Extended Dosing With Eltrombopag for Severe Aplastic Anemia
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|ClinicalTrials.gov Identifier: NCT01891994|
Recruitment Status : Active, not recruiting
First Posted : July 3, 2013
Results First Posted : December 4, 2018
Last Update Posted : March 10, 2020
- Eltrombopag is a drug being tested for treating severe aplastic anemia. It can help improve blood counts in these patients. However, researchers do not know how long the drug can and should be taken for this type of anemia.
- To look at whether 6 months of treatment with eltrombopag can improve patient s blood counts.
- Individuals at least 2 years of age who are taking eltrombopag for severe aplastic anemia.
- Participants will take eltrombopag by mouth once a day for 6 months.
- Blood samples will be collected every 2 weeks for the first 6 months. Bone marrow samples will be collected at 3 and 6 months. These samples will look at the effects of the study drug on the marrow.
- Participants will continue to take the study drug for as long as it is effective and if the side effects are not severe.
|Condition or disease||Intervention/treatment||Phase|
|Severe Aplastic Anemia (SAA)||Drug: Eltrombopag||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||40 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Extended Dosing With Eltrombopag in Refractory Severe Aplastic Anemia|
|Study Start Date :||June 28, 2013|
|Actual Primary Completion Date :||October 16, 2017|
|Estimated Study Completion Date :||December 31, 2021|
Administration of eltrombopag at a dose of 150mg/day for 6 months
Oral administration of eltrombopag 150mg/day (75 mg/day for East Asian ancestry) for 6 months
Other Name: Promacta
- Number of Participants With Drug Response as Defined by Clinically-signficant Hematologic Improvements [ Time Frame: 24 weeks ]Defined as unilineage or multilineage recovery by 1 or more of the following: 1) platelet response (increase to 20 × 103/μL above baseline or stable platelet counts with transfusion independence for a minimum of 8 weeks in those who were transfusion dependent on entry into the protocol); (2) erythroid response (when pretreatment hemoglobin was <9 g/dL, defined as an increase in hemoglobin by 1.5 g/dL or, in transfused patients, a reduction in the units of packed red blood cell transfusions by an absolute number of at least 4 transfusions for 8 consecutive weeks, compared with the pretreatment transfusion number in the previous 8 weeks); and (3) neutrophil response (when pretreatment absolute neutrophil count [ANC] of <0.5 × 103/μL as at least a 100% increase in ANC, or an ANC increase >0.5 × 103/μL, and the toxicity profile as measured using Common Terminology Criteria for Adverse Events).
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): NCT01891994
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike|
|Bethesda, Maryland, United States, 20892|
|Principal Investigator:||Cynthia E Dunbar, M.D.||National Heart, Lung, and Blood Institute (NHLBI)|