Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia
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|ClinicalTrials.gov Identifier: NCT01703169|
Recruitment Status : Completed
First Posted : October 10, 2012
Results First Posted : October 19, 2017
Last Update Posted : October 19, 2017
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|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.
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|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Able to provide written informed consent and any other authorizations required by local law (e.g., Protected Health Information [PHI])
- Have severe or very severe aplastic anemia, or moderate aplastic anemia with platelet counts that have dropped below 20,000/μl
- Have moderate, severe, or very severe aplastic anemia with moderate bleeding during or after a surgical procedure, (including bone marrow biopsy, lumbar puncture, thoracentesis, paracentesis, port placement, dermal biopsy) or minimal mucocutaneous bleeding otherwise noted
- Subjects with current or previous exposure to approved medications for the treatment of aplastic anemia will not be excluded; these include but may not be limited to, anti-thymocyte globulin (ATG), cyclosporine, corticosteroids, and G-CSF.
- Have diagnosis of Fanconi anemia
- Have infection not adequately responding to appropriate therapy
- Have Paroxysmal Nocturnal Hemoglobinuria (PNH) clone size in neutrophils of greater than or equal to 50%
- Have known HIV positivity
- Have creatinine and/or blood urea nitrogen (BUN) ≥2 times the upper limit of normal
- Have serum bilirubin ≥ 1.5 times the upper limit of normal, or ≥4.0 times the upper limit of normal if the patient has been treated with ATG within three weeks of screening.
- Have AST and/or ALT ≥ 3 times the upper limit of normal
- Have hypersensitivity to eltrombopag or its components
- Have chemotherapy given less than or equal to 14 days prior to initiating the study medication. This does not include immunosuppressive agents and growth factor as described above
- Are female and are nursing or pregnant or are unwilling to take oral contraceptives or refrain from pregnancy if of childbearing potential
- Are unable to understand the investigational nature of the study or give informed consent
- Have a history of arterial or venous thrombosis within the last 1 year (excluding those due to indwelling lines)
- Have an ECOG performance status of 3 or greater
- Have had treatment with Campath within 6 months of entry into the study
- Have pre-existing cardiovascular disease (congestive heart failure with New York Heart Association [NYHA] grade III/IV), arrhythmia known to increase the risk of thromboembolic events (e.g. atrial fibrillation), unstable angina, or QTc > 450 msec (QTc 480 msec for subjects with bundle branch block), or myocardial infarction within the preceding 6 months) at study entry
- Have had other TPO-R agonists medication in the previous 4 weeks.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
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|
|Responsible Party:||University of Utah|
|Other Study ID Numbers:||
ELT115895 ( Other Identifier: Novartis )
|First Posted:||October 10, 2012 Key Record Dates|
|Results First Posted:||October 19, 2017|
|Last Update Posted:||October 19, 2017|
|Last Verified:||September 2017|
severe aplastic anemia
very severe aplastic anemia
moderate aplastic anemia
Bone Marrow Failure Disorders
Bone Marrow Diseases