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

Phase 2

• Conditions: Aplastic Anemia
• Interventions: Other: cyclosporin A; Other: Human umbilical cord-derived MSCs and cyclosporin A

• Registration Number: NCT01182662
• Lead Sponsor: Shandong University

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 Timeline

• Status Verified: 2010-08
• Study Start: 2010-08
• Study First Submitted: 2010-08-11
• Study First Submitted QC: 2010-08-16
• Study First Posted: 2010-08-17
• Last Update Submitted: 2010-08-30
• Last Update Posted: 2010-08-31
• Primary Completion: 2013-08
• Study Completion: 2013-08

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 30

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
cyclosporine A	cyclosporin A	cyclosporine A at a dose of 5 mg CsA/kg
Human umbilical cord-derived MSCs and cyclosporin	Human umbilical cord-derived MSCs and cyclosporin A	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

Primary Outcome Measures

Name	Time	Method
SAA clinical symptoms	1 year	Anemia symptoms, bleeding and infection will be mainly observed in every monthly after transplanting MSCs for one year.
The number of blood cells	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
Bone borrow hemocytology	1 year	Bone borrow cytomorphologic examination will be tested in every 3 months after transplantion of MSCs for one year.

Secondary Outcome Measures

Name	Time	Method
Percentage of systemic T regulatory cell population and T lymphocyte subsets	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.

Trial Locations

Locations (1)

Department of Hematology of the 2nd Hospital of Shandong University; 🇨🇳 Jinan, Shandong, China