Table 2 Summary of the study designs of the clinical trials
Items | Definition | All cases completed | Not all cases completed | p value |
|---|---|---|---|---|
n = 95 | n = 47 | |||
n (%) | n (%) | |||
Rare disease | Yes | 27 (28.4) | 13 (27.7) | 0.92 |
Phase | 2 | 28 (29.5) | 18 (38.3) | 0.87 |
2/3 | 2 (2.1) | 0 (0) | ||
3 | 50 (52.6) | 22 (46.8) | ||
Other | 15 (15.8) | 7 (14.9) | ||
Randomized | Yes | 58 (61.1) | 30 (63.8) | 0.75 |
Double-blind | Yes | 42 (44.2) | 19 (40.4) | 0.67 |
Clinical trials that included newborns or infants and toddlers | Yes | 19 (20) | 10 (21.7) | 0.86 |
Median, min–max | Median, min–max | |||
Number of enrolled participants per age categories | All age categories including over 18 years | 111, 1–4176 | 42, 6–1177 | 0.02 |
Newborn | 0, 0–16 | 0, 0–4 | 0.43 | |
Infant and toddler | 0, 0–1229 | 0, 0–110 | 0.67 | |
Child | 2, 0–978 | 0, 0–506 | 0.07 | |
Adolescent | 8, 0–448 | 1, 0–327 | 0.03 | |
Number of countries in which the clinical trial was conducted | 2, 1–10 | 4, 1–18 | < 0.001 | |
Year clinical trial started | 2014 | 41 (43) | 24 (51) | 0.65 |
2015 | 32 (34) | 10 (21) | ||
2016 | 16 (17) | 10 (21) | ||
2017 | 6 (6.3) | 3 (6.4) | ||
Trial period (days) | Final date–start date + 1 | 616, 130–1570 | 723.5, 81–1524 | 0.18 |