Table 1.
Demographics and Clinical Characteristics at Baseline.*
| Tezacaftor/Ivacaftor (n=52) | Elexacaftor/Tezacaftor/ Ivacaftor (n=55) | |
|---|---|---|
| Female gender ― no. (%) | 28 (54) | 31 (56) |
| Age at baseline | ||
| Mean ― yr | 27·9 ±10·8 | 28·8 ±11·5 |
| Distribution ― no. (%)† | ||
| ≥12 to <18 yr | 14 (27) | 16 (29) |
| ≥18 yr | 38 (73) | 39 (71) |
| Geographic region ― no. (%) | ||
| North America | 33 (63) | 34 (62) |
| Europe | 19 (37) | 21 (38) |
| Percentage of predicted FEV1 | ||
| Mean | 60·2 ±14·4 | 61·6 ±15·4 |
| Distribution ― no. (%) | ||
| <40%‡ | 4 (8) | 6 (11) |
| ≥40% to <70% | 34 (65) | 31 (56) |
| ≥70% to ≤90% | 14 (27) | 18 (33) |
| >90% | 0 | 0 |
| Body-mass index, mean§ | 21·88 ±4·12 | 21·75 ±3·19 |
| Sweat chloride concentration, mean ― mmol/L | 90·0 ±12·3 | 91·4 ±11·0 |
| CFQ-R respiratory domain score, mean∥ | 72·6 ±17·9 | 70·6 ±16·2 |
| Pseudomonas aeruginosa-positive within previous 2 years ― no. (%) | 31 (60) | 39 (71) |
| Prior medication use, n (%)¶ | ||
| Dornase alfa | ||
| Yes | 48 (92) | 51 (93) |
| No | 4 (8) | 4 (7) |
| Azithromycin | ||
| Yes | 25 (48) | 33 (60) |
| No | 27 (52) | 22 (40) |
| Inhaled antibiotic | ||
| Yes | 28 (54) | 35 (64) |
| No | 24 (46) | 20 (36) |
| Bronchodilator | ||
| Yes | 47 (90) | 54 (98) |
| No | 5 (10) | 1 (2) |
| Inhaled hypertonic saline | ||
| Yes | 41 (79) | 38 (69) |
| No | 11 (21) | 17 (31) |
| Inhaled corticosteroids | ||
| Yes | 28 (54) | 36 (65) |
| No | 24 (46) | 19 (35) |
| CFTR modulator therapy | ||
| Yes | 34 (65) | 32 (58) |
| No | 18 (35) | 23 (42) |
CFQ-R=Cystic Fibrosis Questionnaire–Revised; FEV1=forced expiratory volume in 1 second.
Plus–minus values are means ±SD.
Age distribution was calculated based on age at the time of screening.
Although those eligible for enrolment were required to have a percent predicted FEV1 ≥40 at screening, some participants experienced a decrease to a value <40 by baseline.
The body-mass index is the weight in kilograms divided by the square of the height in meters.
Scores on the CFQ-R range from 0 to 100, with higher scores indicating a higher participant-reported quality of life with regard to respiratory status.
Includes medications administered during the 56 days before the first dose of trial drug in the treatment period.