Treatment Protocol of Child SAA With the Injection of Mesenchymal Stem Cells（Umbilical Cord Derived）

Phase 4

• Conditions: Complications of Organ Transplant Stem Cells Umbilical Cord
• Interventions: Drug: MSC+ATG

• Registration Number: NCT02218437
• Lead Sponsor: Chinese Academy of Medical Sciences

Brief Summary

Mesenchymal Stem Cells (MSC) is a non Hematopoietic Stem Cells (HSC) in adult bone marrow and takes part in the bone marrow microenvironment. The response rate of early treatment on Children's SAA application combined with anti-thymocyte globulin (ATG) (40-50 days after ATG treatment) is associated with long-term effect. The injection of Umbilical Cord Derived Mesenchymal Stem Cells combined with ATG improves the efficacy of children with SAA.

Detailed Description

1. 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.

2. 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

3. 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 Timeline

• Study Start: 2013-10
• Status Verified: 2014-08
• Study First Submitted: 2014-08-06
• Study First Submitted QC: 2014-08-14
• Last Update Submitted: 2014-08-14
• Study First Posted: 2014-08-18
• Last Update Posted: 2014-08-18
• Primary Completion: 2015-10
• Study Completion: 2015-10

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

• Non-Severe Aplastic Anemia (NSAA)atients

Exclusion Criteria

• Severe Aplastic Anemia (SAA) patients

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
MSC+ATG	MSC+ATG	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.

Primary Outcome Measures

Name	Time	Method
The response and complete remission rate with different doses of Umbilical Cord Derived MSC to treat child with SAA	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).

Secondary Outcome Measures

Name	Time	Method
The relapse rate with different doses of MSC to treat child with SAA	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.

Trial Locations

Locations (1)

Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences and Peking Union Medical College; 🇨🇳 Tianjin, China