Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Severe Aplastic Anemia

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Brief Title

Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Severe Aplastic Anemia

Official Title

Phase II Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat SAA

Brief Summary

      The purpose of this study is to evaluate the safety and efficacy of mesenchymal stem cells
      (MSCs) derived from human umbilical cord/placenta at a dose of 1.0E+6 MSC/kg in subject for
      the therapy of severe aplastic anemia (SAA).
    

Detailed Description

      Severe aplastic anemia (SAA) is a condition that involves a low level of red blood cells,
      white blood cells, and platelets without evidence of another bone marrow disease. Patients
      with severe aplastic anemia produce too few blood cells, causing fatigue, easy bruising and
      bleeding, and susceptibility to infections. In many cases, the very low blood counts result
      from an autoimmune process. The patient's own immune system damages their stem cells in bone
      marrow.

      Although immune-suppressing drugs, such as corticosteroids, CsA and ATG, have been used in
      the treatment of SAA, however, many studies have indicated that the overall response rate to
      these drugs is less than 60%. Addition, the severe side effects of these immune-suppressing
      drugs have also been observed. The management of SAA patients therefore remains
      unsatisfactory and targeted therapies are needed. Human MSCs isolated from human umbilical
      cord/placenta have been shown to have immunosuppressive, stimulating hematopoiesis and tissue
      repairing properties. This study will evaluate the safety and effectiveness of MSC
      transplantation in the SAA patients.

      This study will last 2 to 3 years. Participants will be randomly assigned to receive either
      MSC transplant and CsA therapy (experimental group) or CsA therapy alone (control group).
      Patients will undergo MSC transplant at the start of the study on Day 0. After 3 months,
      patients will receive the second MSC transplantation. After six and twelve months from the
      first transplantation, patients will be evaluated.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

SAA clinical symptoms

Secondary Outcome

 Percentage of systemic T regulatory cell population and T lymphocyte subsets

Condition

Aplastic Anemia

Intervention

Human umbilical cord-derived MSCs and cyclosporin A

Study Arms / Comparison Groups

 Human umbilical cord-derived MSCs and cyclosporin
Description:  Human umbilical cord-derived MSCs at a dose of 1.0E+6 MSC/kg, repeated to apply in trimonthly for 2 cycle and CsA 5mg/kg po for 12 months

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

Other

Estimated Enrollment

30

Start Date

August 2010

Completion Date

August 2013

Primary Completion Date

August 2013

Eligibility Criteria

        Inclusion Criteria:

          1. Patient age 18~80 years old with plan to infuse MSCs.

          2. Standard of diagnosis of aplastic anemia is according to Chinese domestic
             classification of AA for 1987.

          3. Patients must have an ECOG 0~2.

          4. No moderate or sever organ dysfunction: Ejection fraction>45%; Creatinine <176 umol/L.

          5. No active severe viral or fungus infection.

          6. Each patient must sign written informed consent.

        Exclusion Criteria:

          1. Psychiatric condition that would limit informed consent.

          2. HIV positive

          3. Positive Pregnancy Test

          4. Patient has enrolled another clinical trial study within last 4 weeks.
      

Gender

All

Ages

18 Years - 80 Years

Accepts Healthy Volunteers

No

Contacts

chengyun zheng, Ph. D, +86-531-85875635, [email protected]

Location Countries

China

Location Countries

China

Administrative Informations


NCT ID

NCT01182662

Organization ID

kongdx

Secondary IDs

No. 30670903


Study Sponsor

Shandong University

Collaborators

 National Natural Science Foundation of China

Study Sponsor

chengyun zheng, Ph. D, Principal Investigator, Department of Hematology of The 2nd Hospital of Shandong University


Verification Date

August 2010