Table 3.
Clinical efficacy results in the modified intention-to-treat sample
| Endpointa | PR-fampridine (n = 315) | Placebo (n = 318) | p-value vs. placebo |
|---|---|---|---|
| Clinically meaningful improvement (≥ 8 points) in MSWS-12 score from baseline over 24 weeks (primary endpoint) | |||
| Participants with improvement, n (%)b,c | 136 (43.2) | 107 (33.6) | 0.006d |
| Odds ratio vs. placebo (95% CI)d | 1.61 (1.15 to 2.26) | NA | |
| Risk difference for adjusted proportions (95% CI)d | 0.104 (0.030 to 0.178) | ||
| Relative risk (95% CI)d | 1.38 (1.06 to 1.70) | ||
| MSWS-12 score change from baseline over 24 weeks | |||
| LSM change over 24 weeks (95% CI)e | − 6.73 (− 8.80 to − 4.67) | − 2.59 (− 4.71 to − 0.47) | |
| LSM difference vs. placebo (95% CI)e | − 4.14 (− 6.22 to − 2.06) | NA | < 0.001 |
| Clinically meaningful mean improvement (≥ 15%) in TUG speed from baseline over 24 weeks (secondary endpoint: rank group 1) | |||
| Participants with improvement, n (%)c | 137 (43.4) | 110 (34.7) | 0.03d |
| Odds ratio vs. placebo (95% CI)d | 1.46 (1.04 to 2.07) | NA | |
| Risk difference for adjusted proportions (95% CI)d | 0.092 (0.009 to 0.175) | ||
| Relative risk (95% CI)d | 1.25 (0.99 to 1.51) | ||
| TUG speed change from baseline over 24 weeks, ft/s | |||
| LSM change from baseline (95% CI)e | 0.05 (0.04 to 0.06) | 0.03 (0.02 to 0.04) | |
| LSM difference from baseline vs. placebo (95% CI)e | 0.02 (0.01 to 0.03) | < 0.001 | |
| TUG time change from baseline over 24 weeks, s | |||
| LSM change from baseline (95% CI)e | − 3.30 (− 4.78 to − 1.83) | − 1.94 (− 3.46 to − 0.41) | |
| LSM difference from baseline vs. placebo (95% CI)e | − 1.36 (− 2.85 to 0.12) | 0.073 | |
| MSIS-29 PHYS score change from baseline over 24 weeks (secondary endpoint: rank group 1) | |||
| LSM change from baseline (95% CI)e | − 8.00 (− 9.78 to − 6.21) | − 4.68 (− 6.52 to − 2.85) | |
| LSM difference from baseline vs. placebo (95% CI)e | − 3.31 (− 5.13 to − 1.50) | NA | < 0.001 |
| BBS score change from baseline over 24 weeks (secondary endpoint: rank group 2) | |||
| LSM change from baseline (95% CI)e | 1.75 (1.20 to 2.29) | 1.34 (0.78 to 1.89) | |
| LSM difference from baseline vs. placebo (95% CI)e | 0.41 (− 0.13 to 0.95) | 0.141 | |
| ABILHAND score change from baseline over 24 weeks (secondary endpoint: rank group 2) | |||
| Participants, n | 312 | 315 | |
| LSM change from baseline (95% CI)e | 1.49 (0.36 to 2.61) | 0.75 (− 0.41 to 1.91) | |
| LSM difference from baseline vs. placebo (95% CI)e | 0.74 (− 0.38 to 1.86) | NA | 0.197 |
BBS Berg Balance Scale, CI confidence interval, LSM least squares mean. MSIS-29 PHYS Multiple Sclerosis Impact Scale physical impact subscale, MSWS-12 12-item Multiple Sclerosis Walking Scale, NA not applicable, PR prolonged-release, TUG Timed Up and Go
aA complete definition of endpoints is provided in Table 1
bBased on seven on-treatment assessments per participant in the modified intention-to-treat sample. The level of missing post-baseline MSWS-12 data was generally similar between treatment groups except for missing data due to discontinuations (PR-fampridine, 5%; placebo, 9%)
cPercentage based on binomial proportions
dCalculated using an adjusted logistic regression model (missing data imputed using multiple imputation)
eBased on a mixed-effects model for repeated measures using a common variance/covariance matrix structure