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

Overview

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.

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

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: November 2015

Interventions

  • Biological: Mesenchymal Stromal Cells
    • 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).

Arms, Groups and Cohorts

  • Active Comparator: Mesenchymal stem cells (MSC)
    • Patients with newly diagnosed cGvHD receive primary treatment plus MSC: MSC+prednisone+cyclosporine; MSC+prednisone+tacrolimus; MSC+prednisone+mycophenolate mofetil.
  • Placebo Comparator: Placebo
    • Patients with newly diagnosed cGvHD receive primary treatment: Placebo+prednisone+cyclosporine; Placebo+prednisone+tacrolimus; Placebo+prednisone+mycophenolate mofetil.

Clinical Trial Outcome Measures

Primary Measures

  • Proportion of patients responding to treatment of cGvHD with MSC
    • Time Frame: 90 days

Secondary Measures

  • Overall survival
    • Time Frame: 2 year
  • Progression-free survival
    • Time Frame: 2 year
  • Time without systemic immunosuppression
    • Time Frame: 2 year
  • Cumulative incidents of non-relapse mortality
    • Time Frame: 2 year
  • Adverse events
    • Time Frame: 2 year

Participating in This Clinical Trial

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.

Gender Eligibility: All

Minimum Age: 14 Years

Maximum Age: 65 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Guangdong Provincial People’s Hospital
  • Collaborator
    • Sun Yat-sen University
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Xin Du, Prof., Principal Investigator, Guangdong Provincial People’s Hospital
  • Overall Contact(s)
    • Xin Du, Prof., +86 02083827812-62122, miyadu@hotmail.com

References

Weng J, He C, Lai P, Luo C, Guo R, Wu S, Geng S, Xiangpeng A, Liu X, Du X. Mesenchymal stromal cells treatment attenuates dry eye in patients with chronic graft-versus-host disease. Mol Ther. 2012 Dec;20(12):2347-54. doi: 10.1038/mt.2012.208. Epub 2012 Oct 16.

Weng JY, Du X, Geng SX, Peng YW, Wang Z, Lu ZS, Wu SJ, Luo CW, Guo R, Ling W, Deng CX, Liao PJ, Xiang AP. Mesenchymal stem cell as salvage treatment for refractory chronic GVHD. Bone Marrow Transplant. 2010 Dec;45(12):1732-40. doi: 10.1038/bmt.2010.195. Epub 2010 Sep 6.

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