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NCT Number: NCT07796620

Sirolimus+Ruxolitinib+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to CNI

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.

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Key information

Conditions

Acute Leukemia Anemia Anemia, Aplastic Bone Marrow Diseases Bone Marrow Failure Disorders Graft vs Host Disease Hematologic Diseases Hemic and Lymphatic Diseases Immune System Diseases Myelodysplastic Syndromes Severe Aplastic Anemia (SAA)

Age range

14 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

a. Allergy or intolerance to study drugs b. Active infection c. Active GVHD d. Transplantation-associated thrombotic microangiopathy e. Key organ dysfunction: liver injury (total bilirubin more than 2 upper limit of normal) or heart injury (symptomatic heart failure or ejection fraction<50%) f. Eastern Cooperative Oncology Group (ECOG) score >2 g. Expected survival time <30 days h. Patients could not cooperate

Treatment and study plan

sirolimus

Drug

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.

Ruxolitinib

Drug

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.

MMF

Drug

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.

ATG

Drug

2.5 mg/kg, from -5d to -2d

Primary outcomes

  1. 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

Secondary outcomes

  1. Incidence of chronic GVHD (cGVHD) within 1 year post transplantation

    Time frame: Participants will be followed for an expected average of 1 year

  2. Incidence of thrombotic microangiopathy within 1 year post transplantation

    Time frame: Participants will be followed for an expected average of 1 year

  3. Cumulative incidence of relapse

    Time frame: Participants will be followed for an expected average of 1 year

  4. Transplant-related mortality

    Time frame: Participants will be followed for an expected average of 1 year

  5. Overall survival

    Time frame: Participants will be followed for an expected average of 1 year

  6. Incidence of cytomegalovirus (CMV) and Epstein-Barr virus (EBV)

    Time frame: Participants will be followed for an expected average of 1 year

Study contacts

Contact information is provided by the study sponsor or research team.

Yuqian Sun

CONTACT

[email protected]

+86-10-88324577

Sponsors and collaborators

Lead sponsor

Peking University People's Hospital

Other

Registry information

Official study 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

Important dates

Study start
2026
Primary completion
2027
Study completion
2028
First posted
Sep 1, 2026
Registry last updated
Sep 1, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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