Brief Title
Mesenchymal Stem Cells in the Treatment of Relapsed/Refractory Severe Acquired Aplastic Anemia
Official Title
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.
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.
Study Phase
Phase 1/Phase 2
Study Type
Interventional
Primary Outcome
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.
Secondary Outcome
Level of cytopenias
Condition
Aplastic Anemia
Intervention
Intravenous bone marrow mesenchymal stem cells infusion
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
9
Start Date
February 2011
Completion Date
November 2013
Primary Completion Date
November 2013
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of Acquired Aplastic Anemia
- Relapse/refractory to at least 1 immunosuppressive first line treatment
- Not eligible to allogeneic bone marrow transplantation
Exclusion Criteria:
- Previous or current malignancy
- Active or latent infectious disease
- Positive serologic tests for HIV, HCV, HBV, HTLV-1 and 2, Syphilis or Chagas disease
- Previous drug reaction for antithymocyte globulin, cyclosporin or corticosteroids
- Severe organic impairment (renal, hepatic, cardiac, pulmonary)
- Uncontrolled hypertension or diabetes
- Pregnancy
- Previous history of allergic reaction to penicillin or streptomycin
- Severe psychiatric disorder
Gender
All
Ages
18 Years - 65 Years
Accepts Healthy Volunteers
No
Contacts
Diego V Clé, MD, ,
Location Countries
Brazil
Location Countries
Brazil
Administrative Informations
NCT ID
NCT01297972
Organization ID
CONEP 16119
Responsible Party
Principal Investigator
Study Sponsor
University of Sao Paulo
Study Sponsor
Diego V Clé, MD, Principal Investigator, University of São Paulo
Verification Date
February 2014