Umbilical Cord Blood-derived Mesenchymal Stem Cells for the Treatment of Steroid-refractory Acute or Chronic Graft-versus-host-disease

Graft-Versus-Host Disease Clinical Trial

— GVHD-MSC  

Official title:

Umbilical Cord Blood-derived Mesenchymal Stem Cells for the Treatment of Steroid-refractory Acute or Chronic Graft-versus-host-disease

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01549665
• Other study ID #: MP-CR-008
• Status: Recruiting
• Phase: Phase 1/Phase 2
• First received: February 23, 2012
• Last updated: March 14, 2012
• Start date: January 2012
• Est. completion date: December 2012

Study information

• Verified date: March 2012
• Source: Samsung Medical Center
• Contact: n/a
• Is FDA regulated: No
• Health authority: Korea: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 30
• Est. completion date: December 2012
• Est. primary completion date: December 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 30 Years

Eligibility

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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Allogeneic Hematopoietic Transplant
• Disorder Related to Transplantation
• Graft vs Host Disease
• Graft-Versus-Host Disease
• GVHD

Intervention

Biological:
• Human umbilical cord blood-derived mesenchymal stem cells
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.

Locations

Country	Name	City	State
Korea, Republic of	Department of Pediatrics, Samsung Medical Center	Seoul

Sponsors (2)

Lead Sponsor	Collaborator
Samsung Medical Center	Medipost Co Ltd.

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety of UCB-MSC in SR-GVHD	Vital sign; Physical examination; ECOG performance status; Mixed Lymphocyte Reaction(MLR); Adverse effects related to infusion	180 days	Yes
Secondary	Complete, Partial Response Rate at 28 and 180 Days in participants with SR-GVHD treated with UCB-MSC	Complete Response(CR) rate(%) = (Number of CR/Number of Participants)x100; Partial Response(PR)rate(%) = (Number of PR/Number of Participants)x100	180 Days	No