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

Chinese Academy of Medical Sciences & Peking Union Medical College

Status and phase

Unknown

Phase 3

Phase 2

Conditions

Chronic Graft Versus Host Disease

Treatments

Biological: Biological: mesenchymal stem cell

Drug: Cyclosporine and Glucocorticoid

Study type

Interventional

Funder types

Other

Identifiers

NCT01526850

D07050701350701

Details and patient eligibility

About

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.

Full 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.

Enrollment

100 estimated patients

Sex

All

Ages

2 to 60 years old

Volunteers

No Healthy Volunteers

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

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

None (Open label)

100 participants in 2 patient groups

allogenic mesenchymal stem cells (MSCs)

Experimental group

Description:

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.

Treatment:

Biological: Biological: mesenchymal stem cell

Control group

Active Comparator group

Description:

Treatment:

Drug: Cyclosporine and Glucocorticoid