A Phase 1/2 interventional study of Intravenous bone marrow mesenchymal stem cells infusion in Aplastic Anemia, sponsored by University of Sao Paulo. Completed at 1 site in Brazil. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2014-02-04.
Sponsored by University of Sao Paulo · Phase 1/2, Interventional, and Treatment
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.
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.
1,733 studies on the registry are indexed under Anemia; 246 are open to participants now.
This study's enrollment of 9 is below the median of 94 across 1,291 interventional studies indexed under Anemia.
Browse Anemia studies →University of Sao Paulo is the lead sponsor of 1,005 studies on the registry; 90 are open to participants now.
Of its 7 completed or terminated interventional studies of FDA-regulated products, 0 (0%) have results posted.
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Exclusion Criteria:
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.
Adverse events like allergic reactions,infectious diseases,organ dysfunction or other related to mesenchymal stem cells infusion will be assessed.
Time frame: After the mesenchymal stem cells infusion up to 6 mounth after
Level of cytopenias
Time frame: weekly until 6 months
Transfusional requirements
Units of blood or platelets transfused after the mesenchymal stem cells infusion will be measured and compared to previously.
Time frame: weekly until 6 months
Incidence of infections and febrile neutropenia
Time frame: weekly until 6 months
This study is completed, as verified in Feb 2014. You cannot join it, but the record below documents what was studied.
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University of Sao Paulo