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

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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
Sponsor:
Information provided by (Responsible Party):
Xiaofan Zhu, 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.

Condition or disease Intervention/treatment Phase
Complications of Organ Transplant Stem Cells Umbilical Cord Drug: MSC+ATG Phase 4

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: 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.
Drug: MSC+ATG
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)




Primary Outcome Measures :
  1. 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).


Secondary Outcome Measures :
  1. 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.



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Ages Eligible for Study:   1 Month to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

  • Non-Severe Aplastic Anemia (NSAA)atients

Exclusion Criteria:

  • Severe Aplastic Anemia (SAA) patients

Information from the National Library of Medicine

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


Contacts
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Contact: Xiaofan Zhu, MD +86-22-23909001 zhuxf6465@gmail.com

Locations
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China
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    zhuxf6465@gmail.com   
Principal Investigator: Xiaofan Zhu, MD         
Sponsors and Collaborators
Chinese Academy of Medical Sciences
Investigators
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Principal Investigator: Xiaofan Zhu, MD Chinese Academy of Medical Sciences

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Responsible Party: Xiaofan Zhu, chief physician, Chinese Academy of Medical Sciences
ClinicalTrials.gov Identifier: NCT02218437     History of Changes
Other Study ID Numbers: MSC-2014-SAA-1
H0806-MSC-2014 ( Other Grant/Funding Number: NSFC )
First Posted: August 18, 2014    Key Record Dates
Last Update Posted: August 18, 2014
Last Verified: August 2014
Keywords provided by Xiaofan Zhu, Chinese Academy of Medical Sciences:
Mesenchymal Stem Cells (MSC)
Umbilical Cord
thymocyte globulin (ATG)
Severe Aplastic Anemia (SAA)