Acute LeukemiaPhase 1/2NCT07796620
Sirolimus+Ruxolitinib+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to CNI
A Phase 1/2 study testing Sirolimus, Ruxolitinib and MMF in people with Acute Leukemia and Myelodysplastic Syndromes, run by Peking University People's Hospital.
Trial summary
Graft-versus-host disease (GVHD) is an important complication after transplantation, with an incidence of 40-60%, which can increase non-relapse mortality if poorly controlled. At present, the standard prophylaxis for GVHD is cyclosporine combined with methotrexate. Read more about this study
- Who is it for?
- Ages 14 years and older
- Conditions the study is for
- Acute Leukemia, Myelodysplastic Syndromes, Severe Aplastic Anemia (SAA)
- What is being tested?
- Sirolimus, Ruxolitinib and MMF
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- No sites listed yet
- Study dates
- 1 Oct 2026to 30 Sep 2028
- Phase
- Phase 1/2
- 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 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Primary disease: hematological malignancies (including acute leukemia, myelodysplastic syndromes), nonmalignant disorders (including severe aplastic anaemia)
- Renal injury or inability to tolerate the side effects of CNI: such as CNI renal toxicity (creatinine levels above the upper limit of normal), uncontrolled hypertension, and neurotoxicity rrom the time of hematopoietic stem cell infusion until +90 days after transplantation
- Receiving haplo-HSCT for the first time
Who cannot join
- Allergy or intolerance to study drugs
- Active infection
- Active GVHD
- Transplantation-associated thrombotic microangiopathy
- Key organ dysfunction: liver injury (total bilirubin more than 2 upper limit of normal) or heart injury (symptomatic heart failure or ejection fraction<50%)
- Eastern Cooperative Oncology Group (ECOG) score >2
- Expected survival time <30 days
- Patients could not cooperate
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Sirolimus+Ruxolitinib+Mycophenolate Mofetil Regimen for Prophylaxis of Acute Graft-versus-host Disease (aGvHD) in Patients Receiving Haploidentical Hematopoietic Stem Cell Transplantation (Haplo-HSCT) Who Are Intolerant to Calcineurin Inhibitor
Graft-versus-host disease (GVHD) is an important complication after transplantation, with an incidence of 40-60%, which can increase non-relapse mortality if poorly controlled. At present, the standard prophylaxis for GVHD is cyclosporine combined with methotrexate. However, calcineurin inhibitors (CNI) can cause some vital side effects, which are not tolerated by some patients. Therefore, this study aims to explore the safety and efficacy of Sirolimus in combination with Ruxolitinib and Mycophenolate Mofetil for the prophylaxis of GVHD in patients with haplo-HSCT who are intolerant to calcineurin inhibitors.
- Being tested
- Sirolimus, Ruxolitinib, MMF, ATG
- Keywords
- Ruxolitinib, Sirolimus, Mycophenolate Mofetil, Graft-versus-host-disease, Haploidentical, Calcineurin Inhibitor
- Medical subject headings
- Myelodysplastic Syndromes, Anemia, Aplastic, Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SirolimusDrug
Sirolimus 2mg once daily, maintaining the concentration at 5-10 ng/ml. Gradually reduce the dosage after +100 days. If the patient has stable engraftment and no GVHD, discontinue on +180 days.
RuxolitinibDrug
Ruxolitinib is administered at a dose of 5mg twice daily from the start of the study until +90 days. The dose is reduced to 5mg once daily on +90 days, and discontinued on +120 days.
MMFDrug
MMF 0.5g, taken twice daily, is discontinued after 60 days. If it is resumed after 60 days, it should be taken for 2 weeks.
ATGDrug
2.5 mg/kg, from -5d to -2d
| Study group | Role | What they receive |
|---|---|---|
| Sirolimus+Ruxolitinib+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG) | Experimental | Patients receiving haplo-HSCT who are intolerant to calcineurin inhibitors would receive Sirolimus+Ruxolitinib+MMF+ATG (SRMA) for prophylaxis of aGVHD Receives: Drug: Sirolimus; Drug: Ruxolitinib; Drug: MMF; Drug: ATG |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Grade 2-4 aGVHD within 100 days post transplantation
Measured: Participants will be followed for an expected average of 100 days post transplantation
Secondary outcomes (6)
Incidence of chronic GVHD (cGVHD) within 1 year post transplantation
Measured: Participants will be followed for an expected average of 1 year
Incidence of thrombotic microangiopathy within 1 year post transplantation
Measured: Participants will be followed for an expected average of 1 year
Cumulative incidence of relapse
Measured: Participants will be followed for an expected average of 1 year
Transplant-related mortality
Measured: Participants will be followed for an expected average of 1 year
Overall survival
Measured: Participants will be followed for an expected average of 1 year
Incidence of cytomegalovirus (CMV) and Epstein-Barr virus (EBV)
Measured: Participants will be followed for an expected average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Sep 2026
- Study start1 Oct 2026 (estimated)
- Primary completion30 Sep 2027 (estimated)
- Study completion30 Sep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute LeukemiaStudies 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 1/2
- 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
- NCT07796620
- Sponsor's protocol ID
- Sirolimus for GVHD prevention
- Status last verified
- Sep 2026
- First submitted
- 27 Aug 2026
- Last updated
- 9 Sep 2026
- 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 9 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Sirolimus+Ruxolitinib+Mycophenolate Mofetil Regimen for Prophylaxis of Acute Graft-versus-host Disease (aGvHD) in Patients Receiving Haploidentical Hematopoietic Stem Cell Transplantation (Haplo-HSCT) Who Are Intolerant to Calcineurin Inhibitor
- ClinicalTrials.gov ID
- NCT07796620
- Acronym / sponsor ID
- Sirolimus for GVHD prevention
- Keywords
- Ruxolitinib, Sirolimus, Mycophenolate Mofetil, Graft-versus-host-disease, Haploidentical, Calcineurin Inhibitor
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 1 Oct 2026 (estimated)
- Primary completion
- 30 Sep 2027 (estimated)
- Study completion
- 30 Sep 2028 (estimated)
- First posted
- 1 Sep 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.
Graft-versus-host disease (GVHD) is an important complication after transplantation, with an incidence of 40-60%, which can increase non-relapse mortality if poorly controlled. At present, the standard prophylaxis for GVHD is cyclosporine combined with methotrexate. However, calcineurin inhibitors (CNI) can cause some vital side effects, which are not tolerated by some patients. Therefore, this study aims to explore the safety and efficacy of Sirolimus in combination with Ruxolitinib and Mycophenolate Mofetil for the prophylaxis of GVHD in patients with haplo-HSCT who are intolerant to calcineurin inhibitors.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sirolimus+Ruxolitinib+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG) | Experimental | Drug: Sirolimus, Drug: Ruxolitinib, Drug: MMF, Drug: ATGPatients receiving haplo-HSCT who are intolerant to calcineurin inhibitors would receive Sirolimus+Ruxolitinib+MMF+ATG (SRMA) for prophylaxis of aGVHD |
Who may qualify
- Ages
- 14 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Primary disease: hematological malignancies (including acute leukemia, myelodysplastic syndromes), nonmalignant disorders (including severe aplastic anaemia)
- Renal injury or inability to tolerate the side effects of CNI: such as CNI renal toxicity (creatinine levels above the upper limit of normal), uncontrolled hypertension, and neurotoxicity rrom the time of hematopoietic stem cell infusion until +90 days after transplantation
- Receiving haplo-HSCT for the first time
Key exclusion criteria (8 listed)
- Allergy or intolerance to study drugs
- Active infection
- Active GVHD
- Transplantation-associated thrombotic microangiopathy
- Key organ dysfunction: liver injury (total bilirubin more than 2 upper limit of normal) or heart injury (symptomatic heart failure or ejection fraction<50%)
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 |
|---|---|---|
| SirolimusSirolimus 2mg once daily, maintaining the concentration at 5-10 ng/ml. Gradually reduce the dosage after +100 days. If the patient has stable engraftment and no GVHD, discontinue on +180 days. | Drug | Sirolimus+Ruxolitinib+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG) |
| RuxolitinibRuxolitinib is administered at a dose of 5mg twice daily from the start of the study until +90 days. The dose is reduced to 5mg once daily on +90 days, and discontinued on +120 days. | Drug | Sirolimus+Ruxolitinib+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG) |
| MMFMMF 0.5g, taken twice daily, is discontinued after 60 days. If it is resumed after 60 days, it should be taken for 2 weeks. | Drug | Sirolimus+Ruxolitinib+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG) |
| ATG2.5 mg/kg, from -5d to -2d | Drug | Sirolimus+Ruxolitinib+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG) |
Primary outcome
Incidence of Grade 2-4 aGVHD within 100 days post transplantation
Time frame: Participants will be followed for an expected average of 100 days post transplantation
6 secondary outcomes
Incidence of chronic GVHD (cGVHD) within 1 year post transplantation
Time frame: Participants will be followed for an expected average of 1 year
Incidence of thrombotic microangiopathy within 1 year post transplantation
Time frame: Participants will be followed for an expected average of 1 year
Cumulative incidence of relapse
Time frame: Participants will be followed for an expected average of 1 year
Transplant-related mortality
Time frame: Participants will be followed for an expected average of 1 year
Overall survival
Time frame: Participants will be followed for an expected average of 1 year
Incidence of cytomegalovirus (CMV) and Epstein-Barr virus (EBV)
Time frame: Participants will be followed for an expected average of 1 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 9 Sep 2026.
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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.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Sirolimus, Ruxolitinib and MMF in people with Acute Leukemia and Myelodysplastic Syndromes, run by Peking University People's Hospital.
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 3 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 9 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 Yuqian Sun (+86-10-88324577, sunyuqian83@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Grade 2-4 aGVHD within 100 days post transplantation (time frame: Participants will be followed for an expected average of 100 days post transplantation). 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 9 Sep 2026; AzTrials last refreshed its copy on 8 Nov 2026.