Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Severe Aplastic Anemia
|ClinicalTrials.gov Identifier: NCT01182662|
Recruitment Status : Unknown
Verified August 2010 by Shandong University.
Recruitment status was: Recruiting
First Posted : August 17, 2010
Last Update Posted : August 31, 2010
|Condition or disease||Intervention/treatment||Phase|
|Aplastic Anemia||Other: Human umbilical cord-derived MSCs and cyclosporin A Other: cyclosporin A||Phase 2|
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 (Clinical Trial)|
|Estimated Enrollment :||30 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Single (Outcomes Assessor)|
|Official Title:||Phase II Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat SAA|
|Study Start Date :||August 2010|
|Estimated Primary Completion Date :||August 2013|
|Estimated Study Completion Date :||August 2013|
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
- 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.
- 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
- 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.
- 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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01182662
|Contact: chengyun zheng, Ph. Dfirstname.lastname@example.org|
|Department of Hematology of the 2nd Hospital of Shandong University||Recruiting|
|Jinan, Shandong, China, 250033|
|Contact: chengyun zheng, Ph. D +86-531-85875635 email@example.com|
|Principal Investigator:||chengyun zheng, Ph. D||Department of Hematology of The 2nd Hospital of Shandong University|