Clinical trial NCT02291770: Treatment of Chronic Graft-Versus-Host Disease With Mesenchymal Stromal Cells
Official title: Treatment of of Chronic Graft-Versus-Host Disease With Mesenchymal Stromal Cells. A Phase III Randomized Open Label Multi-center Study in Southern China.
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Chronic Graft-Versus-Host Disease
Keywords: Chronic Graft-Versus-Host Disease
Mesenchymal Stromal Cells
Lead sponsor: Guangdong Provincial People's Hospital
Sponsor class: OTHER
Collaborators: name: Sun Yat-sen University
name: Nanfang Hospital, Southern Medical University
name: Guangzhou General Hospital of Guangzhou Military Command
name: Third Affiliated Hospital, Sun Yat-Sen University
name: Guangzhou First People's Hospital
name: Academy Military Medical Science, China
First posted: 2014-11-14
Enrollment: 130
Sex: ALL
Minimum age (years): 14
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Chronic Graft-versus-Host Disease (cGvHD) is a potentially lethal disorder. A variety of second line immunosuppressive agents have been investigated but no optimal treatment has emerged. There is therefore a need for novel treatment strategies. Mesenchymal stromal cells (MSC) exhibit immunomodulatory properties and a recent pilot study suggests a response rate of 70% in steroid- refractory patients. In the present randomized study the efficacy and safety of MSC treatment will be further studied in patients with cGvHD.


Eligibility criteria: Inclusion Criteria:

* Newly diagnosed cGvHD
* Informed consent obtained from patient and donor.
* Any patient who has undergone allogeneic stem cell transplantation with c GvHD.
* Have not received additional agent for cGVHD within 3 months.
* Expected life is more than 90 days.
* Adequate pulmonary function with no evidence of chronic obstructive or severe restrictive pulmonary disease.
* Adequate cardiac function with no evidence of uncontrolled high blood pressure,congestive heart failure, angina pectoris, acute myocardial infarction within 6 months prior to the process.

Exclusion Criteria:

* Invasive fungal disease.
* Active cytomegalovirus (CMV)/Epstein-Barr virus(EBV)/varicella disease).
* Patient is with a history of hypersensitivity to bovine products.
* Relapsed malignancy.

Primary outcomes:
measure: Proportion of patients responding to treatment of cGvHD with MSC
timeFrame: 90 days
Secondary outcomes:
measure: Overall survival
timeFrame: 2 year
measure: Progression-free survival
timeFrame: 2 year
measure: Time without systemic immunosuppression
timeFrame: 2 year
measure: Cumulative incidents of non-relapse mortality
timeFrame: 2 year
measure: Adverse events
timeFrame: 2 year
Other outcomes:
measure: Quality of life
timeFrame: 2 year
measure: Immune reconstitution including monitoring of absolute T-cell subsets, B-cells, NK-cells as well as biomarkers of cGvHD
timeFrame: 2 year
Locations:
city: Guangzhou
country: China
facility: Xin Du
state: Guangdong
Study officials:
affiliation: Guangdong Provincial People's Hospital
name: Xin Du, Prof.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Weng J, He C, Lai P, Luo C, Guo R, Wu S, Geng S, Xiangpeng A, Liu X, Du X. Mesenchymal stromal cells treatment attenuates dry eye in patients with chronic graft-versus-host disease. Mol Ther. 2012 Dec;20(12):2347-54. doi: 10.1038/mt.2012.208. Epub 2012 Oct 16.
citation: Weng JY, Du X, Geng SX, Peng YW, Wang Z, Lu ZS, Wu SJ, Luo CW, Guo R, Ling W, Deng CX, Liao PJ, Xiang AP. Mesenchymal stem cell as salvage treatment for refractory chronic GVHD. Bone Marrow Transplant. 2010 Dec;45(12):1732-40. doi: 10.1038/bmt.2010.195. Epub 2010 Sep 6.