NCT01754454

Safety and Efficacy of UC-MSC in Patients With Acute Severe Graft-versus-host Disease

Open-Label, Single-Center, Self Control, Phase Ⅰ/Ⅱ Clinical Trial to Evaluate the Safety and the Efficacy of Umbilical Cord-derived Mesenchymal Stem Cells in Patients With Acute Graft-versus-host Disease

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: Affiliated Hospital to Academy of Military Medical Sciences · Academic / Other
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

Allogenic haemopoietic stem cell transplantation (allo-HSCT) is the treatment for many malignant and non-malignant hematologic disorders. Acute graft-versus-host disease (aGVHD) is a serious life-threatening complication after allo-HSCT. The outcome for patients with aGVHD is poor and overall survival is low. Acute graft-versus-host disease (GVHD), as the major complication of allogeneic peripheral blood stem cell transplantation(PBSCT), limits the application of this curative therapy. Mesenchymal stem cells (MSCs) are multipotent stem cells, which are able to modulate immune response in vitro and in vivo, and have possibilities of treating diseases caused by immune dysregulation such as aGVHD. MSCs obtained from umbilical cord (UC) have similar immunosuppressive properties as bone marrow-MSCs. In addition, UC-derived MSCs can be used for off-the-shelf use and are obtained without any harm to donors than bone marrow-MSCs. Therefore, the investigators designed this study to evaluate the safety and efficacy of UC-derived MSCs in patients with aGVHD.

Detailed description

The mesenchymal stem cells (MSC) is a class of low immunogenicity from mesoderm, had the self-renewal and differentiation potential as pluripotent stem cells. The umbilical cord is one of the important sources of MSC. Human UC-MSC compared with the bone marrow-derived MSC has the following advantages: (1) the umbilical cord, as a "waste" after giving birth can be obtained without any harm to the donor. (2) easy available, and no ethical issues. (3) its likelihood of contamination is small because of the protection of the placental barrier. (4) human UC-MSC has a stronger amplification capacity, a shorter doubling time, a higher colony forming efficiency and lower immunogenicity. (5) which avoids the activity and differentiation of bone marrow-derived MSC latent with the donor disadvantage of increasing age decreased. human UC-MSC not only for hematopoietic stem cell graft implantation and amplification has a role in promoting and exhibit immunomodulatory effects and characteristics of damaged or targeted sites of chronic inflammation. human UC-MSC in the prevention and treatment of graft-versus-host disease (GVHD) induced organ transplantation immune tolerance and other fields has broad application prospects. After more than 10 years of exploration, the investigators and other practitioners caught studies found that the umbilical cord is more suitable as mesenchymal stem cells seed cells relative to the bone marrow, umbilical cord easily obtained, no ethical issues, better amplification ability in vitro, viral contamination risk is small, and many other advantages. Therefore, over the years, the investigator team for the umbilical cord mesenchymal stem cells in a lot of the basic work has been confirmed that mesenchymal stem cells derived from the umbilical cord had safety and effectiveness in in the treatment of acute graft versus host disease in zoology test, and successfully establish a perfect umbilical cord mesenchymal stem cells in standard operating procedures (including screening umbilical cord, large-scale preparation, quality inspection, preservation, recovery, transportation, infusion, etc.). The investigators will carry out the clinical research based on above preliminary results.

Conditions

• Acute GVH Disease

Interventions

Type	Name	Description
BIOLOGICAL	Human Umbilical Cord Derived MSC	1×10\^6 UC-MSCs per kg intravenous injection the number of infusions: once a week, for four weeks in a row Time interval: estimate the condition of follow-up visit afer 28 days

Timeline

• Start date: 2012-12-01
• Primary completion: 2016-12-01
• Completion: 2016-12-01
• First posted: 2012-12-21
• Last updated: 2016-04-08

Locations

2 sites across 1 country: China