Mesenchymal Stem Cells in the Treatment of Relapsed/Refractory Severe Acquired Aplastic Anemia

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Diego Villa Clé, University of Sao Paulo
ClinicalTrials.gov Identifier:
NCT01297972
First received: February 16, 2011
Last updated: February 2, 2014
Last verified: February 2014

February 16, 2011
February 2, 2014
February 2011
November 2013   (final data collection date for primary outcome measure)
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.
Same as current
Complete list of historical versions of study NCT01297972 on ClinicalTrials.gov Archive Site
  • Level of cytopenias [ Time Frame: weekly until 6 months ] [ Designated as safety issue: No ]
  • Transfusional requirements [ Time Frame: weekly until 6 months ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
Same as current
Not Provided
Not Provided
 
Mesenchymal Stem Cells in the Treatment of Relapsed/Refractory Severe Acquired Aplastic Anemia
Bone Marrow Mesenchymal Stem Cells in the Treatment of Refractory Severe Acquired Aplastic Anemia

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.

Interventional
Phase 1
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Aplastic Anemia
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.
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
9
November 2013
November 2013   (final data collection date for primary outcome measure)

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
Both
18 Years to 65 Years
No
Contact information is only displayed when the study is recruiting subjects
Brazil
 
NCT01297972
CONEP 16119
Yes
Diego Villa Clé, University of Sao Paulo
University of Sao Paulo
Not Provided
Principal Investigator: Diego V Clé, MD University of São Paulo
Study Chair: Rodrigo T Calado, MD University of São Paulo
University of Sao Paulo
February 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP