Status Approved
First Submitted Date
2012/02/07
Registered Date
2012/03/27
Last Updated Date
2012/03/23
CRIS Required
WHO ICTRP (International Clinical Trial Registry Platform) Required
1. Background
CRIS Registration Number |
KCT0000389 |
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Unique Protocol ID | SMC 2011-06-025 |
Public/Brief Title | Investigator initiated trial to evaluate the safety and efficacy of umbilical cord blood-derived mesenchymal stem cells for treatment of pediatric patients with steroid-refractory acute or chronic graft versus host disease. |
Scientific Title | Umbilical cord blood-derived mesenchymal stem cells for the treatment of steroid-refractory acute or chronic graft-versus-host-disease |
Acronym | UCB-MSC GVHD |
MFDS Regulated Study | Yes |
IND/IDE Protocol | Yes |
Registered at Other Registry | No |
Healthcare Benefit Approval Status |
2. Institutional Review Board / Ethics Committee
Board Approval Status | Submitted approval |
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Board Approval Number | SMC 2011-06-025 |
Approval Date | 2011-08-27 |
Institutional Review Board Name | Samsung Medical Center Institutional Review Board |
Institutional Review Board Address | |
Institutional Review Board Telephone | |
Data Monitoring Committee | No |
3. Contact Details
Contact Person for Principal Investigator / Scientific Queries | |
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Name | Hong Hoe Koo |
Title | MD, PhD |
Telephone | +82-2-3410-3524 |
Affiliation | Samsung Medical Center |
Address | |
Contact Person for Public Queries | |
Name | Meong Hi Son |
Title | MD, Fellow |
Telephone | +82-2-3410-0870 |
Affiliation | Samsung Medical Center |
Address | |
Contact Person for Updating Information | |
Name | Na Kyoung Ahn |
Title | Associate Manager |
Telephone | +82-2-3465-6744 |
Affiliation | Medipost |
Address |
4. Status
Study Site | Single | |
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Overall Recruitment Status | Recruiting | |
Date of First Enrollment | 2012-01-05 Actual | |
Target Number of Participant | 30 | |
Primary Completion Date | 2012-12-31 , Anticipated | |
Study Completion Date | 2012-12-31 , Anticipated | |
Recruitment Status by Participating Study Site 1 | ||
Name of Study | Samsung Medical Center | |
Recruitment Status | Recruiting | |
Date of First Enrollment | 2012-01-05 , |
5. Source of Monetary / Material Support
1. Source of Monetary/Material Support | |
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Organization Name | Medipost |
Organization Type | Pharmaceutical Company |
Project ID | MP-CR-008 |
6. Sponsor Organization
1. Sponsor Organization | |
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Organization Name | Samsung Medical Center |
Organization Type | Medical Institute |
7. Study Summary
Lay Summary | Graft-versus-host-disease(GVHD) is a major complication following allogeneic hematopoietic stem cell transplantation 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 used for steroid-refractory cases in pediatric group is limited. 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. We designed this study to evaluate the safety and efficacy of UCB-MSCs in treating pediatric patients with steroid-refractory acute or chronic GVHD. |
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8. Study Design
Study Type | Interventional Study |
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Study Purpose | Treatment |
Phase | Phase1/Phase2 |
Intervention Model | Single Group |
Blinding/Masking | Open |
Allocation | Not Applicable |
Intervention Type | /Biological/Vaccine |
Intervention Description | Allogeneic-Unrelated, Umbilical Cord Blood-Derived Mesenchymal stem cells( PROMOSTEM ®). 1st MSC infusion(1x10^6cells/kg)IV, 2nd MSC infusion(2x10^6cells/kg)IV and 3rd MSC infusion(3x10^6cells/kg)IV if no complete response at 28 days. - Follow-up for 180 days after first infusion. |
Number of Arms | 1 |
Arm 1 |
Arm Label PROMOSTEM |
Target Number of Participant 30 |
|
Arm Type Others |
|
Arm Description Allogeneic-Unrelated, Umbilical Cord Blood-Derived Mesenchymal stem cells( PROMOSTEM ®). 1st MSC infusion(1x10^6cells/kg)IV, 2nd MSC infusion(2x10^6cells/kg)IV and 3rd MSC infusion(3x10^6cells/kg)IV if no complete response at 28 days. - Follow-up for 180 days after first infusion. |
9. Subject Eligibility
Condition(s)/Problem(s) |
* (S00-T98)Injury, poisoning and certain other consequences of external causes (T86.0)Bone-marrow transplant rejection |
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Rare Disease | No |
Inclusion Criteria |
Gender Both |
Age 0Year~30Year |
|
Description 1. Steroid-refractory acute or chronic graft-versus-host-disease 2. Acquisition of consent form 3. 0-30 years of age |
|
Exclusion Criteria |
1. Withdrawal from the study 2. Progression of underlying hematologic diseases 3. Severe adverse effects related to the investigational drug |
Healthy Volunteers | No |
10. Outcome Measure(s)
Type of Primary Outcome | /Safety/Efficacy | |
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Primary Outcome(s) 1 | ||
Outcome | Safety(Vital sign, Physical examination, ECOG performance status/Adverse effects related to infusion) |
|
Timepoint | After 1~2 weeks of infusion/During the infusion |
|
Primary Outcome(s) 2 | ||
Outcome | Treatment response of GVHD |
|
Timepoint | Day28, 180 |
|
Secondary Outcome(s) 1 | ||
Outcome | Pediatric Quality of Life inventory |
|
Timepoint | Day28, 180 |
11. Study Results and Publication
Result Registered |
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12. Sharing of Study Data(Deidentified Individual-Patient Data, IPD)
Sharing Statement | Not provided at time of Registration |
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