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

This study is currently recruiting participants.
Verified August 2012 by University of Sao Paulo
Sponsor:
Information provided by (Responsible Party):
Diego Villa Clé, University of Sao Paulo
ClinicalTrials.gov Identifier:
NCT01297972
First received: February 16, 2011
Last updated: August 24, 2012
Last verified: August 2012
History of Changes
  Purpose

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.


Condition Intervention Phase
Aplastic Anemia
 Biological: Intravenous bone marrow mesenchymal stem cells infusion
 Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Bone Marrow Mesenchymal Stem Cells in the Treatment of Refractory Severe Acquired Aplastic Anemia

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by University of Sao Paulo:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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 ]

Estimated Enrollment: 10
Study Start Date: February 2011
Estimated Study Completion Date: July 2013
Estimated Primary Completion Date: July 2013 (Final data collection date for primary outcome measure)
Intervention Details:
    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.
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.

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01297972

Contacts
Contact: Diego V Clé, MD 55-16-36022610 diegocle@yahoo.com
Contact: Rodrigo T Calado, MD 55-16-36022169 rtcalado@fmrp.usp.br

Locations
Brazil
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo Recruiting
Ribeirão Preto, São Paulo, Brazil, 14048900
Contact: Diego V Clé, MD     55-16-81126986     diegocle@yahoo.com    
Sponsors and Collaborators
University of Sao Paulo
Investigators
Principal Investigator: Diego V Clé, MD University of São Paulo
Study Chair: Rodrigo T Calado, MD University of São Paulo
  More Information

Publications:

Responsible Party: Diego Villa Clé, MD, University of Sao Paulo
ClinicalTrials.gov Identifier: NCT01297972     History of Changes
Other Study ID Numbers: CONEP 16119
Study First Received: February 16, 2011
Last Updated: August 24, 2012
Health Authority: Brazil: National Committee of Ethics in Research

Keywords provided by University of Sao Paulo:
Aplastic Anemia
Mesenchymal Stem Cells

Additional relevant MeSH terms:
Anemia
Anemia, Aplastic
Hematologic Diseases
Bone Marrow Diseases

ClinicalTrials.gov processed this record on June 17, 2013