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

This study is currently recruiting participants.
Verified January 2013 by Nanfang Hospital of Southern Medical University
Sponsor:
Collaborators:
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 Municipal People’s Hospital
Guangdong Provincial People's Hospital
Information provided by (Responsible Party):
Nanfang Hospital of Southern Medical University
ClinicalTrials.gov Identifier:
NCT01765660
First received: January 5, 2013
Last updated: January 8, 2013
Last verified: January 2013
  Purpose

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 Intervention Phase
Chronic Graft-versus-host Disease
Biological: Mesenchymal stem cells
Biological: Non-mesenchymal stem cells
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Mesenchymal Stem Cells From Third-party Donors for Treatment of Refractory Chronic Graft-versus-host Disease

Resource links provided by NLM:


Further study details as provided by Nanfang Hospital of Southern Medical University:

Primary Outcome Measures:
  • The efficacy of treatment for refractory cGVHD [ Time Frame: 1 year ] [ Designated as safety issue: No ]
    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:
  • acute and late toxic side effects of MSCs treatment [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    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:
  • infections [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    Infections will be mainly focused within the first 100 days after MSCs treatment.


Estimated Enrollment: 60
Study Start Date: January 2013
Estimated Study Completion Date: December 2016
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Ages Eligible for Study:   12 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01765660

Locations
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 Municipal People’s Hospital
Guangdong Provincial People's Hospital
Investigators
Principal Investigator: Qifa Liu Nanfang Hospital of Southern Medical University
  More Information

Publications:
Responsible Party: Nanfang Hospital of Southern Medical University
ClinicalTrials.gov Identifier: NCT01765660     History of Changes
Other Study ID Numbers: NFH-MSCs-cGVHD-2013
Study First Received: January 5, 2013
Last Updated: January 8, 2013
Health Authority: China: Ethics Committee

Keywords provided by Nanfang Hospital of Southern Medical University:
Hematopoietic Stem Cell Transplantation
Mesenchymal Stem Cells
Chronic Graft-versus-host disease

Additional relevant MeSH terms:
Graft vs Host Disease
Immune System Diseases

ClinicalTrials.gov processed this record on April 21, 2014