Table 2.
Placebo-treated groups with FXS in failed clinical trials: substantial placebo effect in endpoint CGI-I.
| N Endpoint | Mean Endpoint | Mean Endpoint SEM | Reference |
|---|---|---|---|
| 37 | 3.40 | 0.17 | [23] (>12 yo) |
| 15 | 3.10 | 0.27 | [23] (<12 yo) |
| 82 | 3.50 | 0.14 | [26] (>12 yo) |
| 37 | 3.40 | 0.17 | [27] (>12 yo) |
| 15 | 3.10 | 1.25 | [27] (<12 yo) |
| 27 | 2.60 | 0.16 | [28] (<12 yo) |
| 62 | 3.06 | 0.11 | [29] (>12 yo) * |
| MD | MD | MD | [30] (>12 yo) ** |
| 44 | 3.30 | 1.00 | [25] (<12 yo) *** |
| 62 | 3.10 | 0.12 | [25] (>12 yo) *** |
| ND | ND | ND | [24] (>12 yo) ** |
| 25 | 3.07 | MD | [38] (>12 yo) |
Abbreviations: FXS, fragile X syndrome; CGI-I, Clinician Global Impression-Improvement; N, number; SEM, standard error of the mean; MD, missing data; ND, no data. * Mean endpoint represents mean change. ** Mean endpoint was not reported in the study. *** Mean endpoint was reported, but mean baseline was not reported.