of 3.2 mg/kg on days -4 and -3 as part of the TBF conditioning regimen.
FludarabineDrug
Fludarabine is administered intravenously at a dose of 30 mg/m² daily from day -6 to day -2 as part of the conditioning regimen.
Haploidentical Peripheral Blood Stem Cell TransplantationProcedure or surgery
Haploidentical peripheral blood stem cell transplantation is performed on day 0 following conditioning. Donor stem cells are infused, and standard graft-versus-host disease prophylaxis and supportive care are provided according to institutional protocols.
| Study group | Role | What they receive |
|---|---|---|
| Arms Back to Arms and Interventions * Required * § Required if Study Start Date is on or after Janu | Experimental | Participants will receive a conditioning regimen consisting of thiotepa, busulfan, and fludarabine (TBF) followed by haploidentical peripheral blood stem cell transplantation. Thiotepa is administered on day -7, busulfan on days -4 and -3, and fludarabine on days -6 to -2. Donor stem cells are infused on day 0. Standard graft-versus-host disease prophylaxis and supportive care will be provided according to institutional guidelines. Receives: Drug: Thiotepa; Drug: Busulfan (BU); Drug: Fludarabine; Procedure: Haploidentical Peripheral Blood Stem Cell Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
1-year Relapse-Free Survival (RFS)
Relapse-free survival (RFS) is defined as the time from transplantation to the first occurrence of disease relapse or death from any cause. Patients who are alive without relapse at the last follow-up will be censored.
Measured: 12 months after transplantation
Secondary outcomes (4)
Overall Survival (OS)
Overall survival is defined as the time from transplantation to death from any cause. Patients alive at last follow-up will be censored.
Measured: 12 months after transplantation
Incidence of Acute Graft-Versus-Host Disease (aGVHD)
The cumulative incidence of grade I-IV and grade III-IV acute graft-versus-host disease within 180 days after transplantation, assessed according to standard criteria.
Measured: Up to 180 days after transplantation
Incidence of Chronic Graft-Versus-Host Disease (cGVHD)
The cumulative incidence of all-grade and moderate-to-severe chronic graft-versus-host disease, assessed according to NIH consensus criteria.
Measured: 12 months after transplantation
Non-Relapse Mortality (NRM)
Non-relapse mortality is defined as death without evidence of disease relapse. Relapse is treated as a competing event.
Measured: 12 months after transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 May 2026
- Study startJun 2026 (estimated)
- Primary completionJun 2029 (estimated)
- Study completionJun 2030 (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 Shanghai General Hospital, Shanghai Jiao Tong University School of MedicineThe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- All participants receive the TBF conditioning regimen followed by haploidentical peripheral blood stem cell transplantation.
- Masking
- None (open label)
- Enrolment
- 93 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xianmin Song, MD, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
- Investigators
- Xianmin Song, Principal investigator, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07563920
- Sponsor's protocol ID
- SHSYXY-202510-TBF-AML
- Acronym
- TBF-AML
- Status last verified
- Apr 2026
- First submitted
- 26 Apr 2026
- Last updated
- 4 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Individual participant data (IPD) will not be shared due to patient privacy concerns and institutional data protection policies. De-identified data may be available from the corresponding investigator upon reasonable request and with appropriate institutional approvals.
Study at a glance
From the registry record, last updated by the sponsor on 4 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-Center, Prospective, Single-Arm Clinical Study Evaluating the Efficacy and Safety of Thiotepa, Busulfan, and Fludarabine (TBF) Conditioning Regimen in Haploidentical Peripheral Blood Stem Cell Transplantation for Elderly Acute Myeloid Leukemia Patients in First Complete Remission
- ClinicalTrials.gov ID
- NCT07563920
- Acronym / sponsor ID
- TBF-AML / SHSYXY-202510-TBF-AML
- Conditions
- Acute Myeloid Leukemia
- Keywords
- Haploidentical Stem Cell Transplantation, TBF Conditioning Regimen, Thiotepa Busulfan Fludarabine, Elderly AML, Allogeneic Hematopoietic Stem Cell Transplantation, Relapse-Free Survival
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 93 participants (estimated)
- Lead sponsor
- Shanghai General Hospital, Shanghai Jiao Tong University School of MedicineUniversity, hospital or other
- Study start
- Jun 2026 (estimated)
- Primary completion
- Jun 2029 (estimated)
- Study completion
- Jun 2030 (estimated)
- First posted
- 4 May 2026 (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.
Acute myeloid leukemia (AML) is a serious blood cancer that mainly affects older adults. For patients who achieve their first complete remission (CR1), allogeneic hematopoietic stem cell transplantation (HSCT) may provide a chance for long-term survival. However, relapse after transplantation remains a major challenge.
This study aims to evaluate the effectiveness and safety of a conditioning regimen that combines thiotepa, busulfan, and fludarabine (TBF) before haploidentical peripheral blood stem cell transplantation (haplo-PBSCT) in elderly patients with AML in first complete remission.
Eligible patients will receive the TBF conditioning regimen followed by stem cell transplantation from a partially matched donor. Participants will be followed to assess relapse-free survival, overall survival, transplant-related complications, and infections.
The results of this study may help improve treatment strategies and outcomes for elderly AML patients undergoing transplantation.
Detailed description
Acute myeloid leukemia (AML) is a heterogeneous hematologic malignancy with increasing incidence in older populations. Despite achieving first complete remission (CR1) after induction chemotherapy, elderly patients remain at high risk of relapse and have poor long-term outcomes.
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is considered a potentially curative treatment for AML. With the development of reduced-intensity conditioning regimens and haploidentical transplantation strategies, more elderly patients are now eligible for transplantation. However, relapse after transplantation remains a major limitation.
The conditioning regimen plays a critical role in determining transplant outcomes. The combination of thiotepa, busulfan, and fludarabine (TBF) has been proposed to enhance anti-leukemic activity while maintaining acceptable toxicity. Previous retrospective and registry-based studies suggest that TBF conditioning may reduce relapse risk compared with conventional regimens, but prospective data in elderly AML patients, especially in Asian populations, remain limited.
This study is a single-center, prospective, single-arm clinical trial designed to evaluate the efficacy and safety of the TBF conditioning regimen in elderly AML patients in first complete remission undergoing haploidentical peripheral blood stem cell transplantation (haplo-PBSCT).
Eligible patients aged 55-75 years with AML in CR1 or CRi will receive a conditioning regimen consisting of thiotepa (day -7), busulfan (days -4 and -3), and fludarabine (days -6 to -2), followed by infusion of donor stem cells on day 0.
The primary endpoint is 1-year relapse-free survival (RFS), defined as the time from transplantation to relapse or death from any cause. Secondary endpoints include overall survival, incidence of acute and chronic graft-versus-host disease (GVHD), non-relapse mortality, hematopoietic engraftment, donor chimerism, and infection rates.
Participants will be followed regularly after transplantation with clinical assessments, laboratory tests, and bone marrow evaluations according to protocol-defined schedules.
The findings from this study are expected to provide prospective evidence for the use of TBF conditioning in elderly AML patients and support optimization of transplantation strategies in this population.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- All participants receive the TBF conditioning regimen followed by haploidentical peripheral blood stem cell transplantation.
| Arm | Type | Receives |
|---|---|---|
| Arms Back to Arms and Interventions * Required * § Required if Study Start Date is on or after Janu | Experimental | Drug: Thiotepa, Drug: Busulfan (BU), Drug: Fludarabine, Procedure: Haploidentical Peripheral Blood Stem Cell TransplantationParticipants will receive a conditioning regimen consisting of thiotepa, busulfan, and fludarabine (TBF) followed by haploidentical peripheral blood stem cell transplantation. Thiotepa is administered on day -7, busulfan on days -4 and -3, and fludarabine on days -6 to -2. Donor stem cells are infused on day 0. Standard graft-versus-host disease prophylaxis and supportive care will be provided according to institutional guidelines. |
Who may qualify
- Ages
- 55 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Age 55 to 75 years
- Diagnosed with acute myeloid leukemia (AML) based on morphology, immunophenotyping, cytogenetics, or molecular testing
- First complete remission (CR1) or complete remission with incomplete hematologic recovery (CRi)
- Eligible for haploidentical hematopoietic stem cell transplantation
- Availability of a suitable haploidentical donor
Key exclusion criteria (7 listed)
- Secondary AML (including AML evolving from myelodysplastic syndrome or therapy-related AML)
- Active, uncontrolled infection
- Severe uncontrolled systemic disease (e.g., unstable cardiovascular disease, recent stroke, or severe organ dysfunction)
- HIV infection
- Active hepatitis B or C requiring antiviral treatment
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 |
|---|---|---|
| ThiotepaThiotepa is administered intravenously at a dose of 5 mg/kg on day -7 as part of the TBF conditioning regimen prior to haploidentical peripheral blood stem cell transplantation. | Drug | Arms Back to Arms and Interventions * Required * § Required if Study Start Date is on or after Janu |
| Busulfan (BU)Busulfan is administered intravenously at a dose of 3.2 mg/kg on days -4 and -3 as part of the TBF conditioning regimen. | Drug | Arms Back to Arms and Interventions * Required * § Required if Study Start Date is on or after Janu |
| FludarabineFludarabine is administered intravenously at a dose of 30 mg/m² daily from day -6 to day -2 as part of the conditioning regimen. | Drug | Arms Back to Arms and Interventions * Required * § Required if Study Start Date is on or after Janu |
| Haploidentical Peripheral Blood Stem Cell TransplantationHaploidentical peripheral blood stem cell transplantation is performed on day 0 following conditioning. Donor stem cells are infused, and standard graft-versus-host disease prophylaxis and supportive care are provided according to institutional protocols. | Procedure or surgery | Arms Back to Arms and Interventions * Required * § Required if Study Start Date is on or after Janu |
Primary outcome
1-year Relapse-Free Survival (RFS)
Time frame: 12 months after transplantation
Relapse-free survival (RFS) is defined as the time from transplantation to the first occurrence of disease relapse or death from any cause. Patients who are alive without relapse at the last follow-up will be censored.
4 secondary outcomes
Overall Survival (OS)
Time frame: 12 months after transplantation
Incidence of Acute Graft-Versus-Host Disease (aGVHD)
Time frame: Up to 180 days after transplantation
Incidence of Chronic Graft-Versus-Host Disease (cGVHD)
Time frame: 12 months after transplantation
Non-Relapse Mortality (NRM)
Time frame: 12 months after transplantation
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 May 2026.
- Individual participant dataSharing planned: No. Individual participant data (IPD) will not be shared due to patient privacy concerns and institutional data protection policies. De-identified data may be available from the corresponding investigator upon reasonable request and with appropriate institutional approvals.
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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.
Xianmin Song principal investigator
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
Questions about this trial
What is this clinical trial studying?
A study testing Thiotepa, Busulfan (BU) and Fludarabine in people with Acute Myeloid Leukemia, run by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine.
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 14 inclusion and 7 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 May 2026. Status can change between updates, so confirm with the study contact before making plans.
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 Xianmin Song, MD (+86-13501672508, shongxm@sjtu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
1-year Relapse-Free Survival (RFS) (time frame: 12 months after transplantation). 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 May 2026.