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Mesenchymal Stem Cells for Treatment of Refractory Chronic Graft-versus-host Disease

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ClinicalTrials.gov Identifier: NCT01765660
Recruitment Status : Unknown
Verified January 2013 by Nanfang Hospital of Southern Medical University.
Recruitment status was:  Recruiting
First Posted : January 10, 2013
Last Update Posted : January 10, 2013
Information provided by (Responsible Party):

Study Description
Brief Summary:
The purpose of this study is to evaluate the utility of treating patients experiencing refractory chronic graft-versus-host disease with ex-vivo-expanded BM-drived mesenchymal stem cells from third-party donors. The objective was to evaluate the effect and safety of such treatment on refractory chronic graft-versus-host disease.

Condition or disease Intervention/treatment Phase
Chronic Graft-versus-host Disease Biological: Mesenchymal stem cells Biological: Non-mesenchymal stem cells Phase 2

Detailed Description:

Allogeneic hematopoietic stem cell transplantation(allo-HSCT) can cure many hematologic diseases. Although great progress has been made in the prevention and treatment of side effects associated with transplantation,chronic graft-versus-host disease(cGVHD) remains an important complication that occurs in about 50% patients. The mortality of cGVHD and its complication could reach up to 50%,and cGVHD seriously influence the quality of life. At present, glucocorticoids and cyclosporine (CsA) are the first line treatment of cGVHD, but their effective rates are only 50%. If first line treatment is ineffective, second line drugs would be taken, such as mycophenolate mofetil(MMF)and rituximab. The effective rates of second line drugs are 30%-61%. The effective rates and prognosis of refractory cGVHD are even worse.

Mesenchymal stem cells (MSCs) are a form of multipotent adult stem cells that can be isolated from bone marrow (BM), adipose tissue, and cord blood. Clinical applications of human MSCs are evolving rapidly with goals of improving hematopoietic engraftment, preventing and treating GVHD after allo-HSCT and so on. However, the efficacy of treatment of refractory cGVHD using expanded BM-derived MSCs from a third-party donor is rarely reported. If such treatment could be shown to be effective and safe, BM-derived MSCs could potentially be used as an universal donor material. This would have a major impact because the generation of donor-specific MSCs is time-consuming, costly, and often impractical if the clinical status of a patient is urgent.

In the present study, the investigators will prospectively evaluate the efficacy and safety of ex-vivo-expanded BM-derived MSCs from third-party donors in treating patients with refractory cGVHD.

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Mesenchymal Stem Cells From Third-party Donors for Treatment of Refractory Chronic Graft-versus-host Disease
Study Start Date : January 2013
Estimated Primary Completion Date : December 2015
Estimated Study Completion Date : December 2016

Arms and Interventions

Arm Intervention/treatment
Experimental: Non-mesenchymal stem cells
Non-mesenchymal stem cell group refers to treatment with other second line drugs
Biological: Non-mesenchymal stem cells
Other second line drugs are taken.
Other Name: Non-MSCs
Experimental: Mesenchymal stem cells
Mesenchymal stem cell group refers to treatment with mesenchymal stem cells (1×10^6 cells/kg, intravenously)every two weeks, four times for a cycle
Biological: Mesenchymal stem cells
Mesenchymal stem cells will be intravenously infused via a central venous catheter(at a dose of 1×10^6 cells/kg, over 15 mins) every two weeks, four times for a cycle.
Other Name: MSCs

Outcome Measures

Primary Outcome Measures :
  1. The efficacy of treatment for refractory cGVHD [ Time Frame: 1 year ]
    The response criteria include complete response (CR), part response (PR), stable disease(SD) and progressive disease(PD). CR:cGVHD symptoms and signs disappear; PR:cGVHD symptoms and signs improve; SD:cGVHD symptoms and signs remain (without improvement or deterioration);PD: cGVHD symptoms and signs deteriorate.

Secondary Outcome Measures :
  1. acute and late toxic side effects of MSCs treatment [ Time Frame: 1 year ]
    Toxic side effects of treatment includes acute toxicity and late side effects. Acute toxicity principally involves the heart,live and kidney. Late toxic side effects involves principally the development of secondary tumors and relapse of the primary disease.

Other Outcome Measures:
  1. infections [ Time Frame: 1 year ]
    Infections will be mainly focused within the first 100 days after MSCs treatment.

Eligibility Criteria

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Ages Eligible for Study:   12 Years to 65 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • A patient age of 12-65 years
  • Recipients of allogeneic hematopoietic stem cell transplantation
  • Patients with refractory cGVHD
  • On a voluntary basis, patients are divided into MSCs and Non-MSCs group
  • Subjects (or their legally acceptable representatives) must have signed an informed consent document indicating that they understand the purpose of and procedures required for the study and are willing to participate in the study

Exclusion Criteria:

  • Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
  • Patients with any conditions not suitable for the trial (investigators' decision)
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01765660

China, Guangdong
Department of Hematology,Nanfang Hospital, Southern Medical University Recruiting
Guangzhou, Guangdong, China, 510515
Contact: Li Xuan    +86-020-61641612    356135708@qq.com   
Principal Investigator: Qifa Liu         
Sponsors and Collaborators
Nanfang Hospital of Southern Medical University
Sun Yat-sen University
Peking University People's Hospital
Guangdong General Hospital
Guangzhou General Hospital of Guangzhou Military Command
Southern Medical University, China
Third Affiliated Hospital, Sun Yat-Sen University
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
Guangzhou First People's Hospital
Zhongshan People's Hospital, Guangdong, China
Principal Investigator: Qifa Liu Nanfang Hospital of Southern Medical University