A Phase 1/2 interventional study of bone marrow derived mesenchymal stem cells in Aplastic Anemia, sponsored by Guangzhou General Hospital of Guangzhou Military Command. Status unknown at 1 site in China. Open to participants aged 16 Years and older. Per ClinicalTrials.gov, last updated 2011-03-01.
Sponsored by Guangzhou General Hospital of Guangzhou Military Command · Phase 1/2, Interventional, and Treatment
The study is a phase I/II trial designed to establish the safety and efficacy of intravenous administration of bone marrow derived mesenchymal stem cells from related donor to patients with relapsed/refractory aplastic anemia.
Aplastic anemia (AA) is an autoimmune hematologic stem cell disease mediated by activated T-lymphocytes that leads to bone marrow dysfunction. In the presence of an empty marrow, pancytopenia, and transfusion dependence, the severity of the disease is based on neutrophil (PMN) count: nonsevere AA (nSAA; PMN > 0.5 × 109/L), severe AA (SAA;PMN 0.2- 0.5 × 109/L), and very severe AA (vSAA; PMN\< 0.2 × 109/L). Patients with nSAA can be offered supportive care, anabolic steroids, and/or low-dose steroids or cyclosporine (CsA).Patients with SAA and vSAA can be offered immunosuppressive treatment involving injections of Anti-thymocyte globulin (ATG) in combination with cyclosporine (CsA). However, some nSAA patients remains dependent to transfusion, the treatment response with ATG for SAA is at best between 50-60%,30%-40% patients relapse following an initial response to treatment, they also do not have a HLA-matched donor for bone marrow transplantation. These patients have a high risk of dying without additional treatment. Since the prognosis of these refractory and relapsed AA patients remains poor, there is a need for more safe and effective therapy that can improve response rates and remission duration in refractory and relapsed AA.
Mesenchymal stem cells (MSCs) are part of the bone marrow stem cells repertoire. The main role of MSCs is to support hematopoiesis. Recently, significant interactions between MSCs and cells from the immune system have been demonstrated:MSCs were found to downregulate T and B lymphocytes, natural killer cells (NK) and antigen presenting cells through various mechanisms, including cell-to-cell interaction and soluble factor production. MSCs can fully suppress T cell function which involves some degree of MSC activation or 'licensing' thought to involve interferon (IFN)-γ in conjunction with IL-1α, IL-1β or tumour necrosis factor-a. Non-specific suppression of T cell proliferation is mediated by soluble factors such as transforming growth factor (TGF)-β, kynurenine, prostaglandin E2 (PGE2), nitric oxide, haem oxygenase products and insulin-like growth factor binding protein. Since the haematopoietic support and immunomodulatory effects, bone marrow-derived human MSCs transplantation maybe a safe novel therapeutic approach for patients with refractory and relapsed AA.
1,733 studies on the registry are indexed under Anemia; 246 are open to participants now.
This study's planned enrollment of 50 is below the median of 94 across 1,291 interventional studies indexed under Anemia.
Browse Anemia studies →Guangzhou General Hospital of Guangzhou Military Command is the lead sponsor of 46 studies on the registry; 1 is open to participants now.
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There must be at least two of the following:
haemoglobin \< 100g/L; platelet count \< 50 x 109/L; neutrophil count \< 1.5 x 109/L, and a hypocellular bone marrow;
SAA as defined by a hypocellular bone marrow of \<25% cellularity and two of the following:
neutrophil count \< 0.5 x 109/L platelets \< 20 x 109/L reticulocytes \< 20 x 109/L nSAA as defined by a hypocellular bone marrow and cytopenia in at least two cell lines and neutrophil count > 0.5 x 109/L, and red cell and/or platelet transfusion dependence.
total bilirubin within normal institutional limits (NV: 0.0-20.5 umol/L) AST(SGOT)/ALT(SGPT) \< 2.5 × institutional upper limit of normal AST (NV: 0-35 U/L); ALT (NV: 0-40 U/L) Creatinine within normal institutional limits (NV: 53-106 umol/L) or Creatinine clearance > 1.25 ml/s for patients with creatinine levels above institutional normal.
Exclusion Criteria:
Intravenous bone marrow derived mesenchymal stem cells infusion from related donor to patients with relapsed/refractory aplastic anemia.
Biological: bone marrow derived mesenchymal stem cells
Intravenous administration of up to 6x10\^5 MSCs per kg,qw,for 4 weeks
Also known as: Mesenchymal Stem Cells, Multipotent Mesenchymal Stem Cells, Multipotent Mesenchymal Stromal Cells
Number of participants with adverse events
Time frame: up to 30 days
Hematologic response
Time frame: up to 1 year
Relapse
Time frame: up to 1 year
Clonal evolution to PNH, myelodysplasia or acute leukemia
Time frame: up to 1 year
Survival
Time frame: up to 1 year
This study is status unknown, as verified in Feb 2011. You cannot join it, but the record below documents what was studied.
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Guangzhou General Hospital of Guangzhou Military Command