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Table 2 List of decision rules for treatments proceeding to the next stage or concluding treatment efficacy

From: Statistical analysis plan for the motor neuron disease systematic multi-arm adaptive randomised trial (MND-SMART)

Stage

Analysis

Decision rule for treatment to proceed to next stage or conclude treatment efficacy

1

Interim analysis 1

The 95% confidence interval of the rate of change in ALS-FRS-R compared to placebo must include a relative improvement of 25% in the rate of decline

2

Interim analysis 2

One-sided p-value must be below the 10% significance level

3

Final analysis of ALS-FRS-R

One-sided p-value must be below the 2.5% significance level

4

Survival analysis

One-sided p-values for survival and stage 3 ALS-FRS-R analysis must be below the 2.5% significance level to conclude treatment efficacy