Mesenchymal Stem Cells in the Treatment of Relapsed/Refractory Severe Acquired Aplastic Anemia
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01297972|
Recruitment Status : Completed
First Posted : February 17, 2011
Last Update Posted : February 4, 2014
|Condition or disease||Intervention/treatment||Phase|
|Aplastic Anemia||Biological: Intravenous bone marrow mesenchymal stem cells infusion||Phase 1 Phase 2|
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.
|Study Type :||Interventional|
|Actual Enrollment :||9 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Bone Marrow Mesenchymal Stem Cells in the Treatment of Refractory Severe Acquired Aplastic Anemia|
|Study Start Date :||February 2011|
|Actual Primary Completion Date :||November 2013|
|Actual Study Completion Date :||November 2013|
- 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.
- 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 ]Adverse events like allergic reactions,infectious diseases,organ dysfunction or other related to mesenchymal stem cells infusion will be assessed.
- Level of cytopenias [ Time Frame: weekly until 6 months ]
- Transfusional requirements [ Time Frame: weekly until 6 months ]Units of blood or platelets transfused after the mesenchymal stem cells infusion will be measured and compared to previously.
- Incidence of infections and febrile neutropenia [ Time Frame: weekly until 6 months ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01297972
|Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo|
|Ribeirão Preto, São Paulo, Brazil, 14048900|
|Principal Investigator:||Diego V Clé, MD||University of São Paulo|
|Study Chair:||Rodrigo T Calado, MD||University of São Paulo|