Allogeneic Hematopoietic Transplant, Disorder Related to Transplantation, Graft-Versus-Host Disease, GVHD
Conditions
Keywords
Graft-Versus-Host Disease, GVHD, Hematopoietic Stem Cell Transplantation, Stem Cell Transplantation, Allogeneic hematopoietic transplant, Mesenchymal Stem Cells, Unrelated Donors, Cord Blood Stem Cell Transplantation
Brief summary
Graft-versus-host-disease (GVHD) is a major complication following allogeneic hematopoietic stem cell transplantation (allo-HSCT) which may cause acute life-threatening morbidity or chronic disabilities. Although corticosteroid, the primary agent to treat GVHD, may be effective for some patients, outcomes of those who are refractory to corticosteroid are dismal. Secondary agents can be used for steroid-refractory cases; however, their efficacy is variable and usually limited. The quality of life issue of chronic GVHD is especially important for pediatric survivors who have longer life expectancy than adults. Many in-vitro and in-vivo data support immunoregulatory properties of mesenchymal stem cells(MSCs)and possibilities of treating diseases caused by immune dysregulation such as GVHD. Recent data revealed that bone marrow-derived MSCs were very useful to treat steroid-refractory acute GVHD, which led to improved overall survival compared with controls. More recently, a number of reports suggest MSCs may also be useful in treating chronic GVHD as well as acute GVHD. It has been also reported that third party MSCs are also useful as well as those from autologous or HLA-matched donors. The investigator recently demonstrated that MSCs obtained from umbilical cord blood (UCB) have similar immunosuppressive properties as bone marrow-MSCs. UCB-MSCs can be obtained without doing any harm to donors that it may be more appropriate source of MSCs than bone marrow for off-the-shelf use. However, little is known about the safety and efficacy of UCB-MSCs in treating GVHD. Therefore, the investigator designed this study to evaluate the safety and efficacy of UCB-MSCs in treating pediatric patients with steroid-refractory acute or chronic GVHD.
Interventions
1st infusion(1x1,000,000 hMSC/kg)IV, 2nd infusion(2x1,000,000 hMSC/kg)IV and 3rd infusion(3x1,000,000 hMSC/kg)IV if no complete response at 28 days.
Sponsors
Medipost Co Ltd.
Samsung Medical Center
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Days to 30 Years
Healthy volunteers
No
Inclusion criteria
* Steroid-refractory acute or chronic graft-versus-host-disease * Acquisition of consent form * 0-30 years of age
Exclusion criteria
* Withdrawal from the study * Progression of underlying hematologic diseases * Severe adverse effects related to the investigational drug
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety of UCB-MSC in SR-GVHD | 180 days | * Vital sign * Physical examination * ECOG performance status * Mixed Lymphocyte Reaction(MLR) * Adverse effects related to infusion |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Complete, Partial Response Rate at 28 and 180 Days in participants with SR-GVHD treated with UCB-MSC | 180 Days | * Complete Response(CR) rate(%) = (Number of CR/Number of Participants)x100 * Partial Response(PR)rate(%) = (Number of PR/Number of Participants)x100 |
Countries
South Korea
Outcome results
None listed