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

Chinese Academy of Medical Sciences & Peking Union Medical College

Status and phase: Unknown; Phase 4

Conditions: Complications of Organ Transplant Stem Cells Umbilical Cord

Treatments: Drug: MSC+ATG

Study type: Interventional

Funder types: Other

Identifiers

NCT02218437

H0806-MSC-2014 (Other Grant/Funding Number)

MSC-2014-SAA-1

Details and patient eligibility

About

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.

Full 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.

Enrollment: 20 estimated patients

Sex: All

Ages: 1 month to 18 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

• Non-Severe Aplastic Anemia (NSAA)atients

Exclusion criteria

• Severe Aplastic Anemia (SAA) patients

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

MSC+ATG

Experimental group

Description:

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.

Treatment:

Drug: MSC+ATG