Clinical trial NCT01549665: Umbilical Cord Blood-derived Mesenchymal Stem Cells for the Treatment of Steroid-refractory Acute or Chronic Graft-versus-host-disease
Official title: Umbilical Cord Blood-derived Mesenchymal Stem Cells for the Treatment of Steroid-refractory Acute or Chronic Graft-versus-host-disease
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Graft-Versus-Host Disease
GVHD
Allogeneic Hematopoietic Transplant
Disorder Related to Transplantation
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
Lead sponsor: Samsung Medical Center
Sponsor class: OTHER
Collaborators: name: Medipost Co Ltd.
First posted: 2012-03-09
Enrollment: 30
Sex: ALL
Minimum age (years): 1
Maximum age (years): 30
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Safety of UCB-MSC in SR-GVHD
timeFrame: 180 days
Secondary outcomes:
measure: Complete, Partial Response Rate at 28 and 180 Days in participants with SR-GVHD treated with UCB-MSC
timeFrame: 180 Days
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Department of Pediatrics, Samsung Medical Center
Study officials:
References: