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

Aplastic Anemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01182662
• Other study ID #: kongdx
• Secondary ID: No. 30670903
• Status: Recruiting
• Phase: Phase 2
• First received: August 11, 2010
• Last updated: August 30, 2010
• Start date: August 2010
• Est. completion date: August 2013

Study information

• Verified date: August 2010
• Source: Shandong University
• Contact: chengyun zheng, Ph. D
• Phone: +86-531-85875635
• Email: chengyun.zheng[@]ki.se
• Is FDA regulated: No
• Health authority: China: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 30
• Est. completion date: August 2013
• Est. primary completion date: August 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 80 Years

Eligibility

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

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Single Blind (Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Aplastic Anemia

Intervention

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
• cyclosporin A
cyclosporin A 5mg/kg po for 12 months

Locations

Country	Name	City	State
China	Department of Hematology of the 2nd Hospital of Shandong University	Jinan	Shandong

Sponsors (2)

Lead Sponsor	Collaborator
Shandong University	National Natural Science Foundation of China

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	SAA clinical symptoms	Anemia symptoms, bleeding and infection will be mainly observed in every monthly after transplanting MSCs for one year.	1 year	Yes
Primary	The number of blood cells	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	Yes
Primary	Bone borrow hemocytology	Bone borrow cytomorphologic examination will be tested in every 3 months after transplantion of MSCs for one year.	1 year	Yes
Secondary	Percentage of systemic T regulatory cell population and T lymphocyte subsets	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	Yes