Clinical trial NCT01526850: Efficacy and Safety Study of Allogenic Mesenchymal Stem Cells for Patients With Chronic Graft Versus Host Disease
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 type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
PHASE3
Conditions: Chronic Graft Versus Host Disease
Keywords: cGVHD
Lead sponsor: ChineseAMS
Sponsor class: UNKNOWN
Collaborators: name: Zhejiang University
name: Chinese PLA General Hospital
name: 307 Hospital of PLA
name: Peking Union Medical College
First posted: 2012-02-06
Enrollment: 100
Sex: ALL
Minimum age (years): 2
Maximum age (years): 60
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: The total Response rate defined as patients with complete and partial response
timeFrame: 1 year after MSCs administration.
Secondary outcomes:
measure: one-year survival rate
timeFrame: 1 year after MSCs administration
measure: disease relapse
timeFrame: 2 years after MSCs administration
measure: quality of life
timeFrame: 2 years after MSCs administration
Other outcomes:
Locations:
city: Hangzhou
country: China
facility: Zhejiang University
state: Zhejiang
Study officials:
affiliation: Zhejiang University
name: He Huang, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Peking Union Medical College
name: Chunhua Zhao, MD,PHD
role: PRINCIPAL_INVESTIGATOR
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