NCT01549665

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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
30

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Jan 2012

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2012

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

February 23, 2012

Completed
15 days until next milestone

First Posted

Study publicly available on registry

March 9, 2012

Completed
9 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2012

Completed
Last Updated

March 16, 2012

Status Verified

March 1, 2012

Enrollment Period

11 months

First QC Date

February 23, 2012

Last Update Submit

March 14, 2012

Conditions

Graft-Versus-Host DiseaseGVHDAllogeneic Hematopoietic TransplantDisorder Related to Transplantation

Keywords

Graft-Versus-Host DiseaseGVHDHematopoietic Stem Cell TransplantationStem Cell TransplantationAllogeneic hematopoietic transplantMesenchymal Stem CellsUnrelated DonorsCord Blood Stem Cell Transplantation

Outcome Measures

Primary Outcomes (1)

  • 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

Secondary Outcomes (1)

  • Complete, Partial Response Rate at 28 and 180 Days in participants with SR-GVHD treated with UCB-MSC

    180 Days

Interventions

Human umbilical cord blood-derived mesenchymal stem cellsBIOLOGICAL

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.

Also known as: PROMOSTEM®

Eligibility Criteria

Age1 Day - 30 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Steroid-refractory acute or chronic graft-versus-host-disease
  • Acquisition of consent form
  • years of age

You may not qualify if:

  • Withdrawal from the study
  • Progression of underlying hematologic diseases
  • Severe adverse effects related to the investigational drug

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Department of Pediatrics, Samsung Medical Center

Seoul, South Korea

RECRUITING

MeSH Terms

Conditions

Graft vs Host Disease

Condition Hierarchy (Ancestors)

Immune System Diseases

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD, PhD

Study Record Dates

First Submitted

February 23, 2012

First Posted

March 9, 2012

Study Start

January 1, 2012

Primary Completion

December 1, 2012

Study Completion

December 1, 2012

Last Updated

March 16, 2012

Record last verified: 2012-03

Locations

KR(1)