Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia
|ClinicalTrials.gov Identifier: NCT01703169|
Recruitment Status : Completed
First Posted : October 10, 2012
Results First Posted : October 19, 2017
Last Update Posted : October 19, 2017
|Condition or disease||Intervention/treatment||Phase|
|Severe Aplastic Anemia Very Severe Aplastic Anemia Moderate Aplastic Anemia||Drug: Eltrombopag||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||13 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia|
|Study Start Date :||November 2012|
|Actual Primary Completion Date :||June 2016|
|Actual Study Completion Date :||June 2016|
Single arm study. Dose Escalation.
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
- Proportion of Participants With Platelet Response [ Time Frame: up to 12 weeks ]Defined as a stable platelet count of 50,000/μl or more during any 4 week period within the possible 12 weeks while on study,and including maximal platelet counts achieved in patients with moderate to very severe aplastic anemia.
- Platelet Count Twice Baseline. [ Time Frame: Between weeks 1-12. ]Proportion of subjects who achieve platelet counts at least twice their baseline value at any point while on study medication, in patients with moderate to very severe aplastic anemia.
- Hematology Labs [ Time Frame: 12 weeks ]Association between eltrombopag use and response in hemoglobin, hematocrit, total white blood cell count, and absolute neutrophil count to be evaluate by maximal hemoglobin, hematocrit, total white blood cell count, and absolute neutrophil counts achieved in patients with moderate to very severe aplastic anemia
- Number of Patients With AE to Measure Toxicity, Using NCI CTCAE [ Time Frame: 12 weeks ]Evaluated weekly, up to 12 weeks. Association between eltrombopag use, dose, and tolerability in patients with moderate to very severe aplastic anemia
- 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 ]Samples will for PK analysis will collected as a trough level weeks 2, 6 and 12, prior to dose of eltrombopag. Additional PK level drawn at 2, 4 and 6 hours post-dose at the scheduled week 2 visit.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01703169
|United States, Utah|
|University of Utah|
|Salt Lake City, Utah, United States, 84112|
|Principal Investigator:||George M Rodgers, M.D.||University of Utah|