A multi-center, randomized, double-blind, placebo-controlled clinical trial of deferasirox in patients with myelodysplastic syndromes (low/int-1risk) and transfusional iron overload (TELESTO)
The goal of this clinical research study is to study the effectiveness of the drug Exjade® (deferasirox) in controlling MDS. Researchers will also study the effect of the study drug/placebo on the heart, kidneys, and liver.
Treatment Location: N/A
The purpose of this study is to evaluate in low/int-1 risk MDS patients, treated as per standard practice, the clinical benefit of deferasirox versus placebo, while rigorously monitoring relevant clinical parameters (cardiac and liver function and transformation to AML) potentially affected by iron overload complications. The primary objective is to compare deferasirox to placebo with regard to event-free survival (a composite primary endpoint including death and non-fatal events related to cardiac and liver function) in low and int-1 risk MDS patients with transfusional iron overload. Primary objective The primary objective is to evaluate deferasirox and placebo with regard to event-free survival (a composite primary endpoint including death and non-fatal events related to cardiac and liver function and transformation to AML) in low and int-1 risk MDS patients with transfusional iron overload. The secondary objectives are to evaluate: Hematologic improvement (HI) in terms of erythroid response Overall survival Change in endocrine function (thyroid and glycemic control) Disease progression (which includes MDS progression and progression to AML) Change in serum ferritin level Change in cardiac function Frequency of infections requiring intravenous antimicrobials Safety, in particular to assess the levels of increased risk for pre-specified adverse events (renal dysfunction, neutropenia, thrombocytopenia, gastrointestinal bleeding, and laboratory abnormalities) that would be clinically unacceptable in the context of the level of benefit that is likely to be provided by iron chelation using deferasirox in MDS patients with iron overload
IRB Review and Approval Date: 09/02/2010
Recruitment Status: Closed
Projected Accrual: N/A