Myelodysplastic SyndromePhase 2NCT06984536
Reduced ATG Plus Mini PTCy for GVHD Prophylaxis in Haplo-SCT
A Phase 2 study testing Reduced ATG plus mini PTCy in people with Myelodysplastic Syndrome and Acute Leukemia, run by Peking University People's Hospital at 1 site in China.
Trial summary
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is regarded as a curative therapy for a variety of hematological malignancies and nonmalignant diseases. However, donor limitations have restricted the widespread use of allo-HSCT for a long period. Read more about this study
- Who is it for?
- Ages 12 to 65 years
- Conditions the study is for
- Myelodysplastic Syndrome, Acute Leukemia
- What is being tested?
- Reduced ATG plus mini PTCy
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteChina
- Study dates
- 21 May 2025to 31 Jul 2026
- 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 12 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients with AL - CR and/or myelodysplastic syndromes undergoing allogeneic hematopoietic stem cell transplantation for the first time;
- No gender limit, aged 12 - 65 years;
- Planned haploidentical donor transplantation, excluding transplantation from maternal and collateral donors;
- Eastern Cooperative Oncology Group (ECOG) performance status score≤3 points;
- Baseline organ function tests meet the following criteria:
- Left ventricular ejection fraction (LVEF) > 55%; (2) Serum creatinine ≤ 1.5 × upper limit of normal (ULN).
Who cannot join
- Patients with severe dysfunction of brain, heart, kidney or liver;
- Those in refractory malignant status;
- Patients with other malignancies requiring treatment;
- Presence of uncontrolled severe active infection clinically;
- Expected survival period of less than 3 months;
- History of severe allergic reactions;
- Pregnant or breastfeeding women; (8)Presence of any condition deemed by the investigator as unsuitable for 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, 1 recruiting
- Recruiting
Peking University People'S Hospital
Beijing
yu wang, 861088326000, ywyw3172@sina.com
About this study
Official title: Reduced-dose Anti-thymocyte Globulin Plus Mini-dose Post-transplant Cyclophosphamide for GVHD Prevention in Haploidentical Donor HCT for Hematologic Malignancy
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is regarded as a curative therapy for a variety of hematological malignancies and nonmalignant diseases. However, donor limitations have restricted the widespread use of allo-HSCT for a long period. The development and success of haploidentical allografts worldwide makes "everyone has a donor" a reality. In the past two decades, researchers have established several haploidentical HSCT (haplo-HSCT) protocols based on different approaches to induce immune tolerance. The representative approaches for haplo-HSCT without in vitro. T cell depletion include granulocyte colony-stimulating factor (G-CSF) plus Anti-human Thymocyte Immunoglobulin (ATG) based (Beijing Protocol) and post-transplantation cyclophosphamide based (PT-Cy, Baltimore Protocol) protocols. Both of two protocols have common problems that need to be solved, including infection transplantation related mortality and disease relapse. The main aim of this study is to explore whether the combined protocol can improve the efficacy of haploidentical transplantation further.
- Conditions
- Myelodysplastic Syndrome, Acute Leukemia
- Being tested
- Reduced ATG plus mini PTCy
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Reduced ATG plus mini PTCyDrug
The conditioning protocol comprises cytarabine (Ara-C) (4 g/m2/day, days -9), busulfan (Bu) (3.2 mg/kg/day, days -8 to -6), cyclophosphamide (Cy) (1.8 g/m2/kg, days -5 and -4), simustine (250 mg/m2, day -3) and r-ATG (total 7.5mg/kg ,from days -5 to -2). Mini PTCy 14.5mg/kg/day will be given on day +3 and +4.
| Study group | Role | What they receive |
|---|---|---|
| Reduced ATG puls mini PTCy | Experimental | Patients recieved ATG 7.5mg/kg plus PTCy 14.5mg/kg on day +3 and +4 for GVHD prophylaxis in haplo-SCT Receives: Drug: Reduced ATG plus mini PTCy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Non-relapse mortality
None-relapse mortality within 100 days post transplantation
Measured: 100 days
Secondary outcomes (4)
Regimen related toxicity
Regimen related toxicity will be evaluated by Bearman Criteria from the begining of conditioning to 1 month post HSCT. The organ functions that need to be assessed include the heart, liver, kidneys, cystitis, oral mucositis, and the gastrointestinal tract.
Measured: 30 days post transplantation
Engraftment
Myeloid and platelet engraftment
Measured: Within 30 days post transplant. Myeloid engraftment was defined as the first of three consecutive days with an ANC 0.5×109 /L, and platelet engraftment was defined as the day the platelet count met or exceeded 20×10^9 /L without transfusion for a week.
Disease relapse
The incidence of disease relapse
Measured: 1 year post transplantation
Disease free survival
The time from the start of transplantation until a patient experiences leukemia relapse or death from any cause, whichever occurs first.
Measured: 1 year post transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2025
- Study start21 May 2025
- Primary completion31 Jul 2025 (estimated)
- Study completion31 Jul 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromeStudies 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
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 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
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06984536
- Sponsor's protocol ID
- Lower ATG plus miniPTCy
- Status last verified
- May 2025
- First submitted
- 3 May 2025
- Last updated
- 25 Jul 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 25 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Reduced-dose Anti-thymocyte Globulin Plus Mini-dose Post-transplant Cyclophosphamide for GVHD Prevention in Haploidentical Donor HCT for Hematologic Malignancy
- ClinicalTrials.gov ID
- NCT06984536
- Acronym / sponsor ID
- Lower ATG plus miniPTCy
- Conditions
- Myelodysplastic Syndrome, Acute Leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 21 May 2025 (actual)
- Primary completion
- 31 Jul 2025 (estimated)
- Study completion
- 31 Jul 2026 (estimated)
- First posted
- 22 May 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 stem cell transplantation (allo-HSCT) is regarded as a curative therapy for a variety of hematological malignancies and nonmalignant diseases. However, donor limitations have restricted the widespread use of allo-HSCT for a long period. The development and success of haploidentical allografts worldwide makes "everyone has a donor" a reality. In the past two decades, researchers have established several haploidentical HSCT (haplo-HSCT) protocols based on different approaches to induce immune tolerance. The representative approaches for haplo-HSCT without in vitro. T cell depletion include granulocyte colony-stimulating factor (G-CSF) plus Anti-human Thymocyte Immunoglobulin (ATG) based (Beijing Protocol) and post-transplantation cyclophosphamide based (PT-Cy, Baltimore Protocol) protocols. Both of two protocols have common problems that need to be solved, including infection transplantation related mortality and disease relapse. The main aim of this study is to explore whether the combined protocol can improve the efficacy of haploidentical transplantation further.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Reduced ATG puls mini PTCy | Experimental | Drug: Reduced ATG plus mini PTCyPatients recieved ATG 7.5mg/kg plus PTCy 14.5mg/kg on day +3 and +4 for GVHD prophylaxis in haplo-SCT |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Patients with AL - CR and/or myelodysplastic syndromes undergoing allogeneic hematopoietic stem cell transplantation for the first time;
- No gender limit, aged 12 - 65 years;
- Planned haploidentical donor transplantation, excluding transplantation from maternal and collateral donors;
- Eastern Cooperative Oncology Group (ECOG) performance status score≤3 points;
- Baseline organ function tests meet the following criteria:
Key exclusion criteria (7 listed)
- Patients with severe dysfunction of brain, heart, kidney or liver;
- Those in refractory malignant status;
- Patients with other malignancies requiring treatment;
- Presence of uncontrolled severe active infection clinically;
- Expected survival period of less than 3 months;
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 |
|---|---|---|
| Reduced ATG plus mini PTCyThe conditioning protocol comprises cytarabine (Ara-C) (4 g/m2/day, days -9), busulfan (Bu) (3.2 mg/kg/day, days -8 to -6), cyclophosphamide (Cy) (1.8 g/m2/kg, days -5 and -4), simustine (250 mg/m2, day -3) and r-ATG (total 7.5mg/kg ,from days -5 to -2). Mini PTCy 14.5mg/kg/day will be given on day +3 and +4. | Drug | Reduced ATG puls mini PTCy |
Primary outcome
Non-relapse mortality
Time frame: 100 days
None-relapse mortality within 100 days post transplantation
4 secondary outcomes
Regimen related toxicity
Time frame: 30 days post transplantation
Engraftment
Time frame: Within 30 days post transplant. Myeloid engraftment was defined as the first of three consecutive days with an ANC 0.5×109 /L, and platelet engraftment was defined as the day the platelet count met or exceeded 20×10^9 /L without transfusion for a week.
Disease relapse
Time frame: 1 year post transplantation
Disease free survival
Time frame: 1 year 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 25 Jul 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.
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 Reduced ATG plus mini PTCy in people with Myelodysplastic Syndrome and Acute Leukemia, 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 6 inclusion and 7 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 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 Yu Wang, M.D. (86-010-8832-6000, ywyw3172@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Non-relapse mortality (time frame: 100 days). 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.