Treatment Protocol of Child SAA With the Injection of Mesenchymal Stem Cells（Umbilical Cord Derived） (MSC-SAA)
|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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02218437
|Contact: Xiaofan Zhu, MDemail@example.com|
|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 firstname.lastname@example.org|
|Principal Investigator: Xiaofan Zhu, MD|
|Principal Investigator:||Xiaofan Zhu, MD||Chinese Academy of Medical Sciences|