Relapsed or Refractory Acute Myeloid LeukemiaNCT06879847
A Single-arm, Prospective Study of TBI + BUMEL As a Conditioning Regimen for Salvage HSCT in Patients with R/R AML
A study testing Salvage Allogeneic Hematopoietic Stem Cell Transplantation in people with Relapsed or Refractory Acute Myeloid Leukemia, run by The First Affiliated Hospital of Soochow University at 1 site in China.
Trial summary
Acute myeloid leukemia (AML) is one of the hematologic malignancies, for which patients typically undergo chemotherapy to achieve complete remission. However, approximately 30% of patients fail to respond to initial treatment, and many experience relapse after achieving remission. Read more about this study
- Who is it for?
- Ages 14 to 70 years
- Conditions the study is for
- Relapsed or Refractory Acute Myeloid Leukemia
- What is being tested?
- Salvage Allogeneic Hematopoietic Stem Cell Transplantation
- Type of treatment
- Pill or oral medicine, Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteChina
- Study dates
- 1 Dec 2024to 30 Nov 2027
- 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 14 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age between 14 and 70 years (inclusive of the age limits);
- Patients diagnosed with relapsed/refractory (R/R) AML, meeting the World Health Organization (WHO) 2016 AML diagnostic criteria, must meet one of the following definitions:
- Relapsed AML: Leukemic cells reappear in peripheral blood or bone marrow blasts >5% after achieving complete remission (CR, CRi) (excluding other causes such as bone marrow regeneration following consolidation chemotherapy), or extramedullary leukemic infiltration occurs.
- Refractory AML:Initial cases that do not respond to two courses of standard treatment. Relapse within 12 months after consolidation therapy. Relapse after 12 months with no response to conventional chemotherapy. Two or more relapses. Persistent extramedullary leukemia.
- Heart, liver, and kidney function must meet the following criteria:
- Alanine aminotransferase (ALT) and Aspartate aminotransferase (AST) ≤ 3× the upper limit of normal (ULN); Total bilirubin ≤ 3× ULN; Serum creatinine ≤ 2× ULN or creatinine clearance ≥ 40 mL/min; Left ventricular ejection fraction (LVEF), as measured by echocardiography or multi-gated acquisition (MUGA) scan, must be within the normal range (>50%).
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- Availability of a suitable allogeneic donor;
- Life expectancy of ≥3 months;
- Karnofsky Performance Status (KPS) ≥ 60%, Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2;
- The patient understands the study protocol and voluntarily signs the informed consent form.
Who cannot join
- Patients had serious adverse reactions to investigational drugs such as allergies;
- Patients with a history of immunodeficiency, or other acquired or congenital diseases, immunodeficiency diseases, and a history of organ transplantation;
- Patients with hypertension, ventricular arrhythmia requiring clinical intervention, acute coronary syndrome, congestive heart failure, stroke, or other grade III or higher cardiovascular events within 6 months;
- Patients received Class II or higher surgery within 4 weeks prior to enrollment;
- Patient has an active and difficult-to-control infection, including but not limited to active fungal, bacterial, or viral infections that require systemic treatment, such as active HIV, hepatitis B or C;
- Patient has active central nervous system leukemia infiltration;
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- Pregnant or lactating patients;
- Patient is currently participating in another clinical studies;
- Other conditions where the investigator deems the patient unsuitable for inclusion.
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
Hematology Department, The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215006
Depei Wu, Prof., 13951102021, wudepei@suda.edu.cn
Xiaojin Wu, Prof., 13951102021, wuxiaojin@suda.edu.cn
About this study
Official title: A Single-arm, Prospective Clinical Study on the Efficacy of Total Body Irradiation Combined with Busulfan and Melphalan As Conditioning Regimen for Patients with Relapsed/Refractory Acute Myeloid Leukemia Undergoing Salvage HSCT
Acute myeloid leukemia (AML) is one of the hematologic malignancies, for which patients typically undergo chemotherapy to achieve complete remission. However, approximately 30% of patients fail to respond to initial treatment, and many experience relapse after achieving remission. For patients with relapsed or refractory AML, allogeneic hematopoietic stem cell transplantation (HSCT) offers a potentially curative option. Sibling-matched HSCT has demonstrated a disease-free survival rate of 20-30%, while unrelated donor transplants yield an overall survival rate of approximately 22%. Haploidentical transplantation is a viable alternative for patients lacking a sibling donor. A 2019 study involving 1,693 patients with relapsed/refractory (R/R) AML revealed that haploidentical transplants yielded outcomes comparable to other transplant modalities, including HLA-matched and 9/10 matched unrelated donor transplants, thus supporting haploidentical transplantation as a viable therapeutic option.
The conditioning regimen is a critical component of the transplantation. In China, the modified BU/CY conditioning regimen, which combines busulfan (BU) and cyclophosphamide (CTX), is widely utilized for tumor cytoreduction and immunosuppression. Some centers also employ post-transplant cyclophosphamide (PTCy) to mitigate the risk of graft-versus-host disease (GVHD). Despite advances, relapse remains a significant challenge. Optimizing conditioning regimens to enhance tumor cell targeting and achieve deeper remission is crucial. Additionally, many patients are unfit due to prior chemotherapy, infections, and organ dysfunction, which may make them unable to tolerate high-intensity conditioning.
Read more
Recent studies suggest that melphalan (MEL)-based conditioning regimens may offer advantages over CTX-based protocols. While total body irradiation (TBI) has been traditionally used in conditioning for HSCT, it is associated with considerable organ toxicity.
A low-dose TBI regimen combined with BU+MEL represents a promising conditioning regimen for R/R AML. In preliminary studies, 7 patients treated with this regimen successfully achieved hematopoietic stem cell engraftment. Building on these results, a clinical study is planned to evaluate further the safety and efficacy of the TBI+BUMEL (IBM) conditioning regimen in relapsed/refractory AML, with a focus on improving engraftment rates, reducing relapse rates, minimizing GVHD incidence, and enhancing overall survival outcomes.
Read the detailed description
The aim of this study is to observe the efficacy-related factors and adverse events of the TBI combined with BUMEL regimen as conditioning for allogeneic hematopoietic stem cell transplantation in patients with relapsed/refractory acute myeloid leukemia .
- Being tested
- Salvage Allogeneic Hematopoietic Stem Cell Transplantation
- Keywords
- Salvage Allogeneic Hematopoietic Stem Cell Transplantation, Relapsed or Refractory Acute Myeloid Leukemia, Conditioning Regimen, Total Body Irradiation, Melphalan
- Medical subject headings
- Recurrence, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Salvage Allogeneic Hematopoietic Stem Cell TransplantationProcedure or surgery
- TBI+BUMEL (IBM) Conditioning Regimen Day -7: Semustine (Me-CCNU) 250mg/m², orally, Day -7; Day -6: Total Body Irradiation (TBI) 4Gy, in two parts, Day -6; Day -5 to Day -4: Busulfan (Bu) 3.2mg/kg/day, administered in four divided doses, IV infusion; Day -3 to Day -2: Melphalan (Mel) 50mg/m²/day, IV infusion
- Donor Stem Cell Infusion (Hematopoietic Stem Cell Transplantation) Day 0: Intravenous infusion of donor hematopoietic stem cells (MNC ≥ 8×10⁸/kg, CD34+ cells ≥ 4×10⁶/kg).
| Study group | Role | What they receive |
|---|---|---|
| 40 patients with relapsed/refractory acute myeloid leukemia who undergo HSCT | Experimental | patients undergo HSCT using TBI + BUMEL as a conditioning regimen Receives: Procedure: Salvage Allogeneic Hematopoietic Stem Cell Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
The cumulative incidence of neutrophil engraftment and platelet engraftment
Neutrophil and platelet engraftment is defined as the first occurrence of 3 consecutive days with an absolute neutrophil count of at least 0.5×109/L and a platelet count of over 20×109/L for 7 consecutive days without transfusion support.
Measured: on day 28±7 following HSCT
The time to reconstitution of hematopoiesis
recovery of hemopoietic function after treatment
Measured: on day 28±7 following HSCT
The cumulative incidence of transplant-related mortality (TRM)
Transplant-related mortality was defined as mortality due to any cause other than disease progression within 100 days of transplantation.
Measured: within 100 days following HSCT
Secondary outcomes (4)
The cumulative incidence and grade of graft-versus-host disease (GVHD)
Graft-versus-host disease (GVHD) is a medical complication following the receipt of transplanted tissue from a genetically different person.
Measured: within 1 year following HSCT
The cumulative incidence of relapse
We defined relapse as any clinical evidence of progression or recurrence of original diseases.
Measured: within 1 year following HSCT
Overall survival rate
We estimated OS from the time of transplant until the date of death of any cause or last follow-up for patients still alive.
Measured: within 1 year following HSCT
The cumulative incidence of adverse events
Following extraction of the tumor, a blood vessel burst causing increased blood loss during the procedure, an adverse event the surgeon did not expect.
Measured: within 1 year following HSCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Mar 2025
- Study start1 Dec 2024
- Primary completion1 Nov 2027 (estimated)
- Study completion30 Nov 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Relapsed or Refractory Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by The First Affiliated Hospital of Soochow UniversityThe 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
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Depei Wu, Prof., Study chair, The First Affiliated Hospital of Soochow UniversityXiaojin Wu, Prof., Study chair, The First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06879847
- Sponsor's protocol ID
- SOOCHOW-WXJ-2024-475
- Status last verified
- Mar 2025
- First submitted
- 11 Mar 2025
- Last updated
- 17 Mar 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-arm, Prospective Clinical Study on the Efficacy of Total Body Irradiation Combined with Busulfan and Melphalan As Conditioning Regimen for Patients with Relapsed/Refractory Acute Myeloid Leukemia Undergoing Salvage HSCT
- ClinicalTrials.gov ID
- NCT06879847
- Acronym / sponsor ID
- SOOCHOW-WXJ-2024-475
- Keywords
- Salvage Allogeneic Hematopoietic Stem Cell Transplantation, Relapsed or Refractory Acute Myeloid Leukemia, Conditioning Regimen, Total Body Irradiation, Melphalan
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Study start
- 1 Dec 2024 (actual)
- Primary completion
- 1 Nov 2027 (estimated)
- Study completion
- 30 Nov 2027 (estimated)
- First posted
- 17 Mar 2025 (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 one of the hematologic malignancies, for which patients typically undergo chemotherapy to achieve complete remission. However, approximately 30% of patients fail to respond to initial treatment, and many experience relapse after achieving remission. For patients with relapsed or refractory AML, allogeneic hematopoietic stem cell transplantation (HSCT) offers a potentially curative option. Sibling-matched HSCT has demonstrated a disease-free survival rate of 20-30%, while unrelated donor transplants yield an overall survival rate of approximately 22%. Haploidentical transplantation is a viable alternative for patients lacking a sibling donor. A 2019 study involving 1,693 patients with relapsed/refractory (R/R) AML revealed that haploidentical transplants yielded outcomes comparable to other transplant modalities, including HLA-matched and 9/10 matched unrelated donor transplants, thus supporting haploidentical transplantation as a viable therapeutic option.
The conditioning regimen is a critical component of the transplantation. In China, the modified BU/CY conditioning regimen, which combines busulfan (BU) and cyclophosphamide (CTX), is widely utilized for tumor cytoreduction and immunosuppression. Some centers also employ post-transplant cyclophosphamide (PTCy) to mitigate the risk of graft-versus-host disease (GVHD). Despite advances, relapse remains a significant challenge. Optimizing conditioning regimens to enhance tumor cell targeting and achieve deeper remission is crucial. Additionally, many patients are unfit due to prior chemotherapy, infections, and organ dysfunction, which may make them unable to tolerate high-intensity conditioning.
Recent studies suggest that melphalan (MEL)-based conditioning regimens may offer advantages over CTX-based protocols. While total body irradiation (TBI) has been traditionally used in conditioning for HSCT, it is associated with considerable organ toxicity.
A low-dose TBI regimen combined with BU+MEL represents a promising conditioning regimen for R/R AML. In preliminary studies, 7 patients treated with this regimen successfully achieved hematopoietic stem cell engraftment. Building on these results, a clinical study is planned to evaluate further the safety and efficacy of the TBI+BUMEL (IBM) conditioning regimen in relapsed/refractory AML, with a focus on improving engraftment rates, reducing relapse rates, minimizing GVHD incidence, and enhancing overall survival outcomes.
Detailed description
The aim of this study is to observe the efficacy-related factors and adverse events of the TBI combined with BUMEL regimen as conditioning for allogeneic hematopoietic stem cell transplantation in patients with relapsed/refractory acute myeloid leukemia .
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 40 patients with relapsed/refractory acute myeloid leukemia who undergo HSCT | Experimental | Procedure: Salvage Allogeneic Hematopoietic Stem Cell Transplantationpatients undergo HSCT using TBI + BUMEL as a conditioning regimen |
Who may qualify
- Ages
- 14 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Age between 14 and 70 years (inclusive of the age limits);
- Patients diagnosed with relapsed/refractory (R/R) AML, meeting the World Health Organization (WHO) 2016 AML diagnostic criteria, must meet one of the following definitions:
- Relapsed AML: Leukemic cells reappear in peripheral blood or bone marrow blasts >5% after achieving complete remission (CR, CRi) (excluding other causes such as bone marrow regeneration following consolidation chemotherapy), or extramedullary leukemic infiltration occurs.
- Refractory AML:Initial cases that do not respond to two courses of standard treatment. Relapse within 12 months after consolidation therapy. Relapse after 12 months with no response to conventional chemotherapy. Two or more relapses. Persistent extramedullary leukemia.
- Heart, liver, and kidney function must meet the following criteria:
Key exclusion criteria (9 listed)
- Patients had serious adverse reactions to investigational drugs such as allergies;
- Patients with a history of immunodeficiency, or other acquired or congenital diseases, immunodeficiency diseases, and a history of organ transplantation;
- Patients with hypertension, ventricular arrhythmia requiring clinical intervention, acute coronary syndrome, congestive heart failure, stroke, or other grade III or higher cardiovascular events within 6 months;
- Patients received Class II or higher surgery within 4 weeks prior to enrollment;
- Patient has an active and difficult-to-control infection, including but not limited to active fungal, bacterial, or viral infections that require systemic treatment, such as active HIV, hepatitis B or C;
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 |
|---|---|---|
| Salvage Allogeneic Hematopoietic Stem Cell Transplantation1. TBI+BUMEL (IBM) Conditioning Regimen Day -7: Semustine (Me-CCNU) 250mg/m², orally, Day -7; Day -6: Total Body Irradiation (TBI) 4Gy, in two parts, Day -6; Day -5 to Day -4: Busulfan (Bu) 3.2mg/kg/day, administered in four divided doses, IV infusion; Day -3 to Day -2: Melphalan (Mel) 50mg/m²/day, IV infusion 2. Donor Stem Cell Infusion (Hematopoietic Stem Cell Transplantation) Day 0: Intravenous infusion of donor hematopoietic stem cells (MNC ≥ 8×10⁸/kg, CD34+ cells ≥ 4×10⁶/kg). | Procedure or surgery | 40 patients with relapsed/refractory acute myeloid leukemia who undergo HSCT |
Primary outcome
The cumulative incidence of neutrophil engraftment and platelet engraftment
Time frame: on day 28±7 following HSCT
Neutrophil and platelet engraftment is defined as the first occurrence of 3 consecutive days with an absolute neutrophil count of at least 0.5×109/L and a platelet count of over 20×109/L for 7 consecutive days without transfusion support.
The time to reconstitution of hematopoiesis
Time frame: on day 28±7 following HSCT
recovery of hemopoietic function after treatment
The cumulative incidence of transplant-related mortality (TRM)
Time frame: within 100 days following HSCT
Transplant-related mortality was defined as mortality due to any cause other than disease progression within 100 days of transplantation.
4 secondary outcomes
The cumulative incidence and grade of graft-versus-host disease (GVHD)
Time frame: within 1 year following HSCT
The cumulative incidence of relapse
Time frame: within 1 year following HSCT
Overall survival rate
Time frame: within 1 year following HSCT
The cumulative incidence of adverse events
Time frame: within 1 year following HSCT
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 17 Mar 2025.
- Individual participant dataSharing planned: No
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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.
Depei Wu, Prof. study chair
The First Affiliated Hospital of Soochow University
Xiaojin Wu, Prof. study chair
The First Affiliated Hospital of Soochow University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Salvage Allogeneic Hematopoietic Stem Cell Transplantation in people with Relapsed or Refractory Acute Myeloid Leukemia, run by The First Affiliated Hospital of Soochow University 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 10 inclusion and 9 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 17 Mar 2025. 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 Xiaojin Wu, Prof. (13057493105, wuxiaojin@suda.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The cumulative incidence of neutrophil engraftment and platelet engraftment (time frame: on day 28±7 following HSCT). The study lists 3 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 17 Mar 2025.