We are updating the design of this site. Learn more.
Show more
ClinicalTrials.gov
ClinicalTrials.gov Menu

Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01703169
First Posted: October 10, 2012
Last Update Posted: October 19, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
Novartis
Information provided by (Responsible Party):
University of Utah
Results First Submitted: June 16, 2017  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: Severe Aplastic Anemia
Very Severe Aplastic Anemia
Moderate Aplastic Anemia
Intervention: Drug: Eltrombopag

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Eltrombopag

Single arm study. Dose Escalation.

Eltrombopag: Oral eltrombopag 150mg/day by mouth starting on Day 1 with dose modification over 12 weeks to a maximum of 300mg/day determined by platelet count


Participant Flow:   Overall Study
    Eltrombopag
STARTED   13 
COMPLETED   13 
NOT COMPLETED   0 



  Baseline Characteristics
  Hide Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Eltrombopag

Single arm study. Dose Escalation.

Eltrombopag: Oral eltrombopag 150mg/day by mouth starting on Day 1 with dose modification over 12 weeks to a maximum of 300mg/day determined by platelet count


Baseline Measures
   Eltrombopag 
Overall Participants Analyzed 
[Units: Participants]
 13 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      0   0.0% 
Between 18 and 65 years      8  61.5% 
>=65 years      5  38.5% 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      5  38.5% 
Male      8  61.5% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
 
American Indian or Alaska Native      0   0.0% 
Asian      1   7.7% 
Native Hawaiian or Other Pacific Islander      0   0.0% 
Black or African American      0   0.0% 
White      10  76.9% 
More than one race      0   0.0% 
Unknown or Not Reported      2  15.4% 
Region of Enrollment 
[Units: Count of Participants]
 
United States   13 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Proportion of Participants With Platelet Response   [ Time Frame: up to 12 weeks ]

2.  Secondary:   Platelet Count Twice Baseline.   [ Time Frame: Between weeks 1-12. ]

3.  Secondary:   Hematology Labs   [ Time Frame: 12 weeks ]

4.  Secondary:   Number of Patients With AE to Measure Toxicity, Using NCI CTCAE   [ Time Frame: 12 weeks ]

5.  Secondary:   Characterization of the PK Profile of Eltrombopag in Patients With Moderate to Very Severe Aplastic Anemia. Evaluated With AUC, Cmax, Cmin, Tmax.   [ Time Frame: Weeks 2, 6 and 12 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Certain data regarding the primary outcome and adverse events were inadvertently not captured during the clinical investigation resulting in insufficient data for statistical analysis and publication.


  More Information
  Hide More Information

Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Kimberlee Taylor
Organization: Huntsman Cancer Institute
phone: 8012135673
e-mail: Kimberlee.Taylor@hci.utah.edu



Responsible Party: University of Utah
ClinicalTrials.gov Identifier: NCT01703169     History of Changes
Other Study ID Numbers: HCI54443
ELT115895 ( Other Identifier: Novartis )
First Submitted: September 27, 2012
First Posted: October 10, 2012
Results First Submitted: June 16, 2017
Results First Posted: October 19, 2017
Last Update Posted: October 19, 2017