Eltrombopag in the Management of Low Platelet Counts in MDS and AML
Research type
Research Study
Full title
Study PMA112509, a Phase I/II Study of Eltrombopag in Thrombocytopenic Subjects with Advanced Myelodysplastic Syndrome (MDS) or secondary Acute Myeloid Leukemia after MDS (sAML/MDS)
IRAS ID
68494
Contact name
David Bowen MA MB BChir MRCP MD FRCPath
Eudract number
2009-015512-17
Clinicaltrials.gov Identifier
Research summary
Myelodysplastic Syndrome (MDS) is a condition where one or more of the major components of blood are not being produced by the body in sufficient quantities; too few oxygen-carrying red cells, causing anaemia, resulting in tiredness, too few infection-fighting white cells (includes neutrophils) called neutropenia, leading to infection; and too few platelets (also called thrombocytes) called thrombocytopenia, causing bleeding. There are established treatments, which can increase red cell production and also combat neutropenia, however, there is no universal, satisfactory treatment, for thrombocytopenia. MDS gets significantly worse over time in 45% of subjects. This exacerbated condition is known as sAML/MDS (secondary Acute Myeloid Leukemia following MDS.) In this state, leukaemia-like cells, called blasts increase and further squeeze out healthy cells such that symptoms may further become life threatening. sAML/MDS is a rare condition and subjects with primary AML (not following MDS) will also be admitted to the study. Subjects with sAML/MDS may have their platelets replenished by repeated transfusions but the benefit of each transfusion only lasts for a few days. Eltrombopag, taken orally, at doses of 50-300 mg once daily, increases the rate at which platelets are produced by the body. Taking it might be more effective and convenient for patients, than repeated transfusions. This trial compares a group of subjects treated with standard therapy plus eltrombopag n=40, with a similar group of subjects n=20, treated with standard therapy plus a placebo (a tablet with no active medicine). Subjects will be treated and assessed for a minimum of six months, then followed for a further six or twelve months (possibly whilst continuing treatment). The primary objective will be to assess the safety and tolerability of eltromobopag. This study will be carried out on an international, multicentre basis. Neither subjects or investigators will know, which treatment patients are taking (double-blind treatment).
REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
11/H1306/8
Date of REC Opinion
9 Feb 2011
REC opinion
Favourable Opinion