MSCsTcGVHD: Efficacy and Safety Study of Allogenic Mesenchymal Stem Cells for Patients With Chronic Graft Versus Host Disease

Sponsor
Chinese Academy of Medical Sciences (Other)
Overall Status
Unknown status
CT.gov ID
NCT01526850
Collaborator
Zhejiang University (Other), Chinese PLA General Hospital (Other), 307 Hospital of PLA (Other), Peking Union Medical College (Other)
100
Enrollment
1
Location
2
Arms
28
Duration (Months)
3.6
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The primary purpose of the study is to evaluate the safety and efficacy of mesenchymal stem cells (MSC) for the treatment of patients who have developed an extensive chronic graft versus host disease (with skin and/or liver damage) after HSCs transplantation and do not respond to first-line therapy.

The secondary purpose of the study is to evaluate the effect of mesenchymal stem cells (MSC) on one-year survival rate, long-term survival rate, life quality and recurrence of patients who have developed an extensive chronic graft versus host disease (with skin and/or liver damage) after HSCs transplantation and do not respond to hormone treatment.

Condition or DiseaseIntervention/TreatmentPhase
  • Biological: Biological: mesenchymal stem cell
  • Drug: Cyclosporine and Glucocorticoid
Phase 2/Phase 3

Detailed Description

Chronic Graft-versus-host disease (GVHD), with the incidence of 30%-60%, is a serious late complication of allogeneic hematopoietic stem cell transplantation (HSCT) and is the major cause of death in the late stage of transplantation. According to targeted organs, cGVHD is divided into two types, limited cGVHD and extensive cGVHD. Extensive cGVHD needs systemic immunosuppressant treatment. However, currently standard first-line regimen including cyclophosphamide and prednisolone is only effective for some patients. Novel treatment is urgently needed. Our previous study has shown that mesenchymal stem cells (MSCs) are effective for cGVHD patients with multiple skin damage. To further explore the therapeutic effect of MSCs for extensive cGVHD, we plan to conduct a multi-center clinical trial. Patients who developed an extensive cGVHD (with skin and/or liver damage) after HSCs transplantation and do not respond to first-line therapy are enrolled. They will be randomly divided into two groups which will receive MSCs and routine second-line drugs respectively. We will evaluate the efficacy and safety of MSCs for extensive cGVHD by comparison of symptom improvement, survival rate, recurrence as well as side effects in the two groups.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
100 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Phase Ⅱ/Ⅲ Clinical Trial, Multicenter, Randomized, Controlled, for the Evaluation of Efficacy and Safety of Therapy With Allogenic Mesenchymal Stem Cells in Patients With Chronic Graft Versus Host Disease
Study Start Date :
Feb 1, 2012
Anticipated Primary Completion Date :
Dec 1, 2013
Anticipated Study Completion Date :
Jun 1, 2014

Arms and Interventions

ArmIntervention/Treatment
Experimental: allogenic mesenchymal stem cells (MSCs)

patients who have developed an extensive chronic graft versus host disease (with skin and/or liver damage) after HSCs transplantation and do not respond to standard first-line regimen including cyclophosphamide and prednisolone.

Biological: Biological: mesenchymal stem cell
Mesenchymal stem cells, 1-2×107, bone marrow injection, once a week for the first four weeks; whether to continue after four weeks depends on patients' symptoms.
Other Names:
  • Regenerative medicine: MSCs
  • Active Comparator: Control group

    patients who have developed an extensive chronic graft versus host disease (with skin and/or liver damage) after HSCs transplantation and do not respond to standard first-line regimen including cyclophosphamide and prednisolone.

    Drug: Cyclosporine and Glucocorticoid
    Calmodulin inhibitors such as cyclosporine, combined with Glucocorticoid 0.5-1mg/kg/d ,to the end of the study.
    Other Names:
  • Calmodulin inhibitors combined with Glucocorticoid
  • Outcome Measures

    Primary Outcome Measures

    1. The total Response rate defined as patients with complete and partial response [1 year after MSCs administration.]

    Secondary Outcome Measures

    1. one-year survival rate [1 year after MSCs administration]

    2. disease relapse [2 years after MSCs administration]

    3. quality of life [2 years after MSCs administration]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    2 Years to 60 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:
    • Extensive cGVHD with skin and/or liver damage developed after allogeneic hematopoietic stem cell transplantation

    • cGVHD that do not response to conventional immunosuppressant treatment for two months

    • KPS>= 30

    • informed consent from the patient

    Exclusion Criteria:
    • Extensive cGVHD without skin or liver damage

    • With other acute severe complications

    • In pregnancy or lactation

    • Disease relapses

    • With non-hematological malignancy

    • Have a history of mental disorder, drug or alcohol abuse over the past five years

    • Allergic

    • Participate in other clinical trial within three months before the start of this trial

    • With bone marrow fibrosis

    • Have undergone hematopoietic stem cell transplantation to treat solid tumor

    Contacts and Locations

    Locations

    SiteCityStateCountryPostal Code
    1Zhejiang UniversityHangzhouZhejiangChina

    Sponsors and Collaborators

    • Chinese Academy of Medical Sciences
    • Zhejiang University
    • Chinese PLA General Hospital
    • 307 Hospital of PLA
    • Peking Union Medical College

    Investigators

    • Principal Investigator: He Huang, MD, Zhejiang University
    • Principal Investigator: Chunhua Zhao, MD,PHD, Peking Union Medical College

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    Responsible Party:
    Robert Chunhua Zhao, MD, PhD, MD, PhD,Professor of medicine, Chinese Academy of Medical Sciences
    ClinicalTrials.gov Identifier:
    NCT01526850
    Other Study ID Numbers:
    • D07050701350701
    First Posted:
    Feb 6, 2012
    Last Update Posted:
    Aug 3, 2012
    Last Verified:
    Feb 1, 2012
    Keywords provided by Robert Chunhua Zhao, MD, PhD, MD, PhD,Professor of medicine, Chinese Academy of Medical Sciences
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Aug 3, 2012