NCT01286038

Eltrombopag in Myelodysplastic Syndrome (MDS) Patients With Thrombocytopenia

A Sequential Two-Stage Dose Escalation Study to Evaluate the Safety and Efficacy of Eltrombopag in Myelodysplastic Syndrome (MDS) Patients With Thrombocytopenia Who Progressed or Are Resistant to Hypomethylating Agents

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 37 (actual)
• Sponsor: H. Lee Moffitt Cancer Center and Research Institute · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety and efficacy of eltrombopag in people who have myelodysplastic syndrome (MDS) with thrombocytopenia who have progressed or are resistant to decitabine or azacitidine. (These are the only 2 drugs approved by the U.S. Food and Drug Administration \[FDA\] which can improve platelet counts). The investigators (the study doctor, study staff, and sponsor) want to find out what effects, good or bad, eltrombopag (study drug) may have on people with low platelet counts due to MDS. The investigators will also be testing how well eltrombopag may work at different doses in these diseases.

Detailed description

Study was originally designed as a Phase I/II but was terminated during the Phase I portion due to lack of efficacy

Conditions

• Myelodysplastic Syndrome (MDS)
• Thrombocytopenia

Interventions

Type	Name	Description
DRUG	Eltrombopag	In Stage 1 the best dose of eltrombopag will be found to take into Stage 2, which will treat 15 participants at this dose.

Timeline

• Start date: 2011-04-06
• Primary completion: 2017-11-03
• Completion: 2018-11-03
• First posted: 2011-01-31
• Last updated: 2022-11-07

Locations

1 site across 1 country: United States