Eltrombopag in Myelodysplastic Syndrome (MDS) Patients With Thrombocytopenia
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.
Myelodysplastic Syndrome (MDS)
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||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|
- Maximum Tolerated Dose (MTD) [ Time Frame: 24 months ] [ Designated as safety issue: No ]Determine the maximum tolerated dose (MTD) of eltrombopag for the treatment of thrombocytopenia in participants with MDS who have either progressed or are resistant to Hypomethylating Agents (HMTA). The MTD is defined as the highest dose where less than 33% of participants experience a drug related predefined dose limited toxicity (DLT).
- Number of Participants with Complete or Major Platelet Response [ Time Frame: 24 months ] [ Designated as safety issue: No ]Proportion of participant achieving a complete or major platelet response utilizing International Working Group (IWG) 2006 response criteria and duration of platelets response.
- Number of Participants Achieving a Platelets Transfusion Response [ Time Frame: 24 months ] [ Designated as safety issue: No ]Proportion of participants achieving a platelets transfusion response.
- Number and Severity of Bleeding Events [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]The incidence and severity of bleeding events, measured using the World Health Organization (WHO) Bleeding Scale, during the treatment and 4 week follow-up periods for eltrombopag.
- Number of Participants with Overall Survival(OS) [ Time Frame: 24 Months ] [ Designated as safety issue: No ]To evaluate the effect of eltrombopag on overall survival (OS).
|Study Start Date:||March 2011|
|Estimated Study Completion Date:||May 2014|
|Estimated Primary Completion Date:||May 2014 (Final data collection date for primary outcome measure)|
Experimental: Eltrombopag Treatment
Phase I: Dose Escalation. Phase II: Treatment at Maximum Tolerated Dose (MTD)
In Stage 1 the best dose of eltrombopag will be found to take into Stage 2, which will treat 15 participants at this dose.
Other Name: Promacta®
Study drug will be administered on an outpatient basis. The study is divided into two parts: in Stage 1 the best dose of eltrombopag will be found to take forward to Stage 2. In Stage 1 of the study, small groups of participants will be enrolled in steps. The first group will be given a dose of 50 mg per day of study drug for 8 weeks. If these participants have few or manageable side effects, the next group of participants enrolled will receive a higher dose of study drug (100 mg per day for 8 weeks). This increase in doses with groups of participants will continue until the study doctor finds the highest dose of study drug that can be given without causing severe or unmanageable side effects up to a 300 mg daily dose. Stage 2 will include 15 participants at the best dose based on the Stage 1 part of the study. The participant's dose may be adjusted to a lower or higher dose based upon their platelet count, which will be determined by the study doctor. Eltrombopag will be supplied by a company called GlaxoSmithKline as white to off-white, round film coated tablets containing eltrombopag equivalent to 12.5mg, 25mg, 50 mg, 75 mg and 100 mg of eltrombopag.
|United States, Florida|
|H. Lee Moffitt Cancer Center and Research Institute||Recruiting|
|Tampa, Florida, United States, 33612|
|Contact: Debra Mimo 813-745-7362 firstname.lastname@example.org|
|Principal Investigator: Rami Komrokji, M.D.|
|Sub-Investigator: Celeste Bello, M.D.|
|Sub-Investigator: Jeffrey Lancet, M.D.|
|Sub-Investigator: Alan List, M.D.|
|Sub-Investigator: Eric Padron, M.D.|
|Principal Investigator:||Rami Komrokji, M.D.||H. Lee Moffitt Cancer Center and Research Institute|