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A Pilot Study of a Thrombopoietin-Receptor Agonist, Eltrombopag, in Patients With Low to Int-2 Risk Myelodysplastic Syndrome (MDS)

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: NCT00961064
Recruitment Status : Active, not recruiting
First Posted : August 18, 2009
Last Update Posted : February 15, 2019
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )

Brief Summary:


  • Myelodysplastic syndromes (MDS) are bone marrow disorders characterized by anemia, neutropenia, and thrombocytopenia (low red blood cell, white blood cell, and platelet counts). Patients with MDS are at risk for symptomatic anemia, infection, and bleeding, as well as a risk of progression to acute leukemia. Standard treatments for MDS have significant relapse rates. MDS patients with thrombocytopenia who fail standard therapies require regular, expensive, and inconvenient platelet transfusions, and are at risk for further serious bleeding complications.
  • Eltrombopag is a drug designed to mimic the protein thrombopoietin, which causes the body to make more platelets. Eltrombopag has been able to increase platelet counts in healthy volunteers and in patients with chronic ITP (a disease where patients destroy their own platelets very rapidly and thus develop thrombocytopenia), but researchers do not know if the drug can increase platelet counts in patients with MDS.


  • To find out whether eltrombopag can improve platelet counts in patients with MDS.
  • To determine whether eltrombopag is safe for patients with MDS.


- Patients 18 years of age and older who have consistently low blood platelet counts related to MDS that has not responded to conventional treatment.


  • Treatment with eltrombopag tablets once per day for 90 days.
  • Participants will be monitored closely throughout the initial treatment, with weekly blood tests and separate evaluations at the National Institutes of Health (NIH) treatment center every 4 weeks. Bone marrow biopsies may be conducted to check for abnormalities in bone marrow.
  • If patients show signs of improved platelet counts after 90 days, treatment will continue with additional doses of eltrombopag.
  • Patients who discontinue taking eltrombopag will be evaluated at the NIH treatment center 4 weeks after ending treatment, and again 6 months after ending treatment to check for potential side effects.

Condition or disease Intervention/treatment Phase
Myelodysplastic Syndromes Thrombocytopenia Drug: Eltrombopag Phase 2

  Show Detailed Description

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 30 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Study of a Thrombopoietin-Receptor Agonist (TPO-R Agonist), Eltrombopag, in Patients With Low to Int-2 Risk Myelodysplastic Syndrome (MDS)
Study Start Date : July 24, 2009
Actual Primary Completion Date : August 10, 2017
Estimated Study Completion Date : December 30, 2021

Resource links provided by the National Library of Medicine

Drug Information available for: Eltrombopag

Arm Intervention/treatment
Experimental: 1
Eltrombopag will be initiated at 50 mg/day (Asians 25 mg/day) and dose adjusted up to 150mg/day based response and safety
Drug: Eltrombopag
Eltrombopag will be initiated at 50 mg/day (Asians 25 mg/day) and dose adjusted up to 150mg/day based response and safety

Primary Outcome Measures :
  1. Assess efficacy of eltrombopag in low to Int-2 risk MDS patients with thrombocytopenia refractory to standard therapies for MDS, including hypomethylating agents, lenalidamide, and immunosuppressive therapy. Safety will be assessed concurrently.... [ Time Frame: 16 to 20 weeks ]
    Platelet response, Erythroid response, & Neutrophil response.

Secondary Outcome Measures :
  1. Secondary endpoints will include incidence of grade 2 or higher bleeding events as measured by CTCAE v. 4.0; changes in serum thrombopoietin level, measured at16 to 20 weeks; progression to higher risk MDS as measured by IWG criteria(1); and dis... [ Time Frame: 16 weeks to 5 years ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 110 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Diagnosis of MDS, with WHO classification of refractory anemia, refractory cytopenia with unilineage dysplasia (RCUD), RARS, RCMD-RS, or RCMD.

IPSS risk scores of low, intermediate-1, or intermediate-2.

Platelet count less than or equal to 30,000/ microL or platelet-transfusion-dependence (requiring at least 4 platelet transfusions in the 8 weeks prior to study entry); or hemoglobin less than 9.0 gr/dL or red cell transfusion-dependence (requiring at least 4 units of PRBCs in the eight weeks prior to study entry) OR ANC less than or equal to 500

Age greater than or equal to 18 years old

Teatment naive or off all other treatments for MDS (except stable dosing of filgrastim [G-CSF], erythropoietin, and transfusion support) for at least four weeks. G-CSF can be used before, during and after the protocol treatment for subjects with documented neutropenia (<500/UI) as long as they meet the criteria for other cytopenia as stated above. G-CSF must be held for 3 weeks prior to enrollment bone marrow biopsy and prior to each study assessment bone marrow biopsy, unless clinically indicated to avoid infections per PI discretion.

Adequate liver function, as evidenced by total serum bilirubin less than or equal to 1.5 times the upper limit of normal patients with Gilbert s disease are eligible, provided intermittent indirect hyperbilirubinemia, AST or ALT less than or equal to 5 times the upper limit of normal.

A serum creatinine concentration less than or equal to 2 times ULN


WHO classification of chronic myelomonocytic leukemia (CMML), RAEB-1, RAEB-2, AML

Treatment with horse or rabbit ATG or Campath within 6 months of study entry

Subjects with liver cirrhosis including subjects infected with Hepatitis B or C

Subjects with HIV

Infection not adequately responding to appropriate therapy

History of malignancy treated with chemotherapy and cytogenetic abnormalities suggestive of secondary myelodysplasia.

Moribund status or concurrent hepatic, renal, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient s ability to tolerate protocol therapy

Life expectancy of less than 3 months

Hypersensitivity to eltrombopag or its components

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 or does not have a legally authorized representative or surrogate that can provide informed consent per section

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): NCT00961064

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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: Neal S Young, M.D. 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: NCT00961064     History of Changes
Other Study ID Numbers: 090199
First Posted: August 18, 2009    Key Record Dates
Last Update Posted: February 15, 2019
Last Verified: January 16, 2019

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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) ):
Bone Marrow Fibrosis
Myelodysplastic Syndrome

Additional relevant MeSH terms:
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Myelodysplastic Syndromes
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Blood Platelet Disorders