TABLE 1.
Demographics and clinical characteristics of participants at baseline#
| Subgroup of participants from parent Study 445-102 (F/MF genotypes) |
Subgroup of participants from parent Study 445-103
(F/F genotype) |
All participants in Study 445-105 | |||
|
Placebo
(n=203) |
ELX/TEZ/IVA
(n=196) |
TEZ/IVA
(n=52) |
ELX/TEZ/IVA
(n=55) |
ELX/TEZ/IVA
(n=506) |
|
| Female | 98 (48.3) | 94 (48.0) | 28 (53.8) | 31 (56.4) | 251 (49.6) |
| Age, years | 26.8±11.3 | 25.7±9.7 | 27.9±10.8 | 28.8±11.5 | 26.7±10.7 |
| Age group at screening visit | |||||
| ≥12– <18 years | 60 (29.6) | 55 (28.1) | 14 (26.9) | 16 (29.1) | 145 (28.7) |
| ≥18 years | 143 (70.4) | 141 (71.9) | 38 (73.1) | 39 (70.9) | 361 (71.3) |
| Ethnicity | |||||
| Hispanic or Latino | 12 (5.9) | 4 (2.0) | 3 (5.8) | 2 (3.6) | 21 (4.2) |
| Not Hispanic or Latino | 175 (86.2) | 184 (93.9) | 49 (94.2) | 52 (94.5) | 460 (90.9) |
| Not collected per local regulations | 16 (7.9) | 8 (4.1) | 0 | 1 (1.8) | 25 (4.9) |
| Race¶ | |||||
| White | 184 (90.6) | 183 (93.4) | 52 (100.0) | 54 (98.2) | 473 (93.5) |
| Black or African American | 2 (1.0) | 4 (2.0) | 0 (0) | 0 (0) | 6 (1.2) |
| Asian | 1 (0.5) | 0 (0) | 0 (0) | 0 (0) | 1 (0.2) |
| American Indian or Alaska Native | 1 (0.5) | 0 (0) | 0 (0) | 0 (0) | 1 (0.2) |
| Other | 1 (0.5) | 2 (1.0) | 0 (0) | 0 (0) | 3 (0.6) |
| Not collected per local regulations | 16 (7.9) | 8 (4.1) | 0 (0) | 1 (1.8) | 25 (4.9) |
| Geographical region | |||||
| North America | 120 (59.1) | 117 (59.7) | 33 (63.5) | 34 (61.8) | 304 (60.1) |
| Europe and Australia | 83 (40.9) | 79 (40.3) | 19 (36.5) | 21 (38.2) | 202 (39.9) |
| FEV1 % pred, percentage points | 61.3±15.5 | 61.4±14.9 | 60.2±14.4 | 61.6±15.4 | 61.2±15.1 |
| FEV1 % pred category+ | |||||
| <40 | 16 (7.9) | 18 (9.2) | 4 (7.7) | 6 (10.9) | 44 (8.7) |
| ≥40– <70 | 120 (59.1) | 112 (57.1) | 34 (65.4) | 31 (56.4) | 297 (58.7) |
| ≥70– ≤90 | 62 (30.5) | 65 (33.2) | 14 (26.9) | 18 (32.7) | 159 (31.4) |
| >90 | 5 (2.5) | 1 (0.5) | 0 (0) | 0 (0) | 6 (1.2) |
| Sweat chloride concentration, mmol·L−1 | 102.9±9.8 | 102.4±11.9 | 90.0±12.3 | 91.4±11.0 | 100.1±12.0§ |
| CFQ-R respiratory domain score, points | 70.0±17.8 | 68.2±16.8 | 72.6±17.9 | 70.6±16.2 | 69.6±17.2 |
| BMI, kg·m−2 | 21.31±3.14 | 21.53±3.08 | 21.88±4.12 | 21.75±3.19 | 21.50±3.23 |
Data are presented as n (%) or mean±sd. F: F508del; MF: minimal function; ELX/TEZ/IVA: elexacaftor/tezacaftor/ivacaftor; TEZ/IVA: tezacaftor/ivacaftor; FEV1: forced expiratory volume in 1 s; CFQ-R: Cystic Fibrosis Questionnaire-Revised; BMI: body mass index. #: demographics and baseline characteristics of the full analysis set, which was defined as all enrolled participants who received at least one dose of study drug in the open-label extension study (Study 445-105). Baseline characteristics are based on data obtained at parent study baseline, which was defined as the most recent nonmissing measurement before the first dose of study drug in the parent study treatment period (Study 445-102 or Study 445-103). Baseline in Study 445-103 was assessed after a 4-week run-in period with TEZ/IVA. ¶: the race categories may sum to >100% due to participants being able to indicate more than one race. +: although those eligible for enrolment were required to have FEV1 ≥40% predicted at screening, there were some participants who had decreases to <40% predicted by baseline. §: the baseline mean sweat chloride value is a composite from participants with F/MF and F/F genotypes with different baseline values and is weighted towards the F/MF baseline due to the larger number of participants in this genotype subgroup (n=399 F/MF; n=107 F/F).