Issues regarding study design for initial clinical trials using decitabine.

Decitabine offers a reasonable treatment alternative to hydroxyurea for individuals with sickle cell disease who have experienced multiple painful episodes. Despite the promise that the use of decitabine holds, several issues linger with regards to early trial design, such as what age group should be included and what trial design should be used in studies assessing its safety and clinical efficacy. Other issues that must be considered include determination of which primary clinical outcomes should be evaluated, what route of administration should be tested, and to how to assess toxicity of this novel therapeutic agent. This article will discuss issues regarding the factors that must be considered prior to initiating a phase III trial of decitabine for individuals with sickle cell anemia.