Different Doses of Anti-thymocyte Globin to Treat Child Severe Aplastic Anemia

Severe Aplastic Anemia Clinical Trial

Official title:

Different Doses of Anti-thymocyte Globin With 2.5 or 3.75mg/kg to Treat Child Severe Aplastic Anemia

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01997372
• Other study ID #: Xzhu
• Status: Recruiting
• Phase: Phase 4
• First received: March 1, 2012
• Last updated: December 2, 2013
• Start date: December 2010
• Est. completion date: November 2015

Study information

• Verified date: December 2013
• Source: Chinese Academy of Medical Sciences
• Contact: Xiaofan Zhu, MD
• Phone: +86 22 23909001
• Email: zhuxiaof[@]yahoo.com.cn
• Is FDA regulated: No
• Health authority: China: Ministry of Health
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 100
• Est. completion date: November 2015
• Est. primary completion date: December 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 1 Year to 18 Years

Eligibility

Inclusion Criteria:

• clinical diagnosis of childhood acquired severe aplastic anemia(SAA)

Exclusion Criteria:

• clinical diagnosis of no childhood acquired severe aplastic anemia(SAA)

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Severe Aplastic Anemia

Intervention

Drug:
• ATG
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.

Locations

Country	Name	City	State
China	Department of Pediatrics, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences	Tianjin	Tianjin

Sponsors (1)

Lead Sponsor	Collaborator
Chinese Academy of Medical Sciences

Country where clinical trial is conducted

China, 

References & Publications (1)

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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).	1 years	No
Secondary	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	4-10 years	No