6. Oral olesoxime versus placebo outcomes (Bertini 2017).
| Olesoxime | Placebo | Estimated difference (95% CI)a | P value | |
| Number of participants randomised | 108 | 57 | — | — |
| Number (%) of participants evaluable for analysis MFM‐32 D1+D2 | 103 (95%) | 57 (100%) | — | — |
| Meanab change (95% CI) in disability score (MFM D1+D2) at 24 months | ‐0.18 (‐1.30 to 1.66) | ‐1.82 (‐3.68 to 0.04) | 2.00 (‐0.25 to 4.25) | 0.07 |
| Number (%) of participants evaluable for analysis MFM total score | 103 (95%) | 57 (100%) | — | — |
| Meanab change (95% CI) in disability score (MFM total score) at 24 months | 0.59 (‐0.9 to 2.070 | ‐1.45 (‐3.31 to 0.41) | 2.04 (‐0.21 to 4.28) | 0.08 |
| Number (%) of participants evaluable for analysis HFMS | 103 (95%) | 57 (100%) | — | — |
| Meanb change (95% CI) in disability score (HFMS) at 24 months | ‐0.78 (‐1.60 to 0.04) | ‐1.72 (‐2.74 to ‐0.70) | 0.94 (‐0.28 to 2.17) | 0.13 |
| Number (%) of participants evaluable for analysis of nerve innervation value (CMAP) (in mV) | 70 (65%) | 34 (60%) | — | — |
| Meanb (95% CI) change in total amplitude CMAP from baseline to 24 months | ‐0.07 (‐0.49 to 0.36) | ‐0.16 (‐0.74 to 0.43) | NA | 0.79 |
| Number (%) of participants evaluable for analysis of MUNE | 58 (54%) | 30 (53%) | — | — |
| Meanb (95% CI) change in total MUNE from baseline to 24 months | ‐4.51 (‐12.21 to 3.18) | ‐6.69 (‐16.86 to 3.48) | NA | 0.71 |
| Number (%) of participants evaluable for FVC | 64 (59%) | 38 (67%) | — | — |
| Meanb (95% CI) change in FVC from baseline to 24 months | 4.28 (‐0.32 to 8.88) | 6.16 (1.00 to 11.33) | ‐1.88 (‐3.14 to 6.91) | 0.57 |
| Olesoxime | Placebo | Difference (95% CI) | P value | |
| Number (%) of participants evaluable for analysis of quality of life (PedsQL) from baseline to 24 months | 71 (66%) | 37 (65%) | — | — |
| Mean (SD) change in quality of life (PedsQL) from baseline to 24 months | NA | NA | 0.25 (‐4.58 to 5.08) | 0.92 |
| Olesoxime | Placebo | Risk ratio (95% CI) | P value | |
| Number (%) of participants evaluable for responder analysis MFM‐32 score D1+D2 (24 months) | 103 (95%) | 57 (100%) | — | — |
| Proportion of participants with good response according to MFM‐32 score D1+D2c | 56 (54) | 22 (39) | 1.43 (‐0.98 to 2.08) | 0.06 |
| Number of adverse events | 1104 (64) | 612 (36) | — | — |
| Number (%) of participants with adverse events | 103 (95%) | 57 (100%) | 0.95 (0.91 to 0.99) | 0.02 |
| Number (%) of participants with severe adverse events | 18 (17%) | 14 (25%) | 0.67 (0.37 to 1.26) | — |
| Number of deaths | 1 | 1 | — | — |
| Number (%) of participants discontinued intervention | 10 (9%) | 7 (12%) | — | — |
CI: confidence interval; CMAP: compound muscle action potential; FVC: forced vital capacity; HFMS: Hammersmith Functional Motor Scale; MFM: Motor Function Measure; MFM D1+D2: functional domains 1 and 2 of the Motor Function Measure; MUNE: motor unit number estimation; NA: not available; PedsQL: Pediatric Quality of Life Inventory; SD: standard deviation.
a Least squared mean of MFM, including MFM‐32 and MFM‐20 assessments. b Calculated with available data. c Participants with no change or improvement of scores were considered 'responders', participants with a decline in score were considered 'non‐responders'.