Status:
COMPLETED
A Pilot Study of a Thrombopoietin-Receptor Agonist, Eltrombopag, in Patients With Low to Int-2 Risk Myelodysplastic Syndrome (MDS)
Lead Sponsor:
National Heart, Lung, and Blood Institute (NHLBI)
Conditions:
Myelodysplastic Syndromes
Thrombocytopenia
Eligibility:
All Genders
18-100 years
Phase:
PHASE2
Brief Summary
Background: * Myelodysplastic syndromes (MDS) are bone marrow disorders characterized by anemia, neutropenia, and thrombocytopenia (low red blood cell, white blood cell, and platelet counts). Patient...
Detailed Description
The myelodysplastic syndromes (MDS) are bone marrow disorders characterized by anemia, neutropenia, and thrombocytopenia. Patients with MDS are at risk for symptomatic anemia, infection, and bleeding,...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA:
- 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
- Treatment 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
- EXCLUSION CRITERIA:
- 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
Exclusion
Key Trial Info
Start Date :
March 15 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 30 2021
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT00961064
Start Date
March 15 2011
End Date
December 30 2021
Last Update
November 14 2023
Active Locations (1)
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1
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892