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

August 30, 2010 updated by: Shandong University

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

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).

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Anticipated)

30

Phase

  • Phase 2

Contacts and Locations

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

Study Contact

Study Locations

  • China
    • Shandong
      • Jinan, Shandong, China, 250033
        • Recruiting
        • Department of Hematology of the 2nd Hospital of Shandong University
        • Contact:

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 80 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

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: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Single

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Human umbilical cord-derived MSCs and cyclosporin
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
Other: Human umbilical cord-derived MSCs and cyclosporin A
1.0E+6 MSC/kg, IV drop and repeat to apply in trimonthly for 2 cycle and cyclosporin A 5mg/kg po for 12 months
Active Comparator: cyclosporine A
cyclosporine A at a dose of 5 mg CsA/kg
Other: cyclosporin A
cyclosporin A 5mg/kg po for 12 months

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
SAA clinical symptoms
Time Frame: 1 year
Anemia symptoms, bleeding and infection will be mainly observed in every monthly after transplanting MSCs for one year.
1 year
The number of blood cells
Time Frame: 1 year
The number of blood cells, which contains WBC, Neu, RBC, Hb,PLT and reticulocyte, will be mainly tested monthly after transplantion of MSCs for one year
1 year
Bone borrow hemocytology
Time Frame: 1 year
Bone borrow cytomorphologic examination will be tested in every 3 months after transplantion of MSCs for one year.
1 year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of systemic T regulatory cell population and T lymphocyte subsets
Time Frame: 1 year
Percentages of T regulatory cell population and T lymphocyte subsets in peripheral blood will be tested in every 3 months after transplanting MSCs for one year.
1 year

Collaborators and Investigators

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

Sponsor

Shandong University

Collaborators

National Natural Science Foundation of China

Investigators

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

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

August 1, 2010

Primary Completion (Anticipated)

August 1, 2013

Study Completion (Anticipated)

August 1, 2013

Study Registration Dates

First Submitted

August 11, 2010

First Submitted That Met QC Criteria

August 16, 2010

First Posted (Estimate)

August 17, 2010

Study Record Updates

Last Update Posted (Estimate)

August 31, 2010

Last Update Submitted That Met QC Criteria

August 30, 2010

Last Verified

August 1, 2010

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • kongdx
  • No. 30670903 (Other Identifier: National Natural Science Foundation of China)

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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