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
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.
Interventional
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Maximum Tolerated Dose (MTD)
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).
24 months
No
Rami Komrokji, M.D.
Principal Investigator
H. Lee Moffitt Cancer Center and Research Institute
United States: Food and Drug Administration
MCC-16523
NCT01286038
March 2011
December 2013
Name | Location |
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H. Lee Moffitt Cancer Center and Research Institute | Tampa, Florida 33612 |