MDSPhase 2NCT07085156
Reduced Intensity Conditioning for MDS
A Phase 2 study testing Fludarabine; melphalan; busulfan in people with MDS, run by Ruijin Hospital at 1 site in China.
Trial summary
Allogeneic hematopoietic cell transplantation (allo-HCT) is the only potential cure for Myelodysplastic syndrome (MDS). Currently, the conditioning regimen for MDS allogeneic transplantation still follows AML, and there are fewer explorations in this field. Read more about this study
- Who is it for?
- Ages 14 to 70 years
- Conditions the study is for
- MDS
- What is being tested?
- Fludarabine; melphalan; busulfan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 45 participants
- Where
- 1 siteChina
- Study dates
- 1 Aug 2025to 1 May 2028
- 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 14 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age 14-70 (including boundaries) and gender;
- Diagnosis of MDS is confirmed on the basis of bone marrow cytomorphology, immunophenotyping and chromosomal and molecular biology tests;
- IPSS-R intermediate-risk or higher-risk or IPSS-M intermediate-risk or higher-risk MDS; transfusion-dependent MDS;
- ECOG score≤ 3 points;
- Have appropriate organ function, and laboratory results within 7 days prior to the start of trial treatment need to meet the following criteria:
- Aspartate aminotransferase (AST) ≤ 3 times ULN (upper limit of normal, ULN);
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- Alanine aminotransferase (ALT) ≤ 3x ULN;
- Total serum bilirubin ≤ 1.5 times the upper limit of normal ULN unless the patient has documented Gilbert syndrome; patients with Gilbert-Meulengracht syndrome with bilirubin ≤ 3.0 times the upper limit of normal and direct bilirubin ≤ 1.5 times the upper limit of normal may be included;
- Serum creatinine ≤ 1.5 times ULN or creatinine clearance ≥ 60 mL/min;
- Coagulation function: International Normalised Ratio (INR) ≤ 1.5 x ULN, Activated Partial Thromboplastin Time (APTT) ≤ 1.5 x ULN;
- Left ventricular ejection fraction (LVEF) ≥50%;
- Voluntarily signing the informed consent, understanding and complying with the requirements of the study, good compliance, and cooperating with the follow-up visits.
Who cannot join
- Allergies or contraindications to any of the drugs in the protocol;
- Currently have clinically significant active cardiovascular disease such as uncontrolled arrhythmias, uncontrolled hypertension, congestive heart failure, any grade 3 or 4 heart disease as determined by the New York Heart Association (NYHA) functional class, or a history of myocardial infarction within the 6 months prior to screening;
- Serious medical conditions that may limit the patient's participation in this trial (e.g., progressive infection, uncontrolled diabetes);
- Active autoimmune diseases such as SLE, rheumatoid arthritis, etc;
- Patients with neurological or psychiatric disorders;
- The patient is pregnant or breastfeeding;
- Those who are unable to understand or comply with the study protocol or are unable to sign the informed consent form.
- Other conditions that, in the opinion of the investigator, make the patient otherwise unsuitable for participation in this study;
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 Jiaotong University School of Medicine
Shanghai, Shanghai Municipality
About this study
Official title: The Efficacy and Safety of Reduced-Intensity Conditioning With Fludarabine- Melphalan-Busulfan for Allogeneic Hematopoietic Stem Cell Transplantation in the Treatment of Myelodysplastic Syndromes
Allogeneic hematopoietic cell transplantation (allo-HCT) is the only potential cure for Myelodysplastic syndrome (MDS). Currently, the conditioning regimen for MDS allogeneic transplantation still follows AML, and there are fewer explorations in this field. In a large-scale retrospective analysis with 532 MDS undergoing allo-HCT, reduced intensity conditioning is resulted in improved overall survival (OS), reduced non-relapse mortality (NRM) , while sparing relapse.
Therefore, the investigators conduct a prospective, single-arm, multicentre study to evaluate the efficacy and safety of Fludarabine-Melphalan-Busulfan reduced-intensity conditioning in MDS patients.
Read the detailed description
This study is a prospective, multicenterstudy designed to evaluate the efficacy and safety of evaluate the efficacy and safety of Fludarabine-Melphalan-Busulfan intermediate-intensity transplantation conditioning regimen for the treatment of MDS. . Patients with MDS who are eligible for enrolment were pretreated with a Fludarabine-Melphalan-Busulfan regimen (Fludarabine 30mg/m2d-6 to -2; melphalan 70mg/m2d-6; busulfan 3.2mg/kg d-4 to -3). The primary endpoint is 2-year relapse-free survival (RFS) after transplantation. Secondary endpoints includes 2-year post-transplant survival (OS) and cumulative recurrence rate at 2 years post-transplant.
- Conditions
- MDS
- Being tested
- Fludarabine; melphalan; busulfan
- Keywords
- MDS, Reduced intensity conditioning
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fludarabine; melphalan; busulfanDrug
The conditioning regimen includes fludarabine, busulfan, and melphalan, with a transplant conditioning intensity of 3.0. The RIC is used in the allo-HCT in the MDS patients.
| Study group | Role | What they receive |
|---|---|---|
| FBM arm | Experimental | Adult MDS patients eligible for allo-HCT will receive Fludarabine 30mg/m^2 d-6 to -2; melphalan 70mg/m^2 d-6; busulfan 3.2mg/kg d-4 to -3 as conditioning regimen. Receives: Drug: Fludarabine; melphalan; busulfan |
What is being measured
The primary outcome is the main question the study is designed to answer.
2-year relapse-free survival after transplantation
relapse
Measured: through study completion, an average of 2 year
Secondary outcomes (2)
2-year post-transplant survival
survival
Measured: through study completion, an average of 2 year
Cumulative recurrence rate at 2 years post-transplant
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 posted25 Jul 2025
- Study start1 Aug 2025 (estimated)
- Primary completion1 May 2028 (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 MDSStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 45 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ruijin Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Wuhan TongJi Hospital, Wuhan Children's Hospital, Zhejiang University
- Responsible party
- Principal investigator, Hu Xiaoxia, Ruijin Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07085156
- Sponsor's protocol ID
- RJ-BMT-012
- Status last verified
- Jul 2025
- First submitted
- 6 Jul 2025
- Last updated
- 25 Jul 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Efficacy and Safety of Reduced-Intensity Conditioning With Fludarabine- Melphalan-Busulfan for Allogeneic Hematopoietic Stem Cell Transplantation in the Treatment of Myelodysplastic Syndromes
- ClinicalTrials.gov ID
- NCT07085156
- Acronym / sponsor ID
- RJ-BMT-012
- Conditions
- MDS
- Keywords
- MDS, Reduced intensity conditioning
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 45 participants (estimated)
- Lead sponsor
- Ruijin HospitalUniversity, hospital or other
- Collaborators
- Wuhan TongJi Hospital, Wuhan Children's Hospital, Zhejiang University
- Study start
- 1 Aug 2025 (estimated)
- Primary completion
- 1 May 2028 (estimated)
- Study completion
- 1 May 2028 (estimated)
- First posted
- 25 Jul 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.
Allogeneic hematopoietic cell transplantation (allo-HCT) is the only potential cure for Myelodysplastic syndrome (MDS). Currently, the conditioning regimen for MDS allogeneic transplantation still follows AML, and there are fewer explorations in this field. In a large-scale retrospective analysis with 532 MDS undergoing allo-HCT, reduced intensity conditioning is resulted in improved overall survival (OS), reduced non-relapse mortality (NRM) , while sparing relapse.
Therefore, the investigators conduct a prospective, single-arm, multicentre study to evaluate the efficacy and safety of Fludarabine-Melphalan-Busulfan reduced-intensity conditioning in MDS patients.
Detailed description
This study is a prospective, multicenterstudy designed to evaluate the efficacy and safety of evaluate the efficacy and safety of Fludarabine-Melphalan-Busulfan intermediate-intensity transplantation conditioning regimen for the treatment of MDS. . Patients with MDS who are eligible for enrolment were pretreated with a Fludarabine-Melphalan-Busulfan regimen (Fludarabine 30mg/m2d-6 to -2; melphalan 70mg/m2d-6; busulfan 3.2mg/kg d-4 to -3). The primary endpoint is 2-year relapse-free survival (RFS) after transplantation. Secondary endpoints includes 2-year post-transplant survival (OS) and cumulative recurrence rate at 2 years post-transplant.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FBM arm | Experimental | Drug: Fludarabine; melphalan; busulfanAdult MDS patients eligible for allo-HCT will receive Fludarabine 30mg/m^2 d-6 to -2; melphalan 70mg/m^2 d-6; busulfan 3.2mg/kg d-4 to -3 as 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 (12 listed)
- Age 14-70 (including boundaries) and gender;
- Diagnosis of MDS is confirmed on the basis of bone marrow cytomorphology, immunophenotyping and chromosomal and molecular biology tests;
- IPSS-R intermediate-risk or higher-risk or IPSS-M intermediate-risk or higher-risk MDS; transfusion-dependent MDS;
- ECOG score≤ 3 points;
- Have appropriate organ function, and laboratory results within 7 days prior to the start of trial treatment need to meet the following criteria:
Key exclusion criteria (8 listed)
- Allergies or contraindications to any of the drugs in the protocol;
- Currently have clinically significant active cardiovascular disease such as uncontrolled arrhythmias, uncontrolled hypertension, congestive heart failure, any grade 3 or 4 heart disease as determined by the New York Heart Association (NYHA) functional class, or a history of myocardial infarction within the 6 months prior to screening;
- Serious medical conditions that may limit the patient's participation in this trial (e.g., progressive infection, uncontrolled diabetes);
- Active autoimmune diseases such as SLE, rheumatoid arthritis, etc;
- Patients with neurological or psychiatric disorders;
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 |
|---|---|---|
| Fludarabine; melphalan; busulfanThe conditioning regimen includes fludarabine, busulfan, and melphalan, with a transplant conditioning intensity of 3.0. The RIC is used in the allo-HCT in the MDS patients. | Drug | FBM arm |
Primary outcome
2-year relapse-free survival after transplantation
Time frame: through study completion, an average of 2 year
relapse
2 secondary outcomes
2-year post-transplant survival
Time frame: through study completion, an average of 2 year
Cumulative recurrence rate at 2 years post-transplant
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 25 Jul 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.
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 Fludarabine; melphalan; busulfan in people with MDS, 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 12 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 25 Jul 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.
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 Xiaoxia HU, Doctor (02164370045, hu_xiaoxia@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
2-year relapse-free survival after transplantation (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 25 Jul 2025.