Mesenchymal Stem Cells in the Treatment of Relapsed/Refractory Severe Acquired Aplastic Anemia
This study is currently recruiting participants.
Verified August 2012 by University of Sao Paulo
Sponsor:
University of Sao Paulo
Information provided by (Responsible Party):
Diego Villa Clé, University of Sao Paulo
ClinicalTrials.gov Identifier:
NCT01297972
First received: February 16, 2011
Last updated: August 24, 2012
Last verified: August 2012
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| First Received Date ICMJE | February 16, 2011 | ||||||||
| Last Updated Date | August 24, 2012 | ||||||||
| Start Date ICMJE | February 2011 | ||||||||
| Estimated Primary Completion Date | July 2013 (final data collection date for primary outcome measure) | ||||||||
| Current Primary Outcome Measures ICMJE |
Number of Participants with Adverse Events as a Measure of Safety and Tolerability of intravenous allogeneic unrelated mesenchymal stem cells infusion in patients with severe acquired aplastic anemia. [ Time Frame: After the mesenchymal stem cells infusion up to 6 mounth after ] [ Designated as safety issue: Yes ] Adverse events like allergic reactions,infectious diseases,organ dysfunction or other related to mesenchymal stem cells infusion will be assessed. |
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| Original Primary Outcome Measures ICMJE | Same as current | ||||||||
| Change History | Complete list of historical versions of study NCT01297972 on ClinicalTrials.gov Archive Site | ||||||||
| Current Secondary Outcome Measures ICMJE |
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| 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 | Mesenchymal Stem Cells in the Treatment of Relapsed/Refractory Severe Acquired Aplastic Anemia | ||||||||
| Official Title ICMJE | Bone Marrow Mesenchymal Stem Cells in the Treatment of Refractory Severe Acquired Aplastic Anemia | ||||||||
| Brief Summary | Mesenchymal stem cells have been tested in many autoimmune disorders with encouraging results and may be an alternative to the treatment of immune-mediated severe acquired aplastic anemia. |
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| Detailed Description | Acquired aplastic anemia is a bone marrow failure syndrome characterized by and empty bone marrow and low blood counts. Most of the cases are immune-mediated. Allogeneic bone marrow transplant is the preferable treatment modality for patients younger than 40 years with a matched sibling donor. Patients not eligible for transplant are treated with intensive immunosuppressive therapy often based on anti-thymocyte globulin and cyclosporine. However, up to one third of patients treated with immunosuppression are refractory and one third of those who response eventually relapse. Refractory and relapsed cases may be the result of insufficient immunosuppression and these cases may benefit from additional immunosuppression. Mesenchymal stem cells infusion may be a potential treatment option, considering its properties to modulate the immune system. Refractory or relapsed patients with aplastic anemia will be treated with conventional immunosuppressive regimen (anti-thymocyte globulin plus cyclosporine) combined with intravenous infusion of allogeneic unrelated bone marrow mesenchymal stem cells. |
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| Study Type ICMJE | Interventional | ||||||||
| Study Phase | Phase 1 Phase 2 |
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| Study Design ICMJE | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
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| Condition ICMJE | Aplastic Anemia | ||||||||
| Intervention ICMJE | Biological: Intravenous bone marrow mesenchymal stem cells infusion
After standard immunosuppressive therapy with rabbit antithymocyte globulin 3,5 mg/Kg/day during 5 days, allogeneic unrelated bone marrow derived mesenchymal stem cells will be infused intravenously. Oral cyclosporine 5 mg/Kg/day (with dose correction weekly to keep serum cyclosporine level between 150-250 mg/dl) up to 6 months will be added. |
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| Study Arm (s) | Not Provided | ||||||||
| 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 | 10 | ||||||||
| Estimated Completion Date | July 2013 | ||||||||
| Estimated Primary Completion Date | July 2013 (final data collection date for primary outcome measure) | ||||||||
| Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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| Gender | Both | ||||||||
| Ages | 18 Years to 65 Years | ||||||||
| Accepts Healthy Volunteers | No | ||||||||
| Contacts ICMJE |
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| Location Countries ICMJE | Brazil | ||||||||
| Administrative Information | |||||||||
| NCT Number ICMJE | NCT01297972 | ||||||||
| Other Study ID Numbers ICMJE | CONEP 16119 | ||||||||
| Has Data Monitoring Committee | Yes | ||||||||
| Responsible Party | Diego Villa Clé, University of Sao Paulo | ||||||||
| Study Sponsor ICMJE | University of Sao Paulo | ||||||||
| Collaborators ICMJE | Not Provided | ||||||||
| Investigators ICMJE |
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| Information Provided By | University of Sao Paulo | ||||||||
| Verification Date | August 2012 | ||||||||
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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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