Acute Myeloid LeukemiaPhase 2NCT07562568
SHR2554/AZA + Overlapped TBF for High-risk/Relapsed Leukemia/MDS
A Phase 2 study testing SHR2554/AZA + Overlapped TBF and mBUCY conditioning regimen in people with Acute Myeloid Leukemia and Acute Lymphoblastic Leukemia, run by The First Affiliated Hospital of Soochow University at 1 site in China.
Trial summary
This study was designed as a prospective, multicenter, open-label, randomized controlled trial. Eligible participants were patients aged 15-60 years with high-risk or relapsed/refractory acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), or myelodysplastic neoplasms (MDS), diagnosed based on bone marrow… Read more about this study
- Who is it for?
- Ages 15 to 60 years
- Conditions the study is for
- Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, MDS
- What is being tested?
- SHR2554/AZA + Overlapped TBF and mBUCY conditioning regimen
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 160 participants
- Where
- 1 siteChina
- Study dates
- Jun 2026to Jun 2030
- Phase
- Phase 2
- 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 15 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age 15-60 years, of either sex.
- Diagnosis of AML or ALL according to the WHO 2022 criteria, with an indication for allogeneic hematopoietic stem cell transplantation: AML with high-risk genetics at diagnosis (risk stratification per ELN 2022) or relapsed/refractory AML (meeting any of the following: refractory-failure to achieve complete remission (CR) after two cycles of induction chemotherapy; relapse-reappearance of blasts in peripheral blood or bone marrow (≥5%) after first CR, or extramedullary relapse (EMR)). High-risk B-ALL at diagnosis (risk stratification per ELN 2022) or pre-transplant MRD-positive B-ALL. Confirmed T-ALL. History of central nervous system leukemia (CNSL) or pathologically confirmed extramedullary disease (EMD) during AML or ALL. Myelodysplastic neoplasms (MDS): IPSS score intermediate-2 or high; IPSS-R score high or very high; IPSS-M score high or very high.
- Availability of an appropriate HLA-matched donor.
- ECOG performance status 0-2.
- Adequate major organ function, defined as: Left ventricular ejection fraction ≥50%. Pulmonary function: DLCO ≥50% of predicted value. Liver function: ALT/AST ≤3×ULN, total bilirubin ≤2×ULN. Renal function: estimated creatinine clearance (CrCl) ≥60 mL/min.
- Ability to understand the study and voluntary signed informed consent.
Who cannot join
- Acute promyelocytic leukemia (APL);
- Active central nervous system leukemia;
- Prior allogeneic hematopoietic stem cell transplantation;
- Prior treatment with any EZH2 inhibitor;
- Uncontrolled active infection as assessed by the investigator;
- Myocardial infarction or unstable angina within the previous 6 months;
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- Known hypersensitivity to Zeprumetostat, azacitidine, or any excipient of the mBuCy regimen;
- Pregnant or breastfeeding women;
- Any other medical condition that, in the investigator's judgment, would preclude study enrollment.
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
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215006
About this study
Official title: A Prospective, Multicenter, Open-label, Randomized Controlled Trial of SHR2554 Plus Azacitidine in Overlapped Sequential Combination With TBF Conditioning Regimen in Patients With High-risk or Relapsed/Refractory Acute Leukemia and Myelodysplastic Neoplasms
This study was designed as a prospective, multicenter, open-label, randomized controlled trial. Eligible participants were patients aged 15-60 years with high-risk or relapsed/refractory acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), or myelodysplastic neoplasms (MDS), diagnosed based on bone marrow morphology, immunophenotyping, genetic testing, and treatment response assessment. The experimental group received SHR2554 combined with azacitidine as an overlapped sequential combination with the TBF conditioning regimen, whereas the control group received the mBuCy conditioning regimen, both followed by allogeneic hematopoietic stem cell transplantation (allo-HSCT). The primary endpoint was the 2-year cumulative incidence of relapse (CIR) after allo-HSCT. Secondary endpoints included 2-year overall survival (OS), 2-year disease-free survival (DFS), transplant-related mortality (TRM), the incidence of acute and chronic graft-versus-host disease (GVHD), and safety profiles.
- Conditions
- Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, MDS, Transplantation, Stem Cell, Conditioning Therapy, SHR2554
- Being tested
- SHR2554/AZA + Overlapped TBF, mBUCY conditioning regimen
- Medical subject headings
- Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SHR2554/AZA + Overlapped TBFCombination product
Also called Experimental Group
SHR2554/AZA + Overlapped TBF conditioning Regimen and GVHD Prophylaxis of Haploid transplantation (Haplo-HSCT) and unrelated donor transplantation (UDT) : Cyclosporine A (CsA) + Mycophenolate mofetil dispersible tablets (MMF) + short-term low-dose methotrexate (MTX) + rabbit anti-human antithymocyte globulin (ATG) or GVHD Prophylaxis of Matched Sibling Donor Hematopoietic Stem Cell Transplantation (MSD-HSCT)：Cyclosporine A (CsA) + short-term low-dose methotrexate (MTX), then stem cells are infused into patient's blood.
mBUCY conditioning regimenDrug
Also called Control Group
mBUCY conditioning Regimen and GVHD Prophylaxis of Haploid transplantation (Haplo-HSCT) and unrelated donor transplantation (UDT): Cyclosporine A (CsA) + Mycophenolate mofetil dispersible tablets (MMF) + short-term low-dose methotrexate (MTX) + rabbit anti-human antithymocyte globulin (ATG) or GVHD Prophylaxis of Matched Sibling Donor Hematopoietic Stem Cell Transplantation (MSD-HSCT)：Cyclosporine A (CsA) + short-term low-dose methotrexate (MTX), then stem cells are infused into patient's blood.
| Study group | Role | What they receive |
|---|---|---|
| SHR2554/AZA + Overlapped TBF | Experimental | SHR2554 350 mg BID and azacitidine 75 mg/m² daily on days -9 to -3, overlapping with TBF conditioning: thiotepa 5 mg/kg on days -8 and -7; cytarabine 2 g/m² q12h on day -6; busulfan 0.8 mg/kg q6h on days -5, -4, -3 (total 3.2 mg/kg/day); fludarabine 30 mg/m²/day on days -6 to -2. Receives: Combination Product: SHR2554/AZA + Overlapped TBF |
| mBUCY conditioning Regimen Group | Active comparator | semustine 250 mg/m² on day -8; cytarabine 2 g/m² q12h on day -7; busulfan 0.8 mg/kg q6h on days -6, -5, -4 (total 3.2 mg/kg/day); cyclophosphamide 1.8 g/m²/day on days -3 and -2. Receives: Drug: mBUCY conditioning regimen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cumulative incidence of relapse（CIR）
It is measured the date from complete remission after transplantation to hematological relapse or molecular relapse was recorded. Patients who had no relapse at the last follow-up were considered as censored data, and non-relapse death was regarded as a competing risk event.
Measured: 2 years
Secondary outcomes (7)
Time period for hematopoietic reconstruction
Granulogenetic hematopoietic reconstitution: The absolute neutrophil count in peripheral blood needs to reach or exceed 0.5×10^9 cells/L for 3 consecutive days. Megakaryotic hematopoietic reconstitution: platelet count needs to be more than 20×10^9/L and does not rely on platelet transfusion for 7 consecutive days.
Measured: 24 weeks
graft-versus-host disease (GvHD)
incidence and severity of acute (aGvHD) and chronic graft-versus-host disease (cGvHD) (aGvHD refer to Glucksberg Criteria and cGvHD refer to the National Institutes of Health Consensus)
Measured: 2 years
Overall survival（OS）
It is measured from the date of entry into this trial to the date of death from any cause; patients not known to have died at last follow-up are censored on the date they were last known to be alive.
Measured: 2 years
Disease-free survival（DFS）
It is measured from the time from randomization to the first of relapse or death.
Measured: 2 years
transplant related mortality (TRM)
cumulative incidence of transplant related mortality
Measured: 2 years
Regimen related toxicity
Number of participants with regimen related toxicity as assessed by CTCAE v5.0
Measured: 2 years
veno-occlusive disease (VOD)
incidence of veno-occlusive disease (VOD) events (refer to modified Seattle Criteria of VOD)
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 May 2026
- Study startJun 2026 (estimated)
- Primary completionJun 2030 (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 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 160 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Limin Liu,MD, The First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07562568
- Sponsor's protocol ID
- 20260414125804551
- Status last verified
- Apr 2026
- First submitted
- 26 Apr 2026
- Last updated
- 1 May 2026
- Results posted
- No
- 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 1 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Multicenter, Open-label, Randomized Controlled Trial of SHR2554 Plus Azacitidine in Overlapped Sequential Combination With TBF Conditioning Regimen in Patients With High-risk or Relapsed/Refractory Acute Leukemia and Myelodysplastic Neoplasms
- ClinicalTrials.gov ID
- NCT07562568
- Acronym / sponsor ID
- 20260414125804551
- Conditions
- Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, MDS, Transplantation, Stem Cell, Conditioning Therapy, SHR2554
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 160 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Study start
- Jun 2026 (estimated)
- Primary completion
- Jun 2030 (estimated)
- Study completion
- Jun 2030 (estimated)
- First posted
- 1 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.
This study was designed as a prospective, multicenter, open-label, randomized controlled trial. Eligible participants were patients aged 15-60 years with high-risk or relapsed/refractory acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), or myelodysplastic neoplasms (MDS), diagnosed based on bone marrow morphology, immunophenotyping, genetic testing, and treatment response assessment. The experimental group received SHR2554 combined with azacitidine as an overlapped sequential combination with the TBF conditioning regimen, whereas the control group received the mBuCy conditioning regimen, both followed by allogeneic hematopoietic stem cell transplantation (allo-HSCT). The primary endpoint was the 2-year cumulative incidence of relapse (CIR) after allo-HSCT. Secondary endpoints included 2-year overall survival (OS), 2-year disease-free survival (DFS), transplant-related mortality (TRM), the incidence of acute and chronic graft-versus-host disease (GVHD), and safety profiles.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| SHR2554/AZA + Overlapped TBF | Experimental | Combination Product: SHR2554/AZA + Overlapped TBFSHR2554 350 mg BID and azacitidine 75 mg/m² daily on days -9 to -3, overlapping with TBF conditioning: thiotepa 5 mg/kg on days -8 and -7; cytarabine 2 g/m² q12h on day -6; busulfan 0.8 mg/kg q6h on days -5, -4, -3 (total 3.2 mg/kg/day); fludarabine 30 mg/m²/day on days -6 to -2. |
| mBUCY conditioning Regimen Group | Active comparator | Drug: mBUCY conditioning regimensemustine 250 mg/m² on day -8; cytarabine 2 g/m² q12h on day -7; busulfan 0.8 mg/kg q6h on days -6, -5, -4 (total 3.2 mg/kg/day); cyclophosphamide 1.8 g/m²/day on days -3 and -2. |
Who may qualify
- Ages
- 15 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Age 15-60 years, of either sex.
- Diagnosis of AML or ALL according to the WHO 2022 criteria, with an indication for allogeneic hematopoietic stem cell transplantation: AML with high-risk genetics at diagnosis (risk stratification per ELN 2022) or relapsed/refractory AML (meeting any of the following: refractory-failure to achieve complete remission (CR) after two cycles of induction chemotherapy; relapse-reappearance of blasts in peripheral blood or bone marrow (≥5%) after first CR, or extramedullary relapse (EMR)). High-risk B-ALL at diagnosis (risk stratification per ELN 2022) or pre-transplant MRD-positive B-ALL. Confirmed T-ALL. History of central nervous system leukemia (CNSL) or pathologically confirmed extramedullary disease (EMD) during AML or ALL. Myelodysplastic neoplasms (MDS): IPSS score intermediate-2 or high; IPSS-R score high or very high; IPSS-M score high or very high.
- Availability of an appropriate HLA-matched donor.
- ECOG performance status 0-2.
- Adequate major organ function, defined as: Left ventricular ejection fraction ≥50%. Pulmonary function: DLCO ≥50% of predicted value. Liver function: ALT/AST ≤3×ULN, total bilirubin ≤2×ULN. Renal function: estimated creatinine clearance (CrCl) ≥60 mL/min.
Key exclusion criteria (9 listed)
- Acute promyelocytic leukemia (APL);
- Active central nervous system leukemia;
- Prior allogeneic hematopoietic stem cell transplantation;
- Prior treatment with any EZH2 inhibitor;
- Uncontrolled active infection as assessed by the investigator;
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 |
|---|---|---|
| SHR2554/AZA + Overlapped TBFAlso: Experimental GroupSHR2554/AZA + Overlapped TBF conditioning Regimen and GVHD Prophylaxis of Haploid transplantation (Haplo-HSCT) and unrelated donor transplantation (UDT) : Cyclosporine A (CsA) + Mycophenolate mofetil dispersible tablets (MMF) + short-term low-dose methotrexate (MTX) + rabbit anti-human antithymocyte globulin (ATG) or GVHD Prophylaxis of Matched Sibling Donor Hematopoietic Stem Cell Transplantation (MSD-HSCT)：Cyclosporine A (CsA) + short-term low-dose methotrexate (MTX), then stem cells are infused into patient's blood. | Combination product | SHR2554/AZA + Overlapped TBF |
| mBUCY conditioning regimenAlso: Control GroupmBUCY conditioning Regimen and GVHD Prophylaxis of Haploid transplantation (Haplo-HSCT) and unrelated donor transplantation (UDT): Cyclosporine A (CsA) + Mycophenolate mofetil dispersible tablets (MMF) + short-term low-dose methotrexate (MTX) + rabbit anti-human antithymocyte globulin (ATG) or GVHD Prophylaxis of Matched Sibling Donor Hematopoietic Stem Cell Transplantation (MSD-HSCT)：Cyclosporine A (CsA) + short-term low-dose methotrexate (MTX), then stem cells are infused into patient's blood. | Drug | mBUCY conditioning Regimen Group |
Primary outcome
Cumulative incidence of relapse（CIR）
Time frame: 2 years
It is measured the date from complete remission after transplantation to hematological relapse or molecular relapse was recorded. Patients who had no relapse at the last follow-up were considered as censored data, and non-relapse death was regarded as a competing risk event.
7 secondary outcomes
Time period for hematopoietic reconstruction
Time frame: 24 weeks
graft-versus-host disease (GvHD)
Time frame: 2 years
Overall survival（OS）
Time frame: 2 years
Disease-free survival（DFS）
Time frame: 2 years
transplant related mortality (TRM)
Time frame: 2 years
Regimen related toxicity
Time frame: 2 years
veno-occlusive disease (VOD)
Time frame: 2 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 1 May 2026.
- 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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing SHR2554/AZA + Overlapped TBF and mBUCY conditioning regimen in people with Acute Myeloid Leukemia and Acute Lymphoblastic 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 6 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: opening soon, as of the sponsor's last update on 1 May 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 LIMIN LIU, MD (+86-512-6778183, Liminliu1006@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cumulative incidence of relapse（CIR） (time frame: 2 years). 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 1 May 2026.