Treatment Protocol of Child SAA With the Injection of Mesenchymal Stem Cells（Umbilical Cord Derived） (MSC-SAA)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02218437|
Recruitment Status : Unknown
Verified August 2014 by Xiaofan Zhu, Chinese Academy of Medical Sciences.
Recruitment status was: Recruiting
First Posted : August 18, 2014
Last Update Posted : August 18, 2014
|Condition or disease||Intervention/treatment||Phase|
|Complications of Organ Transplant Stem Cells Umbilical Cord||Drug: MSC+ATG||Phase 4|
- Mesenchymal Stem Cells were prepared by in vitro separation, screening, and culture from healthy human umbilical cord tissue; The " injection of mesenchymal stem cells (umbilical) manufacturing and verification regulation " was also formulated.
- The starting dose of Umbilical Cord Derived MSC was 0.5-1.0 * 106 cells /kg, based on the previous human studies; And the maximum tolerated dose was 1 * 107 cells /kg
- The response and complete remission rate, relapse rate of the injection of Umbilical Cord Derived Mesenchymal Stem Cells (or combined with ATG ) for Child with SAA were determined.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Child With Severe Aplastic Anemia (SAA) Therapy: the Injection of Umbilical Cord Derived Mesenchymal Stem Cells.|
|Study Start Date :||October 2013|
|Estimated Primary Completion Date :||October 2015|
|Estimated Study Completion Date :||October 2015|
The first agent MSC injection, began two weeks after ATG application; Each patient was injected three times, one time per week; Study on single dose tolerance and efficacy index; Each subjects received three dose groups of treatment.
MSC+ATG:Starting dose was 0.5-1.0 × 106 cells /kg; And the maximum tolerated dose was 1 ×107 cells /kg after ATG application.
Other Name: Mesenchymal Stem Cells (Umbilical Cord Derived)
- The response and complete remission rate with different doses of Umbilical Cord Derived MSC to treat child with SAA [ Time Frame: 1 year ]Complete response (CR) was defined as achieving normal levels of hemoglobin adjusted for age, platelet count >100*109/L, and absolute neutrophil count (ANC) >1.5*1.0 9/L. Partial response (PR) was defined as injection independence, reticulocyte count>30*109/L, platelet count >20*109/L, and ANC>0.5*1.0 9/L above the baseline. Persistence of injection requirement or death was evidence of no response (NR).
- The relapse rate with different doses of MSC to treat child with SAA [ Time Frame: 3-10 year ]The relapse was defined as injection dependence again; or progressed or paroxysmal nocturnal hemoglobinuria (PNH)/acute myeloid leukemia/myelodysplasia syndrome(MDS); or cyclosporin A (CsA) dependence.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02218437
|Contact: Xiaofan Zhu, MDfirstname.lastname@example.org|
|Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences and Peking Union Medical College||Recruiting|
|Tianjin, China, 300020|
|Contact: Xiaofan Zhu, MD +86-22-23909001 email@example.com|
|Principal Investigator: Xiaofan Zhu, MD|
|Principal Investigator:||Xiaofan Zhu, MD||Chinese Academy of Medical Sciences|