A stratified adaptive two-stage design with co-primary endpoints for phase II clinical oncology trials

Cabarrou, Bastien; Leconte, Eve; Sfumato, Patrick; Boher, Jean-Marie; Filleron, Thomas

doi:10.1186/s12874-022-01748-w

BMC Medical Research Methodology

Table 3 Simulation results

From: A stratified adaptive two-stage design with co-primary endpoints for phase II clinical oncology trials

			Parallel BD	SABD
Case study 1
		Maximum sample size	88	67
1A	p_R⁻ = 0.7/p_T⁻ = 0.9 p_R⁺ = 0.7/p_T⁺ = 0.9	Expected sample size	52.8	42.4
		Probability of early termination	0.458	0.416
		Probability of rejecting H₀⁻ or H₀⁺	0.092	0.094
1B	p_R⁻ = 0.9/p_T⁻ = 0.7 p_R⁺ = 0.9/p_T⁺ = 0.7	Expected sample size	52.8	42.4
		Probability of early termination	0.457	0.416
		Probability of rejecting H₀⁻ or H₀⁺	0.092	0.094
2	p_R⁻ = 0.7/p_T⁻ = 0.7 p_R⁺ = 0.9/p_T⁺ = 0.9	Expected sample size	63.4	50.6
		Probability of rejecting H₀⁺	0.906	0.801
		Probability of detecting heterogeneity (1st stage)	0.840	0.739
3	p_R⁻ = 0.9/p_T⁻ = 0.9 p_R⁺ = 0.9/p_T⁺ = 0.9	Expected sample size	85.1	63.5
3	p_R⁻ = 0.9/p_T⁻ = 0.9 p_R⁺ = 0.9/p_T⁺ = 0.9	Probability of rejecting H₀⁻ and H₀⁺	0.826	0.800
Case study 2
		Maximum sample size	62	39
1A	p_R⁻ = 0.3/p_T⁻ = 0.9 p_R⁺ = 0.3/p_T⁺ = 0.9	Expected sample size	34.5	25.7
		Probability of early termination	0.430	0.555
		Probability of rejecting H₀⁻ or H₀⁺	0.083	0.093
1B	p_R⁻ = 0.6/p_T⁻ = 0.6 p_R⁺ = 0.6/p_T⁺ = 0.6	Expected sample size	35.1	24.3
		Probability of early termination	0.408	0.627
		Probability of rejecting H₀⁻ or H₀⁺	0.054	0.092
2	p_R⁻ = 0.3/p_T⁻ = 0.6 p_R⁺ = 0.6/p_T⁺ = 0.9	Expected sample size	42.4	31.0
		Probability of rejecting H₀⁺	0.905	0.802
		Probability of detecting heterogeneity (1st stage)	0.807	0.799
3	p_R⁻ = 0.6/p_T⁻ = 0.9 p_R⁺ = 0.6/p_T⁺ = 0.9	Expected sample size	59.2	37.4
3	p_R⁻ = 0.6/p_T⁻ = 0.9 p_R⁺ = 0.6/p_T⁺ = 0.9	Probability of rejecting H₀⁻ and H₀⁺	0.821	0.800
Case study 3
		Maximum sample size	79	45
1A	p_R⁻ = 0.1/p_T⁻ = 0.8 p_R⁺ = 0.1/p_T⁺ = 0.9	Expected sample size	43.7	31.2
		Probability of early termination	0.511	0.620
		Probability of rejecting H₀⁻ or H₀⁺	0.055	0.086
1B	p_R⁻ = 0.4/p_T⁻ = 0.6 p_R⁺ = 0.4/p_T⁺ = 0.6	Expected sample size	44.8	32.0
		Probability of early termination	0.493	0.582
		Probability of rejecting H₀⁻ or H₀⁺	0.083	0.096
2	p_R⁻ = 0.1/p_T⁻ = 0.6 p_R⁺ = 0.4/p_T⁺ = 0.9	Expected sample size	45.7	34.9
		Probability of rejecting H₀⁺	0.904	0.802
		Probability of detecting heterogeneity (1st stage)	0.864	0.825
3	p_R⁻ = 0.4/p_T⁻ = 0.8 p_R⁺ = 0.4/p_T⁺ = 0.9	Expected sample size	75.8	43.5
3	p_R⁻ = 0.4/p_T⁻ = 0.8 p_R⁺ = 0.4/p_T⁺ = 0.9	Probability of rejecting H₀⁻ and H₀⁺	0.821	0.801

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ISSN: 1471-2288

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