Table 1.
Baseline Characteristics of Patients
| Pivotal patients with BBS and Alstrӧm syndrome (SAS) (N=38) | Pivotal patients ≥12 years old with BBS and Alstrӧm syndrome (FAS) (n=31) | Pivotal patients with BBS (n=32) | |
|---|---|---|---|
| Age, yearsa | 19·8 (10·2) [16·5; 12·0–24·0] | 21·6 (10·3) [19·0; 13·0–28·0] | 20·2 (10·2) [17·5; 12·0–25·5] |
| Female, n (%) | 23 (60·5) | 17 (54·8) | 17 (53·1) |
| Ethnicity, n (%) | |||
| Hispanic or Latino | 1 (2·6) | 1 (3·2) | 1 (3·1) |
| Not Hispanic or Latino | 37 (97·4) | 30 (96·8) | 31 (96·9) |
| Race, n (%) | |||
| White | 31 (81·6) | 26 (83·9) | 28 (87·5) |
| Black or African American | 3 (7·9) | 2 (6·5) | 1 (3·1) |
| Asian | 1 (2·6) | 0 | 0 |
| Other | 3 (7·9) | 3 (9·7) | 3 (9·4) |
| Cognitive impairment, n (%) | 19 (50·0) | 15 (48·4) | 17 (53·1) |
| Weight, kga | 111·7 (30·4) [112·3; 92·0–129·8] | 117·2 (29·2) [113·8; 98·7–130·2] | 112·3 (27·9) [113·4; 94·6–129·9] |
| BMI, kg/m2a | 42·3 (11·0) [41·0; 34·9–47·1] | 43·5 (11·4) [41·5; 36·9–47·4] | 41·6 (90) [41·3; 35·4–46·7] |
| Maximal hunger scoreb | — | 7·3 (2·0) | 6·8 (1·8) |
Data are mean (SD) [median; interquartile range] unless otherwise specified. BBS, Bardet-Biedl syndrome; BMI, body mass index; FAS, full analysis set; SAS, safety analysis set; SD, standard deviation.
a
At placebo-controlled period baseline.
b
At active treatment baseline in patients ≥12 years old without cognitive impairment; self-reported (FAS: n=16; pivotal patients with BBS: n=18).