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

This study is currently recruiting participants. (see Contacts and Locations)
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

January 5, 2013
January 8, 2013
January 2013
December 2015   (final data collection date for primary outcome measure)
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.
Same as current
Complete list of historical versions of study NCT01765660 on ClinicalTrials.gov Archive Site
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.
Same as current
infections [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
Infections will be mainly focused within the first 100 days after MSCs treatment.
Same as current
 
Mesenchymal Stem Cells for Treatment of Refractory Chronic Graft-versus-host Disease
Mesenchymal Stem Cells From Third-party Donors for Treatment of Refractory Chronic Graft-versus-host Disease

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.

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.

Interventional
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Chronic Graft-versus-host Disease
  • 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
  • Biological: Non-mesenchymal stem cells
    Other second line drugs are taken.
    Other Name: Non-MSCs
  • Experimental: Non-mesenchymal stem cells
    Non-mesenchymal stem cell group refers to treatment with other second line drugs
    Intervention: Biological: Non-mesenchymal stem cells
  • 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
    Intervention: Biological: Mesenchymal stem cells

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
60
December 2016
December 2015   (final data collection date for primary outcome measure)

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)
Both
12 Years to 65 Years
No
China
 
NCT01765660
NFH-MSCs-cGVHD-2013
Yes
Nanfang Hospital of Southern Medical University
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
Principal Investigator: Qifa Liu Nanfang Hospital of Southern Medical University
Nanfang Hospital of Southern Medical University
January 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP