Demographics and baseline characteristics by study arm
| No. (%) | |||
|---|---|---|---|
| Treatment (n=24) | Observational control (n=21) | Total (n=45) | |
| Sex—female | 10 (42) | 10 (48) | 20 (44) |
| Race | |||
| Caucasian | 19 (79) | 17 (81) | 36 (80) |
| Hispanic | 3 (13) | 2 (10) | 5 (11) |
| African-American | 1 (4) | 1 (5) | 2 (4) |
| Other | 1 (4) | 1 (5) | 2 (4) |
| Genotype | |||
| F508 del homozygous | 6 (25) | 12 (57) | 18 (40) |
| F508 del heterozygous | 14 (58) | 7 (33) | 21 (47) |
| Other* | 4 (17) | 2 (10) | 6 (13) |
| Age group (years) | |||
| 4–12 | 13 (54) | 15 (71) | 28 (62) |
| >12–18 | 6 (25) | 5 (24) | 11 (24) |
| >18 | 5 (21) | 1 (5) | 6 (13) |
| Pseudomonas aeruginosa-positive | 4 (17) | 4 (19) | 8 (18) |
| FEV1% predicted group† | |||
| 30–50% predicted | 1 (5) | 0 (0) | 1 (3) |
| >50–75% predicted | 1 (5) | 0 (0) | 1 (3) |
| >75–100% predicted | 7 (35) | 5 (29) | 12 (32) |
| >100% predicted | 11 (55) | 12 (71) | 23 (62) |
| Mean (SD) | |||
| Age (years) | 12.3 (6.6) | 10.5 (5.5) | 11.5 (6.1) |
| FEV1% predicted† | 98.5 (21.6) | 101.2 (11.8) | 99.8 (17.6) |
| Weight (kg) | 40.5 (17.0) | 38.2 (19.8) | 39.4 (18.2) |
| Weight (%)‡ | 50.5 (27.4) | 53.7 (23.9) | 52.0 (25.5) |
| Body mass index (%)‡ | 60.8 (25.4) | 64.6 (20.5) | 62.7 (23.0) |
This table summarises demographic and baseline characteristics by study arm in the ITT population. All measures were recorded at screening.
*Other refers to participants with either two known, non-delta F508 CF mutations, or one known, non-F508 del CF mutation and one unidentified allele which has not been classified as a CF mutation.
‡For participants 6 years or older, FEV1% predicted is calculated based on the Wang (boys <18 years, girls <16 years) or Hankinson (boys ≥18 years, girls ≥16 years) reference equations. Percentages are based on number of participants with FEV1 measurements available (20 in the treatment arm and 17 in the observational control arm).
‡The centiles are derived using CDC standards for participants ≤20 years old.
CF, cystic fibrosis; ITT, intent-to-treat.