Mesenchymal Stem Cells for Treatment of Refractory Chronic Graft-versus-host Disease
| Tracking Information | ||||
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| First Received Date ICMJE | January 5, 2013 | |||
| Last Updated Date | January 8, 2013 | |||
| Start Date ICMJE | January 2013 | |||
| Estimated Primary Completion Date | December 2015 (final data collection date for primary outcome measure) | |||
| Current Primary Outcome Measures ICMJE |
The efficacy of treatment for refractory cGVHD [ Time Frame: 1 year ] [ Designated as safety issue: No ] The response criteria include complete response (CR), part response (PR), stable disease(SD) and progressive disease(PD). CR:cGVHD symptoms and signs disappear; PR:cGVHD symptoms and signs improve; SD:cGVHD symptoms and signs remain (without improvement or deterioration);PD: cGVHD symptoms and signs deteriorate. |
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| Original Primary Outcome Measures ICMJE | Same as current | |||
| Change History | No Changes Posted | |||
| Current Secondary Outcome Measures ICMJE |
acute and late toxic side effects of MSCs treatment [ Time Frame: 1 year ] [ Designated as safety issue: Yes ] Toxic side effects of treatment includes acute toxicity and late side effects. Acute toxicity principally involves the heart,live and kidney. Late toxic side effects involves principally the development of secondary tumors and relapse of the primary disease. |
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| Original Secondary Outcome Measures ICMJE | Same as current | |||
| Current Other Outcome Measures ICMJE |
infections [ Time Frame: 1 year ] [ Designated as safety issue: Yes ] Infections will be mainly focused within the first 100 days after MSCs treatment. |
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| Original Other Outcome Measures ICMJE | Same as current | |||
| Descriptive Information | ||||
| Brief Title ICMJE | Mesenchymal Stem Cells for Treatment of Refractory Chronic Graft-versus-host Disease | |||
| Official Title ICMJE | Mesenchymal Stem Cells From Third-party Donors for Treatment of Refractory Chronic Graft-versus-host Disease | |||
| Brief Summary | The purpose of this study is to evaluate the utility of treating patients experiencing refractory chronic graft-versus-host disease with ex-vivo-expanded BM-drived mesenchymal stem cells from third-party donors. The objective was to evaluate the effect and safety of such treatment on refractory chronic graft-versus-host disease. | |||
| Detailed Description | Allogeneic hematopoietic stem cell transplantation(allo-HSCT) can cure many hematologic diseases. Although great progress has been made in the prevention and treatment of side effects associated with transplantation,chronic graft-versus-host disease(cGVHD) remains an important complication that occurs in about 50% patients. The mortality of cGVHD and its complication could reach up to 50%,and cGVHD seriously influence the quality of life. At present, glucocorticoids and cyclosporine (CsA) are the first line treatment of cGVHD, but their effective rates are only 50%. If first line treatment is ineffective, second line drugs would be taken, such as mycophenolate mofetil(MMF)and rituximab. The effective rates of second line drugs are 30%-61%. The effective rates and prognosis of refractory cGVHD are even worse. Mesenchymal stem cells (MSCs) are a form of multipotent adult stem cells that can be isolated from bone marrow (BM), adipose tissue, and cord blood. Clinical applications of human MSCs are evolving rapidly with goals of improving hematopoietic engraftment, preventing and treating GVHD after allo-HSCT and so on. However, the efficacy of treatment of refractory cGVHD using expanded BM-derived MSCs from a third-party donor is rarely reported. If such treatment could be shown to be effective and safe, BM-derived MSCs could potentially be used as an universal donor material. This would have a major impact because the generation of donor-specific MSCs is time-consuming, costly, and often impractical if the clinical status of a patient is urgent. In the present study, the investigators will prospectively evaluate the efficacy and safety of ex-vivo-expanded BM-derived MSCs from third-party donors in treating patients with refractory cGVHD. |
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| Study Type ICMJE | Interventional | |||
| Study Phase | Phase 2 | |||
| Study Design ICMJE | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment |
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| Condition ICMJE | Chronic Graft-versus-host Disease | |||
| Intervention ICMJE |
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| Study Arm (s) |
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| Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | ||||
| Recruitment Status ICMJE | Recruiting | |||
| Estimated Enrollment ICMJE | 60 | |||
| Estimated Completion Date | December 2016 | |||
| Estimated Primary Completion Date | December 2015 (final data collection date for primary outcome measure) | |||
| Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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| Gender | Both | |||
| Ages | 12 Years to 65 Years (Child, Adult) | |||
| Accepts Healthy Volunteers | No | |||
| Contacts ICMJE | ||||
| Listed Location Countries ICMJE | China | |||
| Removed Location Countries | ||||
| Administrative Information | ||||
| NCT Number ICMJE | NCT01765660 | |||
| Other Study ID Numbers ICMJE | NFH-MSCs-cGVHD-2013 | |||
| Has Data Monitoring Committee | Yes | |||
| Plan to Share Data | Not Provided | |||
| IPD Description | Not Provided | |||
| Responsible Party | Nanfang Hospital of Southern Medical University | |||
| Study Sponsor ICMJE | Nanfang Hospital of Southern Medical University | |||
| Collaborators ICMJE |
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| Investigators ICMJE |
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| Information Provided By | Nanfang Hospital of Southern Medical University | |||
| Verification Date | January 2013 | |||
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ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |
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