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Extended Dosing With Eltrombopag for Severe Aplastic Anemia

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) ) Identifier:
First received: June 28, 2013
Last updated: August 4, 2017
Last verified: July 27, 2017


- 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 Intervention Phase
Severe Aplastic Anemia (SAA) Drug: Eltrombopag Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Extended Dosing With Eltrombopag in Refractory Severe Aplastic Anemia

Resource links provided by NLM:

Further study details as provided by National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) ):

Primary Outcome Measures:
  • The proportion of drug responders as defined by changes in the platelet count and/or platelet transfusion requirements, hemoglobin levels, number of red blood cell transfusions, or neutrophil counts as measured by International Working Group cri... [ Time Frame: 6 months ]

Secondary Outcome Measures:
  • Survival [ Time Frame: 6 months to 5 years ]
  • Health-related quality of life [ Time Frame: 0 months to 5 years ]
  • Relapse [ Time Frame: 6 months to 5 years ]
  • Clonal evolution to PNH, clonal chromosomal population in bone marrow, myelodysplasia by morphology, or acute leukemia [ Time Frame: 6 months to 5 years ]
  • Hematological response at 3 & 12 months and yearly thereafter [ Time Frame: 3 months to 5 years ]

Enrollment: 38
Study Start Date: June 17, 2013
Estimated Study Completion Date: May 1, 2020
Estimated Primary Completion Date: May 1, 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Eltrombopa
administration of eltrombopag for 6 months
Drug: Eltrombopag
administration of eltrombopag for 6 months

  Show Detailed Description


Ages Eligible for Study:   2 Years to 100 Years   (Child, Adult, Senior)
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01891994

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)
Principal Investigator: Thomas Winkler, M.D. National Heart, Lung, and Blood Institute (NHLBI)
  More Information

Additional Information:
Responsible Party: National Heart, Lung, and Blood Institute (NHLBI) Identifier: NCT01891994     History of Changes
Other Study ID Numbers: 130133
Study First Received: June 28, 2013
Last Updated: August 4, 2017

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

Additional relevant MeSH terms:
Anemia, Aplastic
Hematologic Diseases
Bone Marrow Diseases processed this record on September 21, 2017