Table 3.
Stage 2 Efficacy Results
| Primary Outcome: Mean 9-month ALSFRSr decline (SD) by treatment group | |||||
|---|---|---|---|---|---|
| CoQ10 2,700mg* n=75 | Placebo n=75 | Delta (CI)** | t statistic | p-value | |
| Primary analysis1 | 8.80 (7.34) | 9.44 (8.82) | -1.25 (< 0.22) | -1.09 | 0.14 |
| Sensitivity 1 – LTF2 | 8.52 (7.41) | 9.44 (8.82) | -0.97(< 0.51) | -0.84 | 0.20 |
| Sensitivity 2 – Death3 | 8.52 (6.54) | 8.37 (6.56) | -1.82 (< -0.63) | -1.97 | 0.025 |
| Secondary Outcomes: Mean 9-month Decline (SD) by treatment group | |||||
| Forced Vital Capacity (% of predicted) | 0.20 (0.15) | 0.17 (0.18) | -0.03 (-0.08, 0.02) | -1.12 | 0.27 |
| SF-36 – PCS (physical) | 4.22 (8.02) | 6.04 (6.85)*** | 1.83 (-0.60, 4.26) | 1.50 | 0.14 |
| SF-36 – MCS (mental) | 2.87 (11.78) | 5.01 (11.69) | 2.14 (-1.67, 5.95) | 1.11 | 0.27 |
| Fatigue Severity | 0.71 (1.21) | 0.88 (1.51) | 0.17 (-0.27, 0.61) | 0.76 | 0.45 |
Means (SD) include abias correction.
Delta (CI) is consistent with the respective hypothesis tests for the primary and secondary outcomes. For the primary outcome, delta = (0.8 × placebo group mean decline in 9-month ALSFRSr) -CoQ10 group mean decline in 9-month ALSFRSr. For the secondary outcomes delta is the simple difference between placebo and CoQ10 group mean declines. Confidence intervals (CI) are one-sided 90% CIs for the primary outcome and two-sided 95% CIs for the secondary outcomes. For the primary outcome, a 90% upper confidence bound for delta that is negative (less than zero) indicates that the data are not consistent with the hypothesis that CoQ10 is associated with a ≥20% slowing in mean decline on the ALSFRSr. For the primary outcome, the true percent reduction in mean ALSFRSr is related to the true delta via the formula true % reduction = 100% × (0.2 + true delta / true placebo group mean). Given a fixed value of the true placebo group mean such as 9.0 (one point per month) or 9.4 (as observed), the conditional 90% upper confidence limit for the true percent reduction is 100% × (0.2 + upper confidence limit for true delta / assumed true placebo group mean).
n=73
Worst-scenario imputation for the two CoQ10 cases lost to follow-up.
Best-scenarioimputation for the two CoQ10 cases lost to follow-up.
The 6 deceased patients (1 CoQ10, 5 placebo) are scored 16 instead of 0 on the 9-month ALSFRSr. Worst-scenario imputation for the two CoQ10 cases lost to follow-up.