Table 1.
Total N= 406 individuals with paired samples | GOAL (Cohort 1: G551D/Other) (N=131) | GOAL (Cohort 2: R117H/other) (N=59) | GOAL (Cohort 3: Non-G551D Gating/other) (N=18) | PROSPECT (F508del homozygous) (N= 139) | PROSPECT (F508del heterozygous) (N= 59) |
---|---|---|---|---|---|
| |||||
Female, N (%) | 60 (45.8%) | 21 (35.6%) | 7 (38.9%) | 76 (54.7%) | 30 (50.8%) |
| |||||
Age (years) at Enrollment: | |||||
Mean (SD) | 20.9 (10.41) | 29.8 (19.97) | 21.3 (10.83) | 20.8 (10.24) | 26.3 (12.62) |
Min, Max | 6.0,59.4 | 6.0,72.9 | 8.6,45.5 | 6.7,57.6 | 12.3,66.8 |
| |||||
Age (years) at Historic SC: | |||||
Mean (SD) | 4.7 (7.23) | 15.9 (19.82) | 7.1 (7.91) | 1.9 (4.80) | 6.9 (10.77) |
Min, Max | 0.0,42.6 | 0.0,69.0 | 0.1,25.0 | 0.0,33.7 | 0.0,45.6 |
| |||||
Time (years) from Historic SC to Enrollment: | |||||
Mean (SD) | 16.2 (9.26) | 13.9 (12.37) | 14.2 (8.64) | 18.9 (9.28) | 19.4 (10.17) |
Min,Max | 0.1,44.0 | 0.1,54.9 | 1.3,31.0 | 4.7,47.8 | 3.9,49.2 |
| |||||
FEV1 % Predicted at Enrollment: | |||||
Mean (SD) | 79.9 (24.78) | 85.8 (20.65) | 69.8 (23.03) | 82.8 (24.12) | 79.8 (25.13) |
Min, Max | 22.9,138.2 | 31.7,118.9 | 22.2,113.8 | 20.6,132.9 | 34.5,138.8 |