Abstract:
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Ever since Mehta & Pocock introduced the Promising Zone Design in 2011, there has been a great deal of interest, hope, and expectations regarding this "less well understood" design strategy. The poster child for this adaptive two-stage design with unblinded sample size re-estimation has been the VALOR study - a phase III trial evaluating the benefit-risk of Vosaroxin + Ara-C versus Ara-C alone in patients with first relapsed or refractory Acute Myeloid Leukemia. In this talk, we review the design benefits, implementation challenges, lessons learned, and opportunities for the future brought by the VALOR experience.
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