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Study Design Summary

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For a Phase 2 randomized controlled trial in relapsed/refractory multiple myeloma, a parallel-group design with 1:1 randomization is recommended 1. The primary endpoint should be overall response rate (ORR) per IMWG criteria 2, with progression-free survival (PFS) as a key secondary endpoint P1. Based on completed trials with similar patient populations CT1, a sample size of 120 patients (60 per arm) provides 80% power to detect a clinically meaningful difference P2.

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For a Phase 2 randomized controlled trial in relapsed/refractory multiple myeloma, a parallel-group design with 1:1 randomization is recommended per ICH E9(R1) statistical principles 1 2. The primary endpoint should be overall response rate (ORR) per IMWG criteria, with progression-free survival (PFS) as a key secondary endpoint P1. Analysis of completed Phase 2 myeloma trials shows median enrollment of 60-80 patients per arm CT1, supporting a sample size of 120 patients total for adequate statistical power P2.

E9(R1) Statistical Principles for Clinical Trials: Addendum on Estimands

FDA GuidancePage 2294% match

“The primary estimand should clearly define the treatment effect of interest, including the population, variable, and handling of intercurrent events...”

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