Myelodysplastic SyndromesPhase 2NCT07677306
Age-Stratified Conditioning Regimen Efficacy for MDS Haplo-HSCT
A Phase 2 study testing RTC conditioning regimen and Modified Bu/Cy conditioning regimen in people with Myelodysplastic Syndromes, run by Peking University People's Hospital at 1 site in China.
Trial summary
This study plan aims to enroll adult patients diagnosed with myelodysplastic syndrome (MDS) who are scheduled to receive T-cell-replete haploidentical hematopoietic stem cell transplantation. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- RTC conditioning regimen and Modified Bu/Cy conditioning regimen
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 120 participants
- Where
- 1 siteChina
- Study dates
- 15 Jul 2026to 30 Jun 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients who had low- and intermediate-risk MDS without ISD nor URD receiving haploidentical hematopoietic stem cell transplantation
Who cannot join
- Patients having ISD or URD; patients having high-risk MDS; patients with active infection; patients with poor compliance; patients with organ failure
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
Peking University People's Hospital, Beijing Lu Daopei Hematology Hospital, Hebei Yanda Lu Daopei Hospital, Nanfang Hospital, Southern Medical University
Beijing
Xiaojun Huang, 8610-8832-6005, huangxiaojun@bjmu.edu.cn
About this study
Official title: Prospective Cohort Study of Age-Stratified Busulfan-Based Conditioning Regimens in Adult Patients With Myelodysplastic Syndrome Receiving Haploidentical Hematopoietic Stem Cell Transplantation
This study plan aims to enroll adult patients diagnosed with myelodysplastic syndrome (MDS) who are scheduled to receive T-cell-replete haploidentical hematopoietic stem cell transplantation. After obtaining written informed consent, participants will receive either reduced-toxicity Bu/Flu/Cy/ATG conditioning regimen (for patients aged ≥55 years) or standard myeloablative modified Bu/Cy+ATG conditioning regimen (for patients aged <55 years) followed by unified post-transplant immunosuppression and supportive care. The objective is to prospectively characterize the 1-year transplant-related mortality and comprehensively evaluate hematopoietic engraftment, graft-versus-host disease, infection, relapse, survival outcomes and conditioning-related organ toxicity among all enrolled patients undergoing haploidentical transplantation.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- RTC conditioning regimen and Modified Bu/Cy conditioning regimen
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RTC conditioning regimen and Modified Bu/Cy conditioning regimenDrug
RTC preconditioning regimen consisted of cytarabine (2g/m2/day, days -10 to -9), busulfan (3.2 mg/kg/day on days -8 to -6), cyclophosphamide (1.0 g/m2/day, days -5 to -4), fludarabine (30 mg/m2/day, days -6 to -2), semustine (250 mg/m2, day-3), and rabbit antithymocyte globulin (thymoglobulin, 2.5 mg/kg/d, days -5 to -2; Sanofi, France). Bu/Cy preconditioning regimen consisted of tandard institutional myeloablative busulfan + cyclophosphamide + rabbit ATG regimen per department routine dosing guidelines.
| Study group | Role | What they receive |
|---|---|---|
| Age-Stratified Conditioning Regimen | Other | MDS patients without identical sibling donor or unrelated donor would receive haplo-HSCT. Participants will receive either reduced-toxicity Bu/Flu/Cy/ATG conditioning regimen (for patients aged ≥55 years) or standard myeloablative modified Bu/Cy+ATG conditioning regimen (for patients aged <55 years) followed by unified post-transplant immunosuppression and supportive care. Receives: Drug: RTC conditioning regimen and Modified Bu/Cy conditioning regimen |
What is being measured
The primary outcome is the main question the study is designed to answer.
transplant-related mortality (TRM)
Death without disease progression or relapse
Measured: Participants will be followed for an expected average of 1 years
Secondary outcomes (7)
Hematopoietic engraftment
Neutrophil engraftment is defined as the first day of sustained absolute neutrophil count (ANC) ≥0.5×10⁹/L for 3 consecutive days; platelet engraftment is defined as the first day of sustained platelet count (PLT) ≥20×10⁹/L without transfusion support for 7 consecutive days. Cumulative incidence of neutrophil engraftment at Day 30 and platelet engraftment at Day 90 will be calculated.
Measured: Day 30 and Day 90 post-transplantation
Cumulative incidence of graft-versus-host disease (GVHD)
Cumulative incidence of Grade II-IV and Grade III-IV acute GVHD at Day 100, as well as chronic GVHD and moderate-severe chronic GVHD within 3 years after transplantation, graded per Seattle criteria.
Measured: Day 100 and 3 years after transplantation
Cumulative incidence of CMV and EBV reactivation
Defined as any detectable CMV or EBV viral load elevation requiring clinical intervention within 1 year after transplantation.
Measured: 1 year after transplantation
Cumulative incidence of hematologic relapse
Defined as bone marrow blast recurrence or extramedullary disease relapse within 1 year after transplantation.
Measured: 1 year after transplantation
Disease-free survival (DFS)
Defined as the time from transplantation until hematologic relapse or death from any cause.
Measured: 1 year after transplantation
Overall survival（OS）
Defined as the time from treatment until death from any cause or the last follow-up.
Measured: 1 year after transplantation
Conditioning-related organ toxicities
Incidence of grade 1-4 cardiac, renal, hepatic, gastrointestinal and oral mucosal toxicities from conditioning initiation to Day +30 post-transplantation, with separate analysis of Grade 3-4 severe organ injury, hepatic veno-occlusive disease (VOD), and conditioning-related death, graded per WHO organ toxicity scale.
Measured: From conditioning start to Day 30 post-transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jun 2026
- Study start15 Jul 2026
- Primary completion31 Dec 2027 (estimated)
- Study completion30 Jun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by Peking University People's 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
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xiao-Jun Huang, Peking University People's Hospital
- Investigators
- Xiao-Jun Huang, Principal investigator, Peking University Institute of Hematology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07677306
- Sponsor's protocol ID
- RTC Haplo-MDS
- Status last verified
- Jun 2026
- First submitted
- 24 Jun 2026
- Last updated
- 16 Sep 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Cohort Study of Age-Stratified Busulfan-Based Conditioning Regimens in Adult Patients With Myelodysplastic Syndrome Receiving Haploidentical Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT07677306
- Acronym / sponsor ID
- RTC Haplo-MDS
- Conditions
- Myelodysplastic Syndromes
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 15 Jul 2026 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 30 Jun 2028 (estimated)
- First posted
- 30 Jun 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 plan aims to enroll adult patients diagnosed with myelodysplastic syndrome (MDS) who are scheduled to receive T-cell-replete haploidentical hematopoietic stem cell transplantation. After obtaining written informed consent, participants will receive either reduced-toxicity Bu/Flu/Cy/ATG conditioning regimen (for patients aged ≥55 years) or standard myeloablative modified Bu/Cy+ATG conditioning regimen (for patients aged <55 years) followed by unified post-transplant immunosuppression and supportive care. The objective is to prospectively characterize the 1-year transplant-related mortality and comprehensively evaluate hematopoietic engraftment, graft-versus-host disease, infection, relapse, survival outcomes and conditioning-related organ toxicity among all enrolled patients undergoing haploidentical transplantation.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Age-Stratified Conditioning Regimen | Other | Drug: RTC conditioning regimen and Modified Bu/Cy conditioning regimenMDS patients without identical sibling donor or unrelated donor would receive haplo-HSCT. Participants will receive either reduced-toxicity Bu/Flu/Cy/ATG conditioning regimen (for patients aged ≥55 years) or standard myeloablative modified Bu/Cy+ATG conditioning regimen (for patients aged <55 years) followed by unified post-transplant immunosuppression and supportive care. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Patients who had low- and intermediate-risk MDS without ISD nor URD receiving haploidentical hematopoietic stem cell transplantation
Key exclusion criteria (1 listed)
- Patients having ISD or URD; patients having high-risk MDS; patients with active infection; patients with poor compliance; patients with organ failure
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 |
|---|---|---|
| RTC conditioning regimen and Modified Bu/Cy conditioning regimenRTC preconditioning regimen consisted of cytarabine (2g/m2/day, days -10 to -9), busulfan (3.2 mg/kg/day on days -8 to -6), cyclophosphamide (1.0 g/m2/day, days -5 to -4), fludarabine (30 mg/m2/day, days -6 to -2), semustine (250 mg/m2, day-3), and rabbit antithymocyte globulin (thymoglobulin, 2.5 mg/kg/d, days -5 to -2; Sanofi, France). Bu/Cy preconditioning regimen consisted of tandard institutional myeloablative busulfan + cyclophosphamide + rabbit ATG regimen per department routine dosing guidelines. | Drug | Age-Stratified Conditioning Regimen |
Primary outcome
transplant-related mortality (TRM)
Time frame: Participants will be followed for an expected average of 1 years
Death without disease progression or relapse
7 secondary outcomes
Hematopoietic engraftment
Time frame: Day 30 and Day 90 post-transplantation
Cumulative incidence of graft-versus-host disease (GVHD)
Time frame: Day 100 and 3 years after transplantation
Cumulative incidence of CMV and EBV reactivation
Time frame: 1 year after transplantation
Cumulative incidence of hematologic relapse
Time frame: 1 year after transplantation
Disease-free survival (DFS)
Time frame: 1 year after transplantation
Overall survival（OS）
Time frame: 1 year after transplantation
Conditioning-related organ toxicities
Time frame: From conditioning start to Day 30 post-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 16 Sep 2026.
- Individual participant dataSharing planned: Undecided
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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.
Xiao-Jun Huang principal investigator
Peking University Institute of Hematology
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 RTC conditioning regimen and Modified Bu/Cy conditioning regimen in people with Myelodysplastic Syndromes, run by Peking University People's 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 1 inclusion and 1 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 16 Sep 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 Yu Wang (8610-8832-6005, ywyw3172@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
transplant-related mortality (TRM) (time frame: Participants will be followed for an expected average of 1 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 16 Sep 2026; AzTrials last refreshed its copy on 6 Nov 2026.