Table 2.
Shows the baseline characteristics of the included studies
| Study ID | Glaze et al., 2017 | Glaze et al., 2019 | Neul et al., 2023 | ||||||
|---|---|---|---|---|---|---|---|---|---|
| Trofinetide | Placebo | Trofinetide | Placebo | Trofinetide | Placebo | ||||
| 35 mg/kg | 70 mg/kg | 50 mg/kg | 100 mg/kg | 200 mg/kg | 200 mg/kg | ||||
| Number of patients (n) | 13 | 17 | Doses: (35 mg/kg) = 5, (70 mg/kg) = 11 | 15 | 16 | 27 | 24 | 93 | 94 |
| Gender [Female n (%)] | 30 (100%) | 16 (100%) | 58 (100%) | 24 (100%) | 93 (100%) | 94 (100%) | |||
| Age, year [Mean (SD)] | 22.62 (5.582) | 24.52 (5.853) | Doses: (35 mg/kg) = 32.09 (9.324), (70 mg/kg) = 27.09 (8.357) | 10.06 (3.18) | 10.81 (3.10) | 9.23 (3.88) | 9.38 (3.26) | 11.0 (4.69) | 10.9 (4.57) |
| BMI, kg/cm2 [Mean (SD)] | 25.06 (7.930) | 20.48 (6.765) | Doses: (35 mg/kg) = 24.66 (8.04), (70 mg/kg) = 19.24 (3.598) | 16.50 (3.61) | 17.70 (5.06) | 16.31 (3.57) | 16.00 (2.85) | ||
| Ethnicity, n (%) | |||||||||
| Hispanic | 0 | 2 (12%) | Doses: (35 mg/kg) = 1 (20%), (70 mg/kg) = 0 | 1 (7%) | 1 (6%) | 6 (22%) | 0 | ||
| Not Hispanic | 13 (100%) | 15 (88%) | Doses: (35 mg/kg) = 4 (80%), (70 mg/kg) = 11 (100%) | 14 (93%) | 14 (88%) | 21 (78%) | 24 (100%) | ||
| Race, n (%) | |||||||||
| Asian | 0 | 1 (6%) | Doses: (35 mg/kg) = 0, (70 mg/kg) = 0 | 0 | 0 | 2 (7%) | 1 (4%) | 5 (5.4%) | 1 (1.1%) |
| Black or African-American | 3 (23%) | 1 (6%) | Doses: (35 mg/kg) = 0, (70 mg/kg) = 0 | 0 | 1 (6%) | 0 | 0 | 1 (1.1%) | 1 (1.1%) |
| White | 10 (77%) | 15 (88%) | Doses: (35 mg/kg) = 5 (100%), (70 mg/kg) = 11 (100%) | 15 (100%) | 15 (94%) | 25 (93%) | 22 (92%) | 82 (88.2%) | 90 (95.7%) |
| Other | 0 | 0 | 0 | 0 | 1 (4%) | 4 (4.3%) | 2 (2.1%) | ||