A Pilot Study of a Thrombopoietin-Receptor Agonist, Eltrombopag, in Patients With Low to Int-2 Risk Myelodysplastic Syndrome (MDS)
This study is currently recruiting participants.
Verified April 2013 by National Institutes of Health Clinical Center (CC)
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
First received: August 15, 2009
Last updated: March 22, 2014
Last verified: April 2013
- 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.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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)|
Resource links provided by NLM:
U.S. FDA Resources
Further study details as provided by National Institutes of Health Clinical Center (CC):
Primary Outcome Measures:
- 20,000/micro L increase in platelets above baseline and the toxicity profile at 3 months. [ Time Frame: 3 months after first dose medication ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- 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& amp; D System)
|Study Start Date:||July 2009|
|Estimated Study Completion Date:||June 2015|
|Estimated Primary Completion Date:||June 2015 (Final data collection date for primary outcome measure)|
Intervention Details:Show Detailed Description
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00961064
|Contact: Barbara Weinstein, R.N.||(301) firstname.lastname@example.org|
|Contact: Danielle M Townsley, M.D.||(301) email@example.com|
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike||Recruiting|
|Bethesda, Maryland, United States, 20892|
|Contact: Barbara Weinstein 301-594-4180 firstname.lastname@example.org|
Sponsors and Collaborators
|Principal Investigator:||Danielle M Townsley, M.D.||National Heart, Lung, and Blood Institute (NHLBI)|