Safety and Efficacy Study of Umbilical Cord Blood-Drived Mesenchymal Stem Cells to Promote Engraftment of Unrelated Hematopoietic Stem Cell Transplantation

Tracking Information
First Submitted Date ICMJE	January 13, 2009
First Posted Date ICMJE	January 15, 2009
Last Update Posted Date	April 23, 2012
Study Start Date ICMJE	August 2008
Actual Primary Completion Date	February 2010 (Final data collection date for primary outcome measure)
Current Primary Outcome Measures ICMJE; (submitted: January 14, 2009)	- Day of neutrophil engraftment - Day of platelet engraftment - Evaluation of chimerism - Evaluation of engraftment rate [ Time Frame: 28 and 100 days ]
Original Primary Outcome Measures ICMJE	Same as current
Change History	Complete list of historical versions of study NCT00823316 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ICMJE; (submitted: January 14, 2009)	- AGVHD grade [ Time Frame: 100 days ]
Original Secondary Outcome Measures ICMJE	Same as current
Current Other Outcome Measures ICMJE	Not Provided
Original Other Outcome Measures ICMJE	Not Provided
Descriptive Information
Brief Title ICMJE	Safety and Efficacy Study of Umbilical Cord Blood-Drived Mesenchymal Stem Cells to Promote Engraftment of Unrelated Hematopoietic Stem Cell Transplantation
Official Title ICMJE	Phase 1/2 Study of Umbilical Cord Blood-Drived Mesenchymal Stem Cells Infusion for Promotion of Engraftment and Prevention of an Graft Rejection and Graft-versus-Host Disease After Unrelated Hematopoietic Stem Cell Transplantation.
Brief Summary	The purpose of this study is to evaluate the safety and efficacy of PROMOSTEM (human umbilical cord blood-derived mesenchymal stem cells) at a dose of 1 and 5x1,000,000 hMSC/kg in subject for the promotion of an engraftment and prevention of graft rejection and Graft-Versus-Host Disease after unrelated hematopoietic stem cell transplantation for children with acute leukemia.
Detailed Description	Hematopoietic stem cell transplantation (HSCT) is a procedure in which progenitor cells capable of reconstituting normal bone marrow function are administered to a patient. This procedure has been used to treat adults and children with life-threatening hematological malignancies and congenital immunodeficiency disorders.; In HSCT, therapeutic goal is an elimination of disease and enrichment of regenerating capacity to achieve engraftments resulting in continued generation of functional blood elements from the engrafted living cells. Transplantation of unrelated hematopoietic stem cells originating either from adult bone marrow or from peripheral blood often leads to graft-versus host-disease (GvHD), opportunistic infections and graft failure after transplantation.; In HSCT, MSC have been used as a therapy for GvHD and other complications. The aim of MSC infusions in HSCT is to use the cells' immunomodulatory effects to promote engraftment and to reduce the immunological reactions giving rise to GvHD.; There is a growing interest in co-transplantation of MSC and HSC to improve the donor outcome in the unrelated HSCT condition.
Study Type ICMJE	Interventional
Study Phase	Phase 1; Phase 2
Study Design ICMJE	Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment
Condition ICMJE	Acute Leukemia
Intervention ICMJE	Biological: Human umbilical cord blood-derived mesenchymal stem cells 1x1,000,000 hMSC/kg, IV after unrelated HSCT Other Name: PROMOSTEM; Biological: Human umbilical cord blood-derived mesenchymal stem cells 5x1,000,000 hMSC/kg, IV after unrelated HSCT Other Name: PROMOSTEM
Study Arms	Experimental: 1 at a dose of 1x1,000,000 hMSC/kg Intervention: Biological: Human umbilical cord blood-derived mesenchymal stem cells; Experimental: 2 at a dose of 5x1,000,000 hMSC/kg Intervention: Biological: Human umbilical cord blood-derived mesenchymal stem cells
Publications *	Not Provided
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status ICMJE	Completed
Actual Enrollment ICMJE; (submitted: January 14, 2009)	10
Original Estimated Enrollment ICMJE	Same as current
Actual Study Completion Date	February 2010
Actual Primary Completion Date	February 2010 (Final data collection date for primary outcome measure)
Eligibility Criteria ICMJE	Inclusion Criteria: Patient age 2~19 years old with plan to have unrelated hematopoietic stem cell transplantation with acute leukemia; Patient never has an experience of hematopoietic stem cell transplantation; Patient must have an acute leukemia with a complete remission.; Patients must have an ECOG 0~2.; No moderate or sever organ dysfunction : Ejection fraction > 45%; Creatinine <2.0 mg/ml; Serum bilirubin < 2 mg/ml; AST/ALT < 200 IU/L.; Patient must not have an transplantation with different source of hematopoietic stem cell such as bone marrow and cord blood.; Patient must not have an infection needed an administration of non-oral antibiotics.; No active severe infection derived form virus or fungus.; Each patient / patient's guardian must sign written informed consent. Exclusion Criteria: Patient has previously received hematopoietic stem cell transplantation.; Patient plans to have a related hematopoietic stem cell transplantation.; Patient has a severe internal disease.; Patient has enrolled another clinical trial study within last 4 weeks.
Sex/Gender	Sexes Eligible for Study: All
Ages	2 Years to 19 Years (Child, Adult)
Accepts Healthy Volunteers	No
Contacts ICMJE	Contact information is only displayed when the study is recruiting subjects
Listed Location Countries ICMJE	Korea, Republic of
Removed Location Countries
Administrative Information
NCT Number ICMJE	NCT00823316
Other Study ID Numbers ICMJE	MP-CR-MSC003
Has Data Monitoring Committee	Yes
U.S. FDA-regulated Product	Not Provided
IPD Sharing Statement	Not Provided
Responsible Party	Medipost Co Ltd.
Study Sponsor ICMJE	Medipost Co Ltd.
Collaborators ICMJE	Not Provided
Investigators ICMJE	Principal Investigator: Hong Hoe Koo, MD, PhD Samsung Medical Center
PRS Account	Medipost Co Ltd.
Verification Date	April 2012
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP