Treatment of Chronic Graft-Versus-Host Disease With Mesenchymal Stromal Cells

Study Overview
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Brief Summary
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Chronic Graft-versus-Host Disease (cGvHD) is a potentially lethal disorder. A variety of second line immunosuppressive agents have been investigated but no optimal treatment has emerged. There is therefore a need for novel treatment strategies. Mesenchymal stromal cells (MSC) exhibit immunomodulatory properties and a recent pilot study suggests a response rate of 70% in steroid- refractory patients. In the present randomized study the efficacy and safety of MSC treatment will be further studied in patients with cGvHD.

Official Title
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Treatment of of Chronic Graft-Versus-Host Disease With Mesenchymal Stromal Cells. A Phase III Randomized Open Label Multi-center Study in Southern China.

Conditions
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Chronic Graft-Versus-Host Disease

Intervention / Treatment
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* Biological: Mesenchymal Stromal Cells


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Newly diagnosed cGvHD Informed consent obtained from patient and donor. Any patient who has undergone allogeneic stem cell transplantation with c GvHD. Have not received additional agent for cGVHD within 3 months. Expected life is more than 90 days. Adequate pulmonary function with no evidence of chronic obstructive or severe restrictive pulmonary disease. Adequate cardiac function with no evidence of uncontrolled high blood pressure,congestive heart failure, angina pectoris, acute myocardial infarction within 6 months prior to the process. Exclusion Criteria: Invasive fungal disease. Active cytomegalovirus (CMV)/Epstein-Barr virus(EBV)/varicella disease). Patient is with a history of hypersensitivity to bovine products. Relapsed malignancy.

Ages Eligible for Study
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Minimum Age: 14 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Mesenchymal stem cells (MSC)<br>Patients with newly diagnosed cGvHD receive primary treatment plus MSC: MSC+prednisone+cyclosporine; MSC+prednisone+tacrolimus; MSC+prednisone+mycophenolate mofetil. | Biological: Mesenchymal Stromal Cells<br>* Mesenchymal stem cell(MSC). Patients with newly diagnosed cGvHD: prednisone 1mg/kg + cyclosporine or tacrolimus and MSC 2×1,000,000 MSC/kg, IV twice a week for the first two weeks and weekly for the following two weeks(6 doses totally).<br>* Other names: MSC;|
| Placebo Comparator: Placebo<br>Patients with newly diagnosed cGvHD receive primary treatment: Placebo+prednisone+cyclosporine; Placebo+prednisone+tacrolimus; Placebo+prednisone+mycophenolate mofetil. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of patients responding to treatment of cGvHD with MSC |  | 90 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall survival |  | 2 year | 
| Progression-free survival |  | 2 year | 
| Time without systemic immunosuppression |  | 2 year | 
| Cumulative incidents of non-relapse mortality |  | 2 year | 
| Adverse events |  | 2 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Chronic Graft-Versus-Host Disease, Mesenchymal Stromal Cells