NCT01997372

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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
100

participants targeted

Target at P50-P75 for phase_4

Timeline
Completed

Started Dec 2010

Longer than P75 for phase_4

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 1, 2010

Completed
1.2 years until next milestone

First Submitted

Initial submission to the registry

March 1, 2012

Completed
1.7 years until next milestone

First Posted

Study publicly available on registry

November 28, 2013

Completed
3 days until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2013

Completed
1.9 years until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2015

Completed
Last Updated

December 3, 2013

Status Verified

December 1, 2013

Enrollment Period

3 years

First QC Date

March 1, 2012

Last Update Submit

December 2, 2013

Conditions

Keywords

ATGSAAimmunosuppressive treatmentcyclosporine

Outcome Measures

Primary Outcomes (1)

  • 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

Secondary Outcomes (1)

  • the relapse rate with different doses of ATG to treat child severe aplastic anemia

    4-10 years

Study Arms (1)

high dose ATG,low dose ATG

EXPERIMENTAL
Drug: ATG

Interventions

ATGDRUG

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.

high dose ATG,low dose ATG

Eligibility Criteria

Age1 Year - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Department of Pediatrics, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

Related 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.

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

AnemiaHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Failure DisordersBone Marrow Diseases

Central Study Contacts

Study Design

Study Type
interventional
Phase
phase 4
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Department of Pediatrics, Institute of Hematology and Blood Diseases Hospital

Study Record Dates

First Submitted

March 1, 2012

First Posted

November 28, 2013

Study Start

December 1, 2010

Primary Completion

December 1, 2013

Study Completion

November 1, 2015

Last Updated

December 3, 2013

Record last verified: 2013-12

Locations