Eltrombopag With Standard Immunosuppression 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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT01623167
Recruitment Status : Recruiting
First Posted : June 19, 2012
Last Update Posted : July 6, 2018
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )

Brief Summary:


  • Severe aplastic anemia is a rare and serious blood disorder. It happens when the immune system starts to attack the bone marrow cells. This causes the bone marrow to stop making red blood cells, platelets, and white blood cells. Standard treatment for this disease is horse-ATG and cyclosporine, which suppress the immune system and stop it from attacking the bone marrow. However, this treatment does not work in all people. Some people still have poor blood cell counts even after treatment.
  • Eltrombopag is a drug designed to mimic a protein in the body called thrombopoietin. It helps the body to make more platelets. It may also cause the body to make more red and white blood cells. Studies have shown that eltrombopag may be useful when added to standard treatment for severe aplastic anemia. It may help improve poor blood cell counts.


- To test the safety and effectiveness of adding eltrombopag to standard immunosuppressive therapy for severe aplastic anemia.


- Individuals at least 2 years of age who have severe aplastic anemia that has not yet been treated.


  • Participants will be screened with a physical exam, medical history, and blood tests. Blood and urine samples will be collected.
  • Participants will start treatment with horse-ATG and cyclosporine. Treatment will be given according to the standard of care for the disease.
  • Cohort 1: After 14 days, participants will start taking eltrombopag. They will take eltrombopag for up to 6 months.
  • Cohort 2: After 14 days, participants will start taking eltrombopag. They will take eltrombopag for up to 3 months.
  • Cohort 3 and Extension Cohort: Participants will start taking eltrombopag on Day 1. They will take eltrombopag for up to 6 months.
  • Participants may receive other medications to prevent infections during treatment.
  • Treatment will be monitored with frequent blood tests. Participants will also fill out questionnaires about their symptoms and their quality of life.

Condition or disease Intervention/treatment Phase
Aplastic Anemia Neutropenia Pancytopenia Anemia Thrombocytopenia Drug: Eltrombopag Drug: Horse Anti-Thymocyte Globulin (ATG) Drug: Cyclosporine A (CSA) Phase 1 Phase 2

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 182 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Eltrombopag Added to Standard Immunosuppression in Treatment-Naive Severe Aplastic Anemia
Study Start Date : June 12, 2012
Estimated Primary Completion Date : January 30, 2019
Estimated Study Completion Date : January 30, 2024

Primary Outcome Measures :
  1. The primary endpoint will be the rate of complete hematologic response at six months.

Secondary Outcome Measures :
  1. Secondary endpoints are relapse, robust hematologic blood count recovery at 3, 6, and 12 months, survival, clonal evolution to myelodysplasia and leukemia, and marrow stem cell content.

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  1. Severe aplastic anemia characterized by Bone marrow cellularity less than 30 percent (excluding lymphocytes)


    At least two of the following:

    • Absolute neutrophil count less than 500/microL
    • Platelet count less than 20,000/microL

    Absolute reticulocyte count less than 60,000/microL

  2. Age greater than or equal to 2 years old
  3. Weight greater than 12 kg


  1. Known diagnosis of Fanconi anemia
  2. Evidence of a clonal disorder on cytogenetics performed within 12 weeks of study entry. Patients with super severe neutropenia (ANC less than 200 /microL) will not be excluded initially if cytogenetics are not available or pending. If evidence of a clonal disorder consistent with myelodysplasia is later identified, the patient will go off study.
  3. Prior immunosuppressive therapy with any ATG, alemtuzumab, or high dose cyclophosphamide
  4. SGOT or SGPT >5 times the upper limit of normal
  5. Subjects with known liver cirrhosis in severity that would preclude tolerability of cyclosporine and eltrombopag as evidenced by albumin < 35g/L
  6. Hypersensitivity to eltrombopag or its components
  7. Infection not adequately responding to appropriate therapy
  8. 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
  9. Potential subjects with cancer who are on active chemotherapeutic treatment or who take drugs with hematological effects will not be eligible
  10. Current pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from pregnancy if of childbearing potential during the course of this study
  11. Inability to understand the investigational nature of the study or to give informed consent or does not have a legally authorized representative or surrogate that can provide informed consent.

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): NCT01623167

Contact: Olga J Rios, R.N. (301) 496-4462
Contact: Neal S Young, M.D. (301) 496-5093

United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike Recruiting
Bethesda, Maryland, United States, 20892
Contact: For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)    800-411-1222 ext TTY8664111010   
Sponsors and Collaborators
National Heart, Lung, and Blood Institute (NHLBI)
Principal Investigator: Neal S Young, M.D. National Heart, Lung, and Blood Institute (NHLBI)

Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: National Heart, Lung, and Blood Institute (NHLBI) Identifier: NCT01623167     History of Changes
Other Study ID Numbers: 120150
First Posted: June 19, 2012    Key Record Dates
Last Update Posted: July 6, 2018
Last Verified: May 22, 2018

Studies a U.S. FDA-regulated Drug Product: Yes

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
Blood Platelet Disorders
Leukocyte Disorders
Bone Marrow Diseases
Antilymphocyte Serum
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antifungal Agents
Anti-Infective Agents
Dermatologic Agents
Antirheumatic Agents
Calcineurin Inhibitors