Table 1.
Demographics and baseline characteristics of participants with Prader-Willi syndrome
| ITT population | |||
|---|---|---|---|
| DCCR | Placebo | Overall | |
| Total | 82 | 42 | 124 |
| Age, ya | 13.4 (6.82) | 13.6 (7.37) | 13.5 (6.98) |
| Race, n (%) | |||
| White | 71 (86.6) | 34 (81.0) | 105 (84.7) |
| Black or African American | 4 (4.9) | 2 (4.8) | 6 (4.8) |
| Asian | 0 (0.0) | 1 (2.4) | 1 (0.8) |
| American Indian or Alaska Native | 1 (1.2) | 0 (0.0) | 1 (0.8) |
| Native Hawaiian or Other Pacific Islander | 1 (1.2) | 0 (0.0) | 1 (0.8) |
| Other | 1 (1.2) | 2 (4.8) | 3 (2.4) |
| Multiple | 4 (4.9) | 3 (7.1) | 7 (5.6) |
| Ethnicity n (%) | |||
| Hispanic or Latino | 6 (7.3) | 7 (16.7) | 13 (10.5) |
| Not Hispanic or Latino | 75 (91.5) | 34 (81.0) | 109 (87.9) |
| Not reported | 1 (1.2) | 1 (2.4) | 2 (1.6) |
| Sex, n (%) | |||
| Male | 36 (43.9) | 19 (45.2) | 55 (44.4) |
| Female | 46 (56.1) | 23 (54.8) | 69 (55.6) |
| Height, cma | 146.3 (18.5) | 147.5 (20.1) | 146.7 (19.0) |
| HQ-CTa | 23.0 (6.03) | 21.9 (5.08) | 22.6 (5.73) |
| HQ-CT for HQ-CT > mediana | 28.0 (3.38) | 26.6 (2.65) | 27.61 (3.19) |
| Body fat mass, kga | 27.67 (16.62) | 26.47 (17.58) | 27.25 (16.9) |
| Lean body mass, kga | 29.25 (14.16) | 28.31 (12.52) | 28.92 (13.56) |
| Weight, kga | 62.2 (30.4) | 60.4 (29.6) | 61.6 (30.0) |
| BMIa | 27.7 (9.47) | 26.7 (9.88) | 27.3 (9.58) |
| Hormonal measures | |||
| Serum leptin, ng/mLa | 37.6 (28.6) | 36.3 (28.6) | 37.2 (28.5) |
| Serum adiponectin, μg/mLa | 11.21 (6.98) | 10.41 (6.09) | 10.95 (6.68) |
| Acylated ghrelin, pg/mLa | 242.0 (149.8) | 276.7 (187.7) | 253.7 (163.5) |
| Fasting insulin, μIU/mLa | 12.07 (15.25) | 9.74 (5.99) | 11.28 (12.90) |
| PWS genetic subtype, n (%) | |||
| Deletion | 48 (58.5) | 28 (66.7) | 76 (61.3) |
| Nondeletion | 33 (40.2) | 14 (33.3) | 47 (37.9) |
| Not available | 1 (1.2) | 0 (0.0) | 1 (0.8) |
| Country, n (%) | |||
| United Kingdom | 19 (23.2) | 6 (14.3) | 25 (20.2) |
| United States | 63 (76.8) | 36 (85.7) | 99 (79.8) |
| Growth hormone status, n (%) | |||
| Currently treated | 69 (84.1) | 35 (83.3) | 104 (83.9) |
| Not currently treated | 13 (15.9) | 7 (16.7) | 20 (16.1) |
Abbreviations: BMI, body mass index; DCCR, diazoxide choline extended-release tablet; HQ-CT, Hyperphagia Questionnaire for Clinical Trials; ITT, intent-to-treat; PWS, Prader-Willi syndrome.
Mean (SD).