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Extended Dosing With Eltrombopag for Severe Aplastic Anemia

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. Identifier: NCT01891994
Recruitment Status : Active, not recruiting
First Posted : July 3, 2013
Results First Posted : December 4, 2018
Last Update Posted : March 10, 2020
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )

Brief Summary:


- 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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 40 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine

Drug Information available for: Eltrombopag

Arm Intervention/treatment
Experimental: Eltrombopag
Administration of eltrombopag at a dose of 150mg/day for 6 months
Drug: Eltrombopag
Oral administration of eltrombopag 150mg/day (75 mg/day for East Asian ancestry) for 6 months
Other Name: Promacta

Primary Outcome Measures :
  1. 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).

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 100 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
  • Previous diagnosis of refractory severe aplastic anemia and following at least one treatment course of immunosuppression with a regimen containing antithymocyte globulin, alemtuzumab or cyclophosphamide.
  • One or more of the following three clinically-significant cytopenias: platelet count less than or equal to 30,000/micro L or platelet-transfusion-dependence (requiring at least 4 platelet transfusions in the 8 weeks prior to study entry); neutrophil count less than 500/micro L; hemoglobin less than 9.0 g/dL or red cell transfusion-dependence (requiring at least 4 units of PRBCs in the eight weeks prior to study entry)
  • Age greater than or equal to 2 years old
  • Weight > 12 kg


  • Infection not adequately responding to appropriate therapy
  • Evidence of a clonal disorder on cytogenetics performed within 12 weeks of study entry.
  • Creatinine > 2.5 mg/dL
  • Direct Bilirubin > 2.0 mg/dL
  • SGOT or SGPT >5 times the upper limit of normal
  • Hypersensitivity to eltrombopag or its components<TAB>
  • Female subjects who are nursing or pregnant or are unwilling to take oral contraceptives or refrain from pregnancy if of childbearing potential
  • Unable to understand the investigational nature of the study or give informed consent
  • Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient s ability to tolerate protocol therapy, or that death within 7-10 days is likely
  • Treatment with ATG, cyclophophamide or alemtuzamab within 6 months of study entry.

Information from the National Library of Medicine

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 identifier (NCT number): NCT01891994

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United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
National Heart, Lung, and Blood Institute (NHLBI)
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Principal Investigator: Cynthia E Dunbar, M.D. National Heart, Lung, and Blood Institute (NHLBI)
  Study Documents (Full-Text)

Documents provided by National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) ):

Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: National Heart, Lung, and Blood Institute (NHLBI) Identifier: NCT01891994    
Other Study ID Numbers: 130133
First Posted: July 3, 2013    Key Record Dates
Results First Posted: December 4, 2018
Last Update Posted: March 10, 2020
Last Verified: January 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) ):
Additional relevant MeSH terms:
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Anemia, Aplastic
Hematologic Diseases
Bone Marrow Diseases