A Pilot Study of a Thrombopoietin-Receptor Agonist, Eltrombopag, in Patients With Low to Int-2 Risk Myelodysplastic Syndrome (MDS)
|ClinicalTrials.gov Identifier: NCT00961064|
Recruitment Status : Active, not recruiting
First Posted : August 18, 2009
Last Update Posted : February 9, 2018
- 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
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||30 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Estimated Primary Completion Date :||December 30, 2018|
|Estimated Study Completion Date :||December 30, 2021|
- 20,000/micro L increase in platelets above baseline and the toxicity profile at 3 months. [ Time Frame: 3 months after first dose medication ]
- Changes in platelet count (continuous variable), changes in platelet transfusion requirements, incidence of bleeding; changes in serum thrombopoietin level (as measured by enzyme-linked immunosorbent assay, R&D System)
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00961064
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike|
|Bethesda, Maryland, United States, 20892|
|Principal Investigator:||Thomas Winkler, M.D.||National Heart, Lung, and Blood Institute (NHLBI)|