Mixed response and time-to-event endpoints for multistage single-arm phase II design

Table 2 Simulation results of two-stage design for testing H₀: P ≤ P₀ & T^_med ≤ T₀ vs. H₁: P > P₁ or T^_med > T₁ at the nominal level α = 0.05 and 1-β = 0.80^a

Null		Alternative		Sample size^b		Corr^c
P₀	T₀	P₁	T₁	n₁	n	ρ	α	1-β	PET^d	EN₀^e
0.05	3.0	0.20	4.5	15	30	0.8	0.065	0.801	0.786	18.210
						0.5	0.065	0.798	0.820	17.700
						0.2	0.069	0.801	0.853	17.205
						0.0	0.055	0.797	0.893	16.605
0.10	3.0	0.30	5.0	15	30	0.8	0.051	0.794	0.846	17.310
						0.5	0.052	0.790	0.884	16.740
						0.2	0.038	0.797	0.930	16.050
						0.0	0.026	0.791	0.949	15.765
0.20	4.0	0.40	8.0	15	30	0.8	0.046	0.818	0.861	17.085
						0.5	0.044	0.800	0.907	16.395
						0.2	0.033	0.807	0.945	15.825
						0.0	0.021	0.796	0.966	15.510
0.30	4.0	0.50	8.0	15	30	0.8	0.057	0.807	0.841	17.385
						0.5	0.037	0.795	0.911	16.355
						0.2	0.034	0.796	0.928	16.080
						0.0	0.028	0.802	0.950	15.750
0.05	3.0	0.20	4.5	20	40	0.8	0.049	0.807	0.880	22.400
						0.5	0.053	0.802	0.892	22.160
						0.2	0.044	0.797	0.916	21.680
						0.0	0.036	0.804	0.936	21.280
0.10	4.0	0.30	8.0	20	40	0.8	0.053	0.802	0.931	21.380
						0.5	0.044	0.806	0.930	21.400
						0.2	0.028	0.813	0.957	20.860
						0.0	0.025	0.805	0.969	20.620

^aThe censoring rates for time-to-event endpoint are set as 0.1 for both stopping rules and simulated data
^bn₁ and n denote the sample size in the first stage and the total sample size, respectively
^cρ denotes the correlation between response endpoint and time-to-event endpoint
^dPET denotes the early stopping probability under H₀
^eEN₀ denotes the expected sample size under H₀

ISSN: 1745-6215