Does anyone know a phase 2 trial that have used a superiority margin? Particularly regarding dose finding phase 2 trials, this doesn't seem a common approach (i.é., define a superiority margin) to sample size estimation and/or data analysis.
Recently some authors started discussions about phase 2 non-inferiority trials, which implicitly demand a definition of a non-inferiority margin (e.g.: Jung SH, 2017). But non-inferiority trials are rare. Thus, discussion regarding superiority margin in phase 2 trials are not common.
Maybe because for superiority trials, "effect size" rather than "superiority margin" is commonly used.They mean the same.
Dear
The second step in testing an experimental drug (or other treatment) in humans. Typically, Phase 2 trials are done only if Phase 1 trials have shown that the drug is safe, but sometimes Phase 1 and Phase 2 trials are combined. Phase 2 trials are designed to evaluate the drug's effectiveness in people with the disease or condition being studied and to determine the common short-term adverse effects and risks associated with the drug. Phase 2 trials involve more participants (often several hundred) and typically last longer than Phase 1 trials.
Regards
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