Different Doses of Anti-thymocyte Globin to Treat Child Severe Aplastic Anemia
Primary Purpose
Severe Aplastic Anemia
Status
Unknown status
Phase
Phase 4
Locations
China
Study Type
Interventional
Intervention
ATG
Sponsored by
About this trial
This is an interventional treatment trial for Severe Aplastic Anemia focused on measuring ATG, SAA, immunosuppressive treatment, cyclosporine
Eligibility Criteria
Inclusion Criteria:
- clinical diagnosis of childhood acquired severe aplastic anemia(SAA)
Exclusion Criteria:
- clinical diagnosis of no childhood acquired severe aplastic anemia(SAA)
Sites / Locations
- Department of Pediatrics, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical SciencesRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
high dose ATG,low dose ATG
Arm Description
Outcomes
Primary Outcome Measures
the response and complete remission rate with different doses of ATG to treat child severe aplastic anemia
Complete response (CR) was defined as achieving normal levels of hemoglobin adjusted for age, platelet count >100×109/L, and ANC>1.5×109/L. Partial response (PR) was defined as transfusion independence, reticulocyte count >30×109/L, platelet count >20×109/L, and ANC >0.5×109/L above the baseline. Persistence of transfusion requirement or death was evidence of no response (NR).
Secondary Outcome Measures
the relapse rate with different doses of ATG to treat child severe aplastic anemia
relapse was defined as transfusion dependence again; or progressed to paroxysmal nocturnal hemoglobinuria (PNH) /acute myeloid leukemia/myelodysplasia syndrome (MDS); or CSA dependence
Full Information
NCT ID
NCT01997372
First Posted
March 1, 2012
Last Updated
December 2, 2013
Sponsor
Chinese Academy of Medical Sciences
1. Study Identification
Unique Protocol Identification Number
NCT01997372
Brief Title
Different Doses of Anti-thymocyte Globin to Treat Child Severe Aplastic Anemia
Official Title
Different Doses of Anti-thymocyte Globin With 2.5 or 3.75mg/kg to Treat Child Severe Aplastic Anemia
Study Type
Interventional
2. Study Status
Record Verification Date
December 2013
Overall Recruitment Status
Unknown status
Study Start Date
December 2010 (undefined)
Primary Completion Date
December 2013 (Anticipated)
Study Completion Date
November 2015 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Chinese Academy of Medical Sciences
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
Severe acquired aplastic anaemia (SAA) is a life-threatening disease characterized by pancytopenia and hypoplastic bone marrow. Immunosuppressive treatment with antithymocyte globulin (ATG)and cyclosporine remain the standard regimen with response rates of 70% or more and excellent overall survival. However ,there are no clinical trials to illustrate the response and complete remission rate with different doses of ATG.And there are no data reported on children with SAA so far.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Severe Aplastic Anemia
Keywords
ATG, SAA, immunosuppressive treatment, cyclosporine
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
100 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
high dose ATG,low dose ATG
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
ATG
Intervention Description
Drug ATG:2.5mg/kg/d or 3.75mg/kg/d for 5 days; Drug Cyclosporine A (CSA):3-10mg/kg/d ,Adjust the dose to maintain drug levels between 150 and 300ng/ml; Drug prednisone:1mg/kg/d,d1-21 from the first dosage of ATG; Drug Granulocyte Colony-Stimulating Factor(G-CSF):5ug/kg/d until absolute neutrophil count (ANC) >1×109/L.
Primary Outcome Measure Information:
Title
the response and complete remission rate with different doses of ATG to treat child severe aplastic anemia
Description
Complete response (CR) was defined as achieving normal levels of hemoglobin adjusted for age, platelet count >100×109/L, and ANC>1.5×109/L. Partial response (PR) was defined as transfusion independence, reticulocyte count >30×109/L, platelet count >20×109/L, and ANC >0.5×109/L above the baseline. Persistence of transfusion requirement or death was evidence of no response (NR).
Time Frame
1 years
Secondary Outcome Measure Information:
Title
the relapse rate with different doses of ATG to treat child severe aplastic anemia
Description
relapse was defined as transfusion dependence again; or progressed to paroxysmal nocturnal hemoglobinuria (PNH) /acute myeloid leukemia/myelodysplasia syndrome (MDS); or CSA dependence
Time Frame
4-10 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
clinical diagnosis of childhood acquired severe aplastic anemia(SAA)
Exclusion Criteria:
clinical diagnosis of no childhood acquired severe aplastic anemia(SAA)
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Xiaofan Zhu, MD
Phone
+86 22 23909001
Email
zhuxiaof@yahoo.com.cn
Facility Information:
Facility Name
Department of Pediatrics, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences
City
Tianjin
State/Province
Tianjin
ZIP/Postal Code
300020
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Xiaofan Zhu, MD
Phone
+86 22 23909001
Email
zhuxiaof@yahoo.com.cn
12. IPD Sharing Statement
Citations:
PubMed Identifier
1090310
Citation
Camitta BM, Rappeport JM, Parkman R, Nathan DG. Selection of patients for bone marrow transplantation in severe aplastic anemia. Blood. 1975 Mar;45(3):355-63.
Results Reference
result
Learn more about this trial
Different Doses of Anti-thymocyte Globin to Treat Child Severe Aplastic Anemia
We'll reach out to this number within 24 hrs