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

February 2, 2014 updated by: Diego Villa Clé, University of Sao Paulo

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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

Interventional

Enrollment (Actual)

9

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Brazil
    • São Paulo
      • Ribeirão Preto, São Paulo, Brazil, 14048900
        • Hospital Das Clinicas Da Faculdade De Medicina De Ribeirao Preto da Universidade de Sao Paulo

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
After the mesenchymal stem cells infusion up to 6 mounth after

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Level of cytopenias
Time Frame: weekly until 6 months
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.
weekly until 6 months
Incidence of infections and febrile neutropenia
Time Frame: weekly until 6 months
weekly until 6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Sao Paulo

Investigators

  • Principal Investigator: Diego V Clé, MD, University of Sao Paulo
  • Study Chair: Rodrigo T Calado, MD, University of Sao Paulo

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

February 1, 2011

Primary Completion (ACTUAL)

November 1, 2013

Study Completion (ACTUAL)

November 1, 2013

Study Registration Dates

First Submitted

February 16, 2011

First Submitted That Met QC Criteria

February 16, 2011

First Posted (ESTIMATE)

February 17, 2011

Study Record Updates

Last Update Posted (ESTIMATE)

February 4, 2014

Last Update Submitted That Met QC Criteria

February 2, 2014

Last Verified

February 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CONEP 16119

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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