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

Last updated: August 30, 2010
Sponsor: Shandong University
Overall Status: Trial Status Unknown

Phase

2

Condition

Anemia

Red Blood Cell Disorders

Aplastic Anemia

Treatment

N/A

Clinical Study ID

NCT01182662
kongdx
No. 30670903
  • Ages 18-80
  • All Genders

Study 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).

Eligibility Criteria

Inclusion

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 domesticclassification 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

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.

Study Design

Total Participants: 30
Study Start date:
August 01, 2010
Estimated Completion Date:
August 31, 2013

Study 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.

Connect with a study center

  • Department of Hematology of the 2nd Hospital of Shandong University

    Jinan, Shandong 250033
    China

    Active - Recruiting

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