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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
Sponsor:
Information provided by (Responsible Party):
Diego Villa Clé, University of Sao Paulo

Tracking Information
First Submitted Date  ICMJE February 16, 2011
First Posted Date  ICMJE February 17, 2011
Last Update Posted Date February 4, 2014
Study Start Date  ICMJE February 2011
Actual Primary Completion Date November 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 16, 2011)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 16, 2011)
  • 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 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Mesenchymal Stem Cells in the Treatment of Relapsed/Refractory Severe Acquired Aplastic Anemia
Official Title  ICMJE 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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Aplastic Anemia
Intervention  ICMJE 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.
Study Arms  ICMJE Not Provided
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 2, 2014)
9
Original Estimated Enrollment  ICMJE
 (submitted: February 16, 2011)
5
Actual Study Completion Date  ICMJE November 2013
Actual Primary Completion Date November 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Brazil
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01297972
Other Study ID Numbers  ICMJE CONEP 16119
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Diego Villa Clé, University of Sao Paulo
Study Sponsor  ICMJE University of Sao Paulo
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Diego V Clé, MD University of São Paulo
Study Chair: Rodrigo T Calado, MD University of São Paulo
PRS Account University of Sao Paulo
Verification Date February 2014

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