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Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia

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ClinicalTrials.gov Identifier: NCT01231841
Recruitment Status : Completed
First Posted : November 1, 2010
Results First Posted : March 9, 2012
Last Update Posted : February 22, 2018
Sponsor:
Information provided by (Responsible Party):
The Cleveland Clinic

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Aplastic Anemia
Interventions Drug: cyclosporine
Biological: anti-thymocyte globulin
Enrollment 20
Recruitment Details Patients with severe aplastic anemia were recruited over a period of four years (2005-2009) in the hematology clinic at the Cleveland Clinic, a hospital in Cleveland, Ohio.
Pre-assignment Details  
Arm/Group Title Rabbit Antithymocyte Globulin (r-ATG/Thymoglobulin)
Hide Arm/Group Description Patients received rabbit anti-thymocyte globulin IV daily over 4-24 hours on days 1-5 (3.5 mg/kg/day). Beginning on day 6, patients received oral cyclosporine twice daily (5 mg/kg/day) for a period of 6 months and then tapered.
Period Title: Overall Study
Started 20
Completed 20
Not Completed 0
Arm/Group Title rATG
Hide Arm/Group Description Patients receive anti-thymocyte globulin IV daily over 4-24 hours on days 1-5. Beginning on day 6, patients receive oral cyclosporine twice daily for 6 months followed by a taper. Treatment continues in the absence of disease progression or unacceptable toxicity
Overall Number of Baseline Participants 20
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants
<=18 years
0
   0.0%
Between 18 and 65 years
14
  70.0%
>=65 years
6
  30.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 20 participants
51.95  (18.43)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants
Female
7
  35.0%
Male
13
  65.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 20 participants
20
1.Primary Outcome
Title Patients Treated With Rabbit Antithymocyte Globulin (r-ATG/Thymoglobulin) and Cyclosporine (CsA) Achieving at Least a Partial Remission (PR) at 6 Months
Hide Description Patients will be classified as responders if they have transfusion independence and meet two of the following three criteria: ANC greater than 500/mm3; platelet count greater than 20,000/mm3; and reticulocyte count greater than 40,000/mm3. Transfusion independence is defined as no need for transfusions for one month prior to response assessment.
Time Frame At 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Rabbit Antithymocyte Globulin (r-ATG/Thymoglobulin)
Hide Arm/Group Description:
Patients received rabbit anti-thymocyte globulin IV daily over 4-24 hours on days 1-5 (3.5 mg/kg/day). Beginning on day 6, patients received oral cyclosporine twice daily (5 mg/kg/day) for a period of 6 months and then tapered.
Overall Number of Participants Analyzed 20
Measure Type: Number
Unit of Measure: participants
9
2.Secondary Outcome
Title Comparison of the Level of IS as Assessed by Immuknow Assay in Responders and Non-responders
Hide Description [Not Specified]
Time Frame Every 2 weeks for 3 months beginning on day 1 of therapy and then monthly (for a total of 6 months)
Outcome Measure Data Not Reported
3.Secondary Outcome
Title Reduction of VB Repertoire Associated With r-ATG/CsA Combination
Hide Description We will perform molecular analysis of the TCR repertoire to identify “marker” immunodominant clone specimens using VB typing.
Time Frame Every 4 weeks
Outcome Measure Data Not Reported
Time Frame [Not Specified]
Adverse Event Reporting Description The endpoints of this study evaluated response only. As such, data on non-serious adverse events were not collected or reported for any patients on this trial. Data regarding serious adverse events were collected and reported according to FDA and IRB policies and regulations.
 
Arm/Group Title rATG
Hide Arm/Group Description Patients receive anti-thymocyte globulin IV daily over 4-24 hours on days 1-5. Beginning on day 6, patients receive oral cyclosporine twice daily for 6 months followed by a taper. Treatment continues in the absence of disease progression or unacceptable toxicity
All-Cause Mortality
rATG
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
rATG
Affected / at Risk (%) # Events
Total   11/20 (55.00%)    
Blood and lymphatic system disorders   
febrile neutropenia  3/20 (15.00%)  4
Gastrointestinal disorders   
gastrointestinal bleed  1/20 (5.00%)  1
General disorders   
headache  1/20 (5.00%)  1
Immune system disorders   
serum sickness  2/20 (10.00%)  2
Infections and infestations   
colitis  2/20 (10.00%)  2
pneumonia  1/20 (5.00%)  2
sepsis  4/20 (20.00%)  4
urinary tract infection  1/20 (5.00%)  2
Musculoskeletal and connective tissue disorders   
chest pain  2/20 (10.00%)  2
compression fractures  1/20 (5.00%)  1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
rATG
Affected / at Risk (%) # Events
Total   0/0    
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Jaroslaw Maciejewski, MD PhD, Principal Investigator
Organization: Cleveland Clinic Foundation
Phone: 216-445-5962
Responsible Party: The Cleveland Clinic
ClinicalTrials.gov Identifier: NCT01231841     History of Changes
Other Study ID Numbers: CCF7922
NCI-2010-01365 ( Other Identifier: NCI/CTRP )
First Submitted: October 29, 2010
First Posted: November 1, 2010
Results First Submitted: January 31, 2012
Results First Posted: March 9, 2012
Last Update Posted: February 22, 2018