Table 1 Subject demographics and baseline characteristics
From: Phase I clinical trial results of aceneuramic acid for GNE myopathy in Japan
Demographics | Trial-1 | Trial-2 | ||||||
|---|---|---|---|---|---|---|---|---|
800 mg × 1 n = 3 | 800 mg × 3 n = 3 | Total n = 6 | 800 mg × 3 × 5 days n = 3 | 2,000 mg × 1 n = 3 | 2,000 mg × 3 n = 3 | Total n = 6 | 2,000 mg × 3 × 7 days n = 3 | |
Age in years, mean (STDV) | 32.0 (8.2) | 33.0 (5.2) | 32.5 (6.2) | 36.7 (5.8) | 32.0 (5.6) | 29.3 (6.7) | 30.7 (5.7) | 32.7 (6.1) |
Sex (male), n (%) | 2 (66.7) | 1 (33.3) | 3 (50.0) | 1 (33.3) | 2 (66.7) | 1 (33.3) | 3 (50.0) | 2 (66.7) |
Height cm, mean (STDV) | 172.0 (8.2) | 161.7 (11.7) | 166.8 (10.7) | 163.3 (14.6) | 171.0 (8.7) | 169.7 (10.6) | 170.3 (8.7) | 172.3 (8.6) |
Weight kg, mean (STDV) | 61.6 (14.3) | 53.8 (9.8) | 57.7 (11.7) | 59.7 (18.2) | 56.7 (4.2) | 51.6 (14.5) | 54.2 (9.9) | 61.3 (10.3) |
BMI in kg/m2, mean (STDV) | 20.7 (4.4) | 20.6 (3.0) | 20.6 (2.9) | 22.0 (3.3) | 19.6 (3.6) | 17.7 (3.3) | 18.7 (3.2) | 20.7 (3.5) |
Race, n (%) Japanese | 3 (100) | 3 (100) | 6 (100) | 3 (100) | 3 (100) | 3 (100) | 6 (100) | 3 (100) |
Disease duration in years, mean (STDV) | 10.4 (4.4) | 12.1 (5.0) | 11.3 (4.3) | 13.2 (5.1) | 11.7 (3.5) | 10.4 (8.4) | 11.1 (5.8) | 11.6 (5.0) |