Sirolimus+Abatacept+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to Calcineurin Inhibitors

Phase 1

Not yet recruiting

• Conditions: Myelodysplastic Syndromes; Acute Leukemia; Severe Aplastic Anemia
• Interventions: Drug: Sirolimus; Drug: Abatacept; Drug: MMF; Drug: ATG

• Registration Number: NCT06279494
• Lead Sponsor: Peking University People's Hospital

Brief 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. 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 Abatacept and Mycophenolate Mofetil for the prophylaxis of GVHD in patients with haplo-HSCT who are intolerant to calcineurin inhibitors.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-02-09
• Study First Submitted QC: 2024-02-25
• Study First Posted: 2024-02-28
• Status Verified: 2024-03
• Study Start: 2024-03
• Last Update Submitted: 2024-03-19
• Last Update Posted: 2024-03-21
• Primary Completion: 2024-06
• Study Completion: 2025-03

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 6

Inclusion Criteria

Not provided

Exclusion Criteria

1. Allergy or intolerance to study drugs
2. Active infection
3. Active GVHD
4. Transplantation-associated thrombotic microangiopathy
5. Key organ dysfunction: liver injury (total bilirubin more than 2 upper limit of normal) or heart injury (symptomatic heart failure or ejection fraction<50%)
6. Eastern Cooperative Oncology Group (ECOG) score >2
7. Expected survival time <30 days
8. Patients could not cooperate

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Sirolimus+Abatacept+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG)	Sirolimus	Patients receiving haplo-HSCT who are intolerant to calcineurin inhibitors would receive Sirolimus+Abatacept+MMF+ATG (SAMA) for prophylaxis of aGVHD
Sirolimus+Abatacept+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG)	MMF	Patients receiving haplo-HSCT who are intolerant to calcineurin inhibitors would receive Sirolimus+Abatacept+MMF+ATG (SAMA) for prophylaxis of aGVHD
Sirolimus+Abatacept+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG)	Abatacept	Patients receiving haplo-HSCT who are intolerant to calcineurin inhibitors would receive Sirolimus+Abatacept+MMF+ATG (SAMA) for prophylaxis of aGVHD
Sirolimus+Abatacept+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG)	ATG	Patients receiving haplo-HSCT who are intolerant to calcineurin inhibitors would receive Sirolimus+Abatacept+MMF+ATG (SAMA) for prophylaxis of aGVHD

Primary Outcome Measures

Name	Time	Method
Incidence of Grade 2-4 aGVHD within 100 days post transplantation	Participants will be followed for an expected average of 100 days post transplantation

Secondary Outcome Measures

Name	Time	Method
Incidence of chronic GVHD (cGVHD) within 1 year post transplantation	Participants will be followed for an expected average of 1 year
Incidence of thrombotic microangiopathy within 1 year post transplantation	Participants will be followed for an expected average of 1 year