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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified March 2012 by Samsung Medical Center.
Recruitment status was  Recruiting
Sponsor:
Collaborator:
Medipost Co Ltd.
Information provided by (Responsible Party):
Hong Hoe Koo, Samsung Medical Center
ClinicalTrials.gov Identifier:
NCT01549665
First received: February 23, 2012
Last updated: March 14, 2012
Last verified: March 2012
  Purpose

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.


Condition Intervention Phase
Graft-Versus-Host Disease
GVHD
Allogeneic Hematopoietic Transplant
Disorder Related to Transplantation
Biological: Human umbilical cord blood-derived mesenchymal stem cells
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Umbilical Cord Blood-derived Mesenchymal Stem Cells for the Treatment of Steroid-refractory Acute or Chronic Graft-versus-host-disease

Resource links provided by NLM:


Further study details as provided by Samsung Medical Center:

Primary Outcome Measures:
  • Safety of UCB-MSC in SR-GVHD [ Time Frame: 180 days ] [ Designated as safety issue: Yes ]
    • Vital sign
    • Physical examination
    • ECOG performance status
    • Mixed Lymphocyte Reaction(MLR)
    • Adverse effects related to infusion


Secondary Outcome Measures:
  • Complete, Partial Response Rate at 28 and 180 Days in participants with SR-GVHD treated with UCB-MSC [ Time Frame: 180 Days ] [ Designated as safety issue: No ]
    • Complete Response(CR) rate(%) = (Number of CR/Number of Participants)x100
    • Partial Response(PR)rate(%) = (Number of PR/Number of Participants)x100


Estimated Enrollment: 30
Study Start Date: January 2012
Estimated Study Completion Date: December 2012
Estimated Primary Completion Date: December 2012 (Final data collection date for primary outcome measure)
Intervention Details:
    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.
    Other Name: PROMOSTEM®
  Eligibility

Ages Eligible for Study:   up to 30 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01549665

Locations
Korea, Republic of
Department of Pediatrics, Samsung Medical Center Recruiting
Seoul, Korea, Republic of
Contact: Hong Hoe Koo, MD, PhD    +82-2-3410-3524    hhkoo.koo@samsung.com   
Contact: Keon Hee Yoo, M.D., Ph.D.    +82-2-3410-3532    hema2170@skku.edu   
Principal Investigator: Hong Hoe Koo, MD, PhD         
Sponsors and Collaborators
Samsung Medical Center
Medipost Co Ltd.
  More Information

No publications provided

Responsible Party: Hong Hoe Koo, MD, PhD, Samsung Medical Center
ClinicalTrials.gov Identifier: NCT01549665     History of Changes
Other Study ID Numbers: MP-CR-008
Study First Received: February 23, 2012
Last Updated: March 14, 2012
Health Authority: Korea: Food and Drug Administration

Keywords provided by Samsung Medical Center:
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

Additional relevant MeSH terms:
Graft vs Host Disease
Immune System Diseases

ClinicalTrials.gov processed this record on September 18, 2014