Table 1.
Demographic and Baseline Characteristics of Randomized Participants
| Hypertonic Saline (HS) Trial | Dornase Alfa (DA) Trial | |||||
|---|---|---|---|---|---|---|
| Continue N=186 | Discontinue N=184 | Total N=370 | Continue N=237 | Discontinue N=240 | Total N=477 | |
| Sex at Birth | ||||||
| Male | 99 (53.2%) | 97 (52.7%) | 196 (53.0%) | 117 (49.4%) | 127 (52.9%) | 244 (51.2%) |
| Female | 87 (46.8%) | 87 (47.3%) | 174 (47.0%) | 120 (50.6%) | 113 (47.1%) | 233 (48.8%) |
| Age (years) | ||||||
| N | 186 | 184 | 370 | 237 | 240 | 477 |
| Mean (SD) | 22.4 (10.73) | 21.7 (10.28) | 22.0 (10.50) | 23.2 (11.50) | 21.9 (9.19) | 22.6 (10.41) |
| Age Distribution | ||||||
| ≥12 to <18 | 92 (49.5%) | 90 (48.9%) | 182 (49.2%) | 108 (45.6%) | 110 (45.8%) | 218 (45.7%) |
| ≥18 to <24 | 39 (21.0%) | 37 (20.1%) | 76 (20.5%) | 48 (20.3%) | 51 (21.3%) | 99 (20.8%) |
| ≥24 to <30 | 18 (9.7%) | 32 (17.4%) | 50 (13.5%) | 31 (13.1%) | 32 (13.3%) | 63 (13.2%) |
| ≥30 | 37 (19.9%) | 25 (13.6%) | 62 (16.8%) | 50 (21.1%) | 47 (19.6%) | 97 (20.3%) |
| Race | ||||||
| White | 182 (97.8%) | 176 (95.7%) | 358 (96.8%) | 227 (95.8%) | 231 (96.3%) | 458 (96.0%) |
| Black or African American | 1 (0.5%) | 2 (1.1%) | 3 (0.8%) | 4 (1.7%) | 1 (0.4%) | 5 (1.0%) |
| Asian | 0 (0%) | 1 (0.5%) | 1 (0.3%) | 1 (0.4%) | 0 (0%) | 1 (0.2%) |
| American Indian or Alaskan Native | 0 (0%) | 0 (0%) | 0 (0%) | 0 (0%) | 1 (0.4%) | 1 (0.2%) |
| Native Hawaiian or Other Pacific Islander | 0 (0%) | 0 (0%) | 0 (0%) | 0 (0%) | 0 (0%) | 0 (0%) |
| Other/Unknown* | 3 (1.6%) | 5 (2.7%) | 8 (2.2%) | 5 (2.1%) | 7 (2.9%) | 12 (2.5%) |
| Ethnicity | ||||||
| Hispanic or Latino | 6 (3.2%) | 14 (7.6%) | 20 (5.4%) | 15 (6.3%) | 19 (7.9%) | 34 (7.1%) |
| ot Hispanic or Latino | 180 (96.8%) | 170 (92.4%) | 350 (94.6%) | 222 (93.7%) | 221 (92.1%) | 443 (92.9%) |
| Genotype Group | ||||||
| F508del Homozygous | 111 (59.7%) | 111 (60.3%) | 222 (60.0%) | 140 (59.1%) | 129 (53.8%) | 269 (56.4%) |
| F508del Heterozygous | 72 (38.7%) | 71 (38.6%) | 143 (38.6%) | 91 (38.4%) | 106 (44.2%) | 197 (41.3%) |
| Other/Unknown | 3 (1.6%) | 2 (1.1%) | 5 (1.4%) | 6 (2.5%) | 5 (2.1%) | 11 (2.3%) |
| FEV1 (% predicted) | ||||||
| N | 186 | 184 | 370 | 237 | 240 | 477 |
| Mean (SD) | 97.2 (16.50) | 96.7 (17.17) | 97.0 (16.82) | 97.2 (16.32) | 96.5 (17.34) | 96.8 (16.83) |
| FEV1 (% predicted) Distribution | ||||||
| <60 | 1 (0.5%) | 1 (0.5%) | 2 (0.5%) | 3 (1.3%) | 1 (0.4%) | 4 (0.8%) |
| ≥60 to <70 | 10 (5.4%) | 13 (7.1%) | 23 (6.2%) | 11 (4.6%) | 20 (8.3%) | 31 (6.5%) |
| ≥70 to <90 | 53 (28.5%) | 46 (25.0%) | 99 (26.8%) | 63 (26.6%) | 57 (23.8%) | 120 (25.2%) |
| ≥90 to <100 | 33 (17.7%) | 45 (24.5%) | 78 (21.1%) | 51 (21.5%) | 53 (22.1%) | 104 (21.8%) |
| ≥100 | 89 (47.8%) | 79 (42.9%) | 168 (45.4%) | 109 (46.0%) | 109 (45.4%) | 218 (45.7%) |
| LCI2.5 † | ||||||
| N | 47 | 52 | 99 | 61 | 69 | 130 |
| Mean (SD) | 7.8 (2.15) | 8.2 (2.56) | 8.0 (2.37) | 7.9 (1.77) | 8.2 (2.73) | 8.1 (2.32) |
| Prior Study Enrollee | 62 (33.3%) | 62 (33.7%) | 124 (33.5%) | 64 (27.0%) | 65 (27.1%) | 129 (27.0%) |
| Current Chronic Therapy | ||||||
| DA | 156 (83.9%) | 154 (83.7%) | 310 (83.8%) | 237 (100%) | 240 (100%) | 477 (100%) |
| HS | 186 (100%) | 184 (100%) | 370 (100%) | 147 (62.0%) | 148 (61.7%) | 295 (61.8%) |
| ETI | 186 (100%) | 184 (100%) | 370 (100%) | 237 (100%) | 240 (100%) | 477 (100%) |
| Airway clearance | 178 (95.7%) | 174 (94.6%) | 352 (95.1%) | 228 (96.2%) | 226 (94.2%) | 454 (95.2%) |
| Inhaled antibiotic (Continuous) | 7 (3.8%) | 2 (1.1%) | 9 (2.4%) | 3 (1.3%) | 6 (2.5%) | 9 (1.9%) |
| Inhaled antibiotic (Cycled) | 51 (27.4%) | 43 (23.4%) | 94 (25.4%) | 50 (21.1%) | 56 (23.3%) | 106 (22.2%) |
| Inhaled antibiotic (Continuous Alternating) | 17 (9.1%) | 15 (8.2%) | 32 (8.6%) | 25 (10.5%) | 23 (9.6%) | 48 (10.1%) |
| Oral antibiotic | 85 (45.7%) | 81 (44.0%) | 166 (44.9%) | 103 (43.5%) | 104 (43.3%) | 207 (43.4%) |
| Ibuprofen | 8 (4.3%) | 15 (8.2%) | 23 (6.2%) | 20 (8.4%) | 21 (8.8%) | 41 (8.6%) |
| Systemic steroids | 1 (0.5%) | 2 (1.1%) | 3 (0.8%) | 2 (0.8%) | 3 (1.3%) | 5 (1.0%) |
| Previous Modulator Use ‡ | ||||||
| Ivacaftor | 8 (4.3%) | 1 (0.5%) | 9 (2.4%) | 7 (3.0%) | 12 (5.0%) | 19 (4.0%) |
| Lumacaftor/Ivacaftor | 49 (26.3%) | 54 (29.3%) | 103 (27.8%) | 69 (29.1%) | 62 (25.8%) | 131 (27.5%) |
| Tezacaftor/Ivacaftor | 45 (24.2%) | 43 (23.4%) | 88 (23.8%) | 53 (22.4%) | 55 (22.9%) | 108 (22.6%) |
| Positive Microbiology Culture (past year) § | ||||||
| Pseudomonas aeruginosa | 57 (30.6%) | 39 (21.2%) | 96 (25.9%) | 63 (26.6%) | 66 (27.5%) | 129 (27.0%) |
| Staphylococcus aureus | 118 (63.4%) | 137 (74.5%) | 255 (68.9%) | 163 (68.8%) | 158 (65.8%) | 321 (67.3%) |
| Methicillin-resistant staphylococcus aureus | 34 (18.3%) | 40 (21.7%) | 74 (20.0%) | 56 (23.6%) | 54 (22.5%) | 110 (23.1%) |
| Stenotrophomonas maltophilia | 9 (4.8%) | 11 (6.0%) | 20 (5.4%) | 19 (8.0%) | 9 (3.8%) | 28 (5.9%) |
| Achromobacter xylosoxidans | 1 (0.5%) | 2 (1.1%) | 3 (0.8%) | 4 (1.7%) | 4 (1.7%) | 8 (1.7%) |
| Burkholderia cepacia complex | 3 (1.6%) | 2 (1.1%) | 5 (1.4%) | 4 (1.7%) | 4 (1.7%) | 8 (1.7%) |
| Haemophilus influenzae | 9 (4.8%) | 5 (2.7%) | 14 (3.8%) | 12 (5.1%) | 8 (3.3%) | 20 (4.2%) |
| Mycobacterium abscessus | 2 (1.1%) | 1 (0.5%) | 3 (0.8%) | 1 (0.4%) | 2 (0.8%) | 3 (0.6%) |
| Mycobacterium avium complex | 1 (0.5%) | 3 (1.6%) | 4 (1.1%) | 4 (1.7%) | 3 (1.3%) | 7 (1.5%) |
Data are mean (SD) or n (%). Percentages may not sum to 100 in each category due to rounding.
*
Other includes participants of more than one race.
†
LCI result could be obtained at either the Week –2 or Week 0 Visit.
‡
Participants may fall into more than one category of prior modulator use.
§
Culture results obtained clinically within 12 months prior to screening.