Malignant Hematological DiseasesNCT06548958
A Single-Arm, Prospective Study of TBI+BUMEL as Conditioning for SCT2 in Patients With Malignant Hematologic Diseases
A study testing Second Stem Cell Transplantation (SCT2) in people with Malignant Hematological Diseases, run by The First Affiliated Hospital of Soochow University at 1 site in China.
Trial summary
Each year, over 20,000 patients in China undergo hematopoietic stem cell transplantation (HSCT). Unfortunately, the prognosis in patients with disease relapse or graft failure is often inferior. A second allogeneic hematopoietic stem cell transplantation (SCT2) has emerged as a vital salvage therapy option. Read more about this study
- Who is it for?
- Ages 14 to 70 years
- Conditions the study is for
- Malignant Hematological Diseases
- What is being tested?
- Second Stem Cell Transplantation (SCT2)
- 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
- 10 Jul 2024to 30 Jun 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
- Gender is not limited, patients between 14 to 70 years old (including critical value);
- Malignant hematological diseases (acute/chronic myeloid leukemia, acute lymphoblastic leukemia, myelodysplastic syndrome, malignant lymphoma, etc.) diagnosed by bone marrow aspiration or biopsy according to the WHO diagnostic criteria, after the first hematopoietic stem cell transplantation, due to various reasons (including but not limited to disease relapse or graft failure) have indications for a second hematopoietic stem cell transplantation;
- The indexes of cardiac function, liver and kidney function were within the following limits: (1) Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3× Upper limit of normal (ULN); (2)Total bilirubin ≤ 3×ULN; (3) Serum creatinine ≤ 2×ULN or creatinine clearance ≥ 40mL/min; (4) Left ventricular ejection fraction (LVEF) as measured by echocardiography or multi-gated acquisition (MUGA) scan is within the normal range (> 50%);
- Having a suitable allogeneic hematopoietic stem cell donor;
- Expected survival ≥3 months;
- Karnofsky (KPS) score ≥60%, Eastern Tumor Cooperative group (ECOG) status ≤ 2;
- Patient fully understood the nature of the study, and voluntarily participates and signs informed consent.
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;
- Received Class II or higher surgery within 4 weeks prior to enrollment;
- Patients with active viral infections;
- Pregnant or lactating patients;
- The patient is currently participating in another clinical studies;
- Patients deemed unsuitable for inclusion by other investigators.
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
About this study
Official title: A Single-Arm, Prospective Clinical Study of Total Body Irradiation Combined With Busulfan and Melphalan (TBI+BUMEL) as a Conditioning Regimen for Second Hematopoietic Stem Cell Transplantation in Patients With Malignant Hematologic Diseases
Each year, over 20,000 patients in China undergo hematopoietic stem cell transplantation (HSCT). Unfortunately, the prognosis in patients with disease relapse or graft failure is often inferior. A second allogeneic hematopoietic stem cell transplantation (SCT2) has emerged as a vital salvage therapy option. Despite varying prognoses, most patients undergoing SCT2 have a five-year overall survival (OS) rate of less than 30%. The primary challenges of SCT2 include treatment strategy, immune regulation, complication management, and transplantation technique improvements. By optimizing these key aspects, SCT2 can effectively address issues that arose after the first transplant, reduce complications, and provide more effective treatment for patients.
Clinical practice indicates that SCT2 is crucial in treating various hematologic diseases. For patients who failed the first transplant (SCT1), SCT2 can more effectively treat the primary disease, provide timely hematopoietic engraftment, extend survival time, and improve the quality of life. Additionally, the successful application of SCT2 provides clinicians with more treatment options and hope. Currently, the modified BU/CY conditioning regimen, which consists of busulfan (BU) and cyclophosphamide (CY), is commonly used in SCT1 in China. However, for patients who relapse after SCT1, these drugs may become ineffective, and the physical condition often worsens, with a higher likelihood of infections and organ dysfunction. Therefore, finding new conditioning regimens is crucial.
Read more
Studies have shown that a melphalan (MEL)-based conditioning regimen may have better outcomes for patients with acute myeloid leukemia (AML) compared to a Cy-based regimen. The Conditioning regimen that includes total body irradiation (TBI) has also been considered effective for patients with acute leukemia. Thus, low-dose TBI combined with a BU + MEL regimen could be a promising conditioning regimen for SCT2. In the investigators' preliminary studies, three patients who underwent SCT2 with this regimen successfully achieved engraftment and were discharged.
Based on this, the investigators plan to conduct a clinical study to observe the effects of the TBI+BUMEL regimen combined with SCT2 on the engraftment rate, disease relapse rate, GVHD incidence, and survival rate in patients with malignant hematologic diseases who relapsed after SCT1.
Read the detailed description
The primary challenges of SCT2 include:
- treatment strategy: eliminating residual tumor cells and addressing graft failure;
- immune regulation: adjusting the immune system to ensure successful engraftment of new stem cells;
- complication management: due to the poor physical condition of patients after the first transplant (SCT1), the risk of complications after SCT2 is high. This necessitates special attention to the prevention and management of post-transplant complications;
- transplantation technique improvements: more rigorous donor screening, HLA typing, monitoring, and supportive treatment during the transplant procedure.
- Conditions
- Malignant Hematological Diseases
- Being tested
- Second Stem Cell Transplantation (SCT2)
- Keywords
- Second Allogeneic Hematopoietic Stem Cell Transplantation, Malignant Hematological Diseases, Total Body Irradiation, Melphalan, Conditioning Regimen
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Second Stem Cell Transplantation (SCT2)Procedure or surgery
- Conditioning Regimen Day -7: Semustine (Me-CCNU) 250mg/m², orally; Day -6: Total Body Irradiation (TBI) 4Gy; 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 (Second 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 malignant hematological diseases who undergo SCT2 | Experimental | patients undergo SCT2 using TBI + BUMEL as a conditioning regimen Receives: Procedure: Second Stem Cell Transplantation (SCT2) |
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 SCT2
The time to reconstitution of hematopoiesis
Recovery of hemopoietic function after treatment
Measured: on day 28±7 following SCT2
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 SCT2
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 SCT2
The cumulative incidence of relapse
We defined relapse as any clinical evidence of progression or recurrence of original diseases.
Measured: within 1 year following SCT2
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 SCT2
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 SCT2
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Aug 2024
- Study start10 Jul 2024
- Primary completion30 Mar 2027 (estimated)
- Study completion30 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Malignant Hematological DiseasesStudies 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., Principal investigator, The First Affiliated Hospital of Soochow UniversityXiaojin Wu, Prof., Principal investigator, The First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06548958
- Sponsor's protocol ID
- SOOCHOW-WXJ-2024-248
- Status last verified
- Jul 2024
- First submitted
- 8 Aug 2024
- Last updated
- 13 Mar 2025
- 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 13 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-Arm, Prospective Clinical Study of Total Body Irradiation Combined With Busulfan and Melphalan (TBI+BUMEL) as a Conditioning Regimen for Second Hematopoietic Stem Cell Transplantation in Patients With Malignant Hematologic Diseases
- ClinicalTrials.gov ID
- NCT06548958
- Acronym / sponsor ID
- SOOCHOW-WXJ-2024-248
- Conditions
- Malignant Hematological Diseases
- Keywords
- Second Allogeneic Hematopoietic Stem Cell Transplantation, Malignant Hematological Diseases, Total Body Irradiation, Melphalan, Conditioning Regimen
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Study start
- 10 Jul 2024 (actual)
- Primary completion
- 30 Mar 2027 (estimated)
- Study completion
- 30 Jun 2027 (estimated)
- First posted
- 12 Aug 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.
Each year, over 20,000 patients in China undergo hematopoietic stem cell transplantation (HSCT). Unfortunately, the prognosis in patients with disease relapse or graft failure is often inferior. A second allogeneic hematopoietic stem cell transplantation (SCT2) has emerged as a vital salvage therapy option. Despite varying prognoses, most patients undergoing SCT2 have a five-year overall survival (OS) rate of less than 30%. The primary challenges of SCT2 include treatment strategy, immune regulation, complication management, and transplantation technique improvements. By optimizing these key aspects, SCT2 can effectively address issues that arose after the first transplant, reduce complications, and provide more effective treatment for patients.
Clinical practice indicates that SCT2 is crucial in treating various hematologic diseases. For patients who failed the first transplant (SCT1), SCT2 can more effectively treat the primary disease, provide timely hematopoietic engraftment, extend survival time, and improve the quality of life. Additionally, the successful application of SCT2 provides clinicians with more treatment options and hope. Currently, the modified BU/CY conditioning regimen, which consists of busulfan (BU) and cyclophosphamide (CY), is commonly used in SCT1 in China. However, for patients who relapse after SCT1, these drugs may become ineffective, and the physical condition often worsens, with a higher likelihood of infections and organ dysfunction. Therefore, finding new conditioning regimens is crucial.
Studies have shown that a melphalan (MEL)-based conditioning regimen may have better outcomes for patients with acute myeloid leukemia (AML) compared to a Cy-based regimen. The Conditioning regimen that includes total body irradiation (TBI) has also been considered effective for patients with acute leukemia. Thus, low-dose TBI combined with a BU + MEL regimen could be a promising conditioning regimen for SCT2. In the investigators' preliminary studies, three patients who underwent SCT2 with this regimen successfully achieved engraftment and were discharged.
Based on this, the investigators plan to conduct a clinical study to observe the effects of the TBI+BUMEL regimen combined with SCT2 on the engraftment rate, disease relapse rate, GVHD incidence, and survival rate in patients with malignant hematologic diseases who relapsed after SCT1.
Detailed description
The primary challenges of SCT2 include:
- treatment strategy: eliminating residual tumor cells and addressing graft failure;
- immune regulation: adjusting the immune system to ensure successful engraftment of new stem cells;
- complication management: due to the poor physical condition of patients after the first transplant (SCT1), the risk of complications after SCT2 is high. This necessitates special attention to the prevention and management of post-transplant complications;
- transplantation technique improvements: more rigorous donor screening, HLA typing, monitoring, and supportive treatment during the transplant procedure.
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 malignant hematological diseases who undergo SCT2 | Experimental | Procedure: Second Stem Cell Transplantation (SCT2)patients undergo SCT2 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 (7 listed)
- Gender is not limited, patients between 14 to 70 years old (including critical value);
- Malignant hematological diseases (acute/chronic myeloid leukemia, acute lymphoblastic leukemia, myelodysplastic syndrome, malignant lymphoma, etc.) diagnosed by bone marrow aspiration or biopsy according to the WHO diagnostic criteria, after the first hematopoietic stem cell transplantation, due to various reasons (including but not limited to disease relapse or graft failure) have indications for a second hematopoietic stem cell transplantation;
- The indexes of cardiac function, liver and kidney function were within the following limits: (1) Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3× Upper limit of normal (ULN); (2)Total bilirubin ≤ 3×ULN; (3) Serum creatinine ≤ 2×ULN or creatinine clearance ≥ 40mL/min; (4) Left ventricular ejection fraction (LVEF) as measured by echocardiography or multi-gated acquisition (MUGA) scan is within the normal range (> 50%);
- Having a suitable allogeneic hematopoietic stem cell donor;
- Expected survival ≥3 months;
Key exclusion criteria (8 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;
- Received Class II or higher surgery within 4 weeks prior to enrollment;
- Patients with active viral infections;
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 |
|---|---|---|
| Second Stem Cell Transplantation (SCT2)1. Conditioning Regimen Day -7: Semustine (Me-CCNU) 250mg/m², orally; Day -6: Total Body Irradiation (TBI) 4Gy; 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 (Second 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 malignant hematological diseases who undergo SCT2 |
Primary outcome
The cumulative incidence of neutrophil engraftment and platelet engraftment
Time frame: on day 28±7 following SCT2
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 SCT2
Recovery of hemopoietic function after treatment
The cumulative incidence of transplant-related mortality (TRM)
Time frame: within 100 days following SCT2
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 SCT2
The cumulative incidence of relapse
Time frame: within 1 year following SCT2
Overall survival rate
Time frame: within 1 year following SCT2
The cumulative incidence of adverse events
Time frame: within 1 year following SCT2
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 13 Mar 2025.
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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. principal investigator
The First Affiliated Hospital of Soochow University
Xiaojin Wu, Prof. principal investigator
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 Second Stem Cell Transplantation (SCT2) in people with Malignant Hematological Diseases, 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 7 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: looking for participants now, as of the sponsor's last update on 13 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 SCT2). 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 13 Mar 2025.