A Trial Postmortem: Challenges in Conducting a Randomized, Double-blind, Phase 2 Study in Men with Castration-resistant Prostate Cancer

摘要

Randomized clinical trials are the benchmark for definitive phase 3 trials and are becoming increasingly critical in earlier phase, proof-ti'Pconcept trials in oncology, especially urologic oncology. Unfortunately, many of these trials are never completed or accrue extremely slowly, leading to tremendous waste of scientific, patient, and financial resources. The potential reasons for these failures are multiple, but are often difficult to discern in an individual trial and rarely analyzed objectively. A recent experience with a multicenter, randomized, phase 2 trial for which there was a compelling biologic premise, review and support from the US Food and Administration (FDA), good advertising, and acceptance from co-investigators but that still failed to successfully accrue provides an opportunity to examine potential causes for trial failure more closely.