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 |