Background
Methods
Study design
Patients
Treatment
Assessments
Pharmacokinetics
Safety
Taste acceptability
Efficacy assessment
Statistical analysis
Results
Patients
Cohort 1 (12 to < 18 years)
N = 8 | Cohort 2 (6 to < 12 years)
N = 9 | Cohort 3 (2 to < 6 years)
N = 9 | All patients (2 to < 18 years)
N = 26 | |
---|---|---|---|---|
Age at enrollment, years | ||||
Median (Q1–Q3) | 14.0 (12.0–16.0) | 10.0 (8.0–11.0) | 4.0 (4.0–5.0) | 9.5 (5.0–12.0) |
Gender, n (%) | ||||
Female | 5 (62.5) | 5 (55.6) | 7 (77.8) | 17 (65.4) |
Body mass index,a kg/m2
| ||||
Median (Q1–Q3) | 19.9 (17.1–21.9) | 16.6 (15.9–20.4) | 14.4 (14.3–16.2) | 16.5 (15.0–20.2) |
Height, cm | ||||
Median (Q1–Q3) | 168.8 (157.5–171.7) | 135.0 (129.7–141.7) | 109.7 (99.6–111.6) | 134.5 (111.4–155.0) |
Disease duration (years) | ||||
Median (Q1–Q3) | 1.2 (0.7–1.7) | 0.5 (0.3–2.0) | 2.8 (1.3–3.1) | 1.4 (0.6–2.8) |
JIA categories
| ||||
Polyarthritis (RF-)b, n (%) | 6 (75.0) | 7 (77.8) | 9 (100.0) | 22 (84.6) |
Psoriatic arthritis, n (%) | 1 (12.5) | 1 (11.1) | 0 | 2 (7.7) |
Extended oligoarthritis, n (%) | 1 (12.5) | 1 (11.1) | 0 | 2 (7.7) |
Disease characteristics
| ||||
Physician’s global assessment of overall disease activityc
| ||||
Median (Q1–Q3) | 7.5 (6.3–8.0) | 8.0 (6.0–8.5) | 6.5 (6.0–7.5) | 7.3 (6.0–8.0) |
Number of joints with active arthritisc,d
| ||||
Median (Q1–Q3) | 15.5 (13.0–18.0) | 17.0 (10.0–21.0) | 10.0 (8.0–20.0) | 14.0 (8.0–21.0) |
Number of joints with limitation of motionc
| ||||
Median (Q1–Q3) | 14.0 (8.5–16.5) | 9.0 (8.0–15.0) | 8.0 (6.0–20.0) | 10.0 (7.0–17.0) |
CHAQ functional ability | ||||
Median (Q1–Q3) | NA | 1.0 (0.4–1.6) | 0.9 (0.6–1.5) | 0.9 (0.4–1.6) |
C-reactive protein, mg/Le
| ||||
Median (Q1–Q3) | 0.3 (0.2–0.5) | 0.6 (0.4–2.3) | 2.2 (0.3–13.9) | 0.5 (0.2–5.2) |
Concomitant drugs
| ||||
Methotrexate use, mg/week | ||||
n (%) | 1 (12.5%) | 0 | 8 (88.9%) | 9 (34.6%) |
Median dose (Q1–Q3) | 25.0 (25.0–25.0) | 10.0 (9.4–11.9) | 10.0 (10.0–13.7) | |
Prednisone use, mg/day | ||||
n (%) | 2 (22.2%) | 2 (7.7%) | ||
Median dose (Q1–Q3) | 0 | 0 | 3.5 (2.0–5.0) | 3.5 (2.0–5.0) |
Summary of treatment administration
Parameter | Cohort 1 (12 to < 18 years)
N = 8a
| Cohort 2 (6 to < 12 years)
N = 9b
| Cohort 3 (2 to < 6 years)
N = 9 | All patients (2 to < 18 years)
N = 26c
|
---|---|---|---|---|
Dose, median (range), mg BID | 5.0 (3.0–5.0) | 2.5 (2.0–5.0) | 3.0 (2.5–3.5) | 3.0 (2.0–5.0) |
AUCtau, geometric mean (%CV),d ng•h/mL | 156.6 (25) | 118.8 (27) | 142.5 (32) | 138.6 (30) |
Cmax, geometric mean (%CV),d ng/mL | 47.0 (40) | 41.7 (29) | 66.2 (28) | 50.7 (38) |
Tmax, median (range), h | 0.8 (0.5–6.9) | 1.0 (0.5–2.1) | 0.5 (0.5–1.9) | 0.9 (0.5–6.9) |
Ctrough, geometric mean, (%CV),d ng/mL | 2.7 (100) | 0.8 (127) | 0.8 (119) | 1.1 (145) |
Cmin, geometric mean, (%CV),d ng/mL | 2.5 (86) | 0.8 (95) | 0.7 (103) | 1.1 (123) |
t½, arithmetic mean (SD), h | 2.6 ± 0.5 | 1.9 ± 0.3 | 1.8 ± 0.4 | 2.1 ± 0.5 |
CL/F, geometric mean, (%CV),d L/h | 28.1 (22) | 25.5 (40) | 20.5 (33) | 24.3 (34) |
Vz/F, geometric mean, (%CV),d L | 104.9 (35) | 71.0 (40) | 51.4 (34) | 70.5 (47) |
Pharmacokinetics
Safety
Taste evaluation
Taste assessment categories, n (%) | ||||||
---|---|---|---|---|---|---|
Day | Dislike very much | Dislike a little | Not sure | Like a little | Like very much | |
Cohort 1 (12 to < 18 years) N = 2 | 1 | 0 | 0 | 1 (50.0) | 1 (50.0) | 0 |
5 | 0 | 1 (50.0) | 1 (50.0) | 0 | 0 | |
Cohort 2 (6 to < 12 years) N = 7 | 1 | 1 (14.3) | 0 | 1 (14.3) | 3 (42.9) | 2 (28.6) |
5 | 1 (14.3) | 0 | 2 (28.6) | 2 (28.6) | 2 (28.6) | |
Cohort 3 (2 to < 6 years) N = 9 | 1 | 1 (11.1) | 2 (22.2) | 1 (11.1) | 1 (11.1) | 4 (44.4) |
5 | 0 | 3 (33.3) | 1 (11.1) | 2 (22.2) | 3 (33.3) |