Phase 3
N=100
A Safety, Efficacy And Pharmacokinetics Study Of Tofacitinib In Pediatric Patients With sJIA
Arthritis Juvenile Idiopathic
Bottom Line
View on ClinicalTrials.gov: NCT03000439 ↗Enrolled (actual)
100
Serious AEs
4.2%
Results posted
Jun 2025
Primary outcome: Primary: Time to Systemic Juvenile Idiopathic Arthritis (sJIA) Disease Flare: Double-Blind Phase — NA; 295.0 Days — p== 0.1171
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- In open-label phase: treatment with tofacitinib (Drug); In double-blind phase: treatment with tofacitinib or placebo in 1:1 ratio (Drug)
- Age
- Pediatric · 2+ yrs
- Sex
- All
- Sponsor
- Pfizer
- Primary completion
- Mar 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Time to Systemic Juvenile Idiopathic Arthritis (sJIA) Disease Flare: Double-Blind Phase |
NA; 295.0 | = 0.1171 |
| SECONDARY Probability of Occurrence of sJIA Disease Flare at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48 and 52: Double-Blind Phase |
7.1; 6.5; 14.3; 22.6; 21.4; 32.3 | — |
| SECONDARY Percentage of Participants Who Achieved Successful Corticosteroid Tapering: at the End of Open-label Phase Part 2 |
70.37 | — |
| SECONDARY Percentage of Participants Who Achieved Corticosteroid Dose of <= 0.2 mg/kg/Day or 10 mg/Day: at the End of Open-label Phase Part 2 |
59.26 | — |
| SECONDARY Percentage of Participants With Adapted JIA ACR 30/50/70/90/100 Response at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase |
82.14; 93.55; 82.14; 80.65; 71.43; 70.97 | — |
| SECONDARY Percentage of Participants With Adapted JIA American College of Rheumatology (ACR) 30/50/70/90/100 Response at Part 1 Day 7, Weeks 2, 4, 8, 12, and 16: Open-Label Phase Part 1 |
26.26; 42.42; 68.04; 86.59; 90.91; 83.33 | — |
| SECONDARY Percentage of Participants With Adapted JIA ACR 30/50/70/90/100 Response at Part 2 Weeks 4, 8, 12, 16, 20 and 24: Open-label Phase Part 2 |
90.00; 87.50; 90.32; 86.96; 100; 100 | — |
| SECONDARY Percentage of Participants With Fever Attributed to sJIA at Part 1 Days 3, 7 and 14: Open-Label Phase Part 1 |
3.13; 1.03; 4.12 | — |
| SECONDARY Percentage of Participants With C-Reactive Protein (CRP) <= 10 mg/L at Baseline, Part 1 Days 3, 7, Weeks 2, 4, 8, 12, 16: Open-Label Phase Part 1 |
30.00; 32.63; 45.36; 49.48; 55.67; 60.00 | — |
| SECONDARY Percentage of Participants With C-Reactive Protein (CRP) <= 10 mg/L at Part 2 Weeks 4, 8, 12, 16, 20, 24: Open-label Phase Part 2 |
68.00; 62.50; 51.61; 54.55; 76.92; 33.33 | — |
| SECONDARY Percentage of Participants With Absence of Fever Due to sJIA at Part 1 Day 7, Weeks 2, 4, 8, 12, 16: Open-label Phase Part 1 |
83.51; 82.47; 90.72; 96.39; 100; 95.83 | — |
| SECONDARY Percentage of Participants With Absence of Fever Due to sJIA at Part 2 Weeks 4, 8, 12, 16, 20, 24: Open-Label Phase Part 2 |
98.00; 92.31; 96.77; 90.91; 100; 100 | — |
| SECONDARY Percentage of Participants With Absence of Fever Due to sJIA at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48 and 52: Double Blind Phase |
85.71; 93.55; 85.71; 80.65; 75.00; 74.19 | — |
| SECONDARY Time to First Adapted JIA ACR 30 Response: Open-label Phase Part 1 |
27.0 | — |
| SECONDARY Change From Open Label Baseline in Juvenile Arthritis Disease Activity Score (JADAS-27) Erythrocyte Sedimentation Rate (ESR) at Part 1 Day 7, Part 1 Weeks 2, 4, 8, 12 and 16: Open-Label Phase Part 1 |
-5.64; -8.57; -11.95; -15.09; -15.77; -14.08 | — |
| SECONDARY Change From Open-Label Baseline in JADAS-27 CRP at Part 1 Day 7, Part 1 Weeks 2, 4, 8, 12 and 16: Open-Label Phase Part 1 |
-6.25; -8.55; -11.88; -15.07; -15.19; -13.10 | — |
| SECONDARY Change From Open-Label Baseline in JADAS-27 ESR at Part 2 Weeks 4, 8, 12, 16, 20, 24: Open-Label Phase Part 2 |
-17.05; -17.13; -18.17; -18.89; -21.14; -22.17 | — |
| SECONDARY Change From Open-Label Baseline in JADAS-27 CRP at Part 2 Weeks 4, 8, 12, 16, 20, 24: Open-Label Phase Part 2 |
-17.16; -17.44; -18.99; -20.31; -22.16; -25.61 | — |
| SECONDARY Change From Double-Blind Baseline in JADAS-27 ESR at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase |
1.07; 1.08; 1.75; 0.81; 1.47; 1.71 | — |
| SECONDARY Change From Double-Blind Baseline in JADAS-27 CRP at Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase |
1.62; 0.71; 2.12; 0.01; 1.78; 1.80 | — |
| SECONDARY Change From Open-Label Baseline in Number of Joints With Active Arthritis at Part 1 Day 7, Weeks 2, 4, 8, 12, 16: Open-Label Phase Part 1 |
-2.96; -4.21; -6.03; -7.80; -7.52; -6.38 | — |
| SECONDARY Change From Open-Label Baseline in Number of Joints With Active Arthritis at Part 2 Weeks 4, 8, 12, 16, 20, 24: Open-Label Phase Part 2 |
-7.78; -7.73; -9.26; -10.00; -10.85; -13.67 | — |
| SECONDARY Change From Open-Label Baseline in Number of Joints With Limited Range of Motion at Part 1 Day 7, Weeks 2, 4, 8, 12, 16: Open-Label Phase Part 1 |
-1.83; -2.06; -2.90; -4.36; -3.64; -2.33 | — |
| SECONDARY Change From Open-Label Baseline in Number of Joints With Limited Range of Motion at Part 2 Weeks 4, 8, 12, 16, 20, 24: Open-Label Phase Part 2 |
-4.26; -3.90; -5.81; -5.74; -5.92; -15.67 | — |
| SECONDARY Change From Open-Label Baseline in Physician Global Evaluation of Disease Activity at Part 1 Day 7, Weeks 2, 4, 8, 12, 16: Open-label Phase Part 1 |
-1.18; -1.82; -2.62; -3.40; -3.66; -3.50 | — |
| SECONDARY Change From Open-Label Baseline in Physician Global Evaluation of Disease Activity at Part 2 Weeks 4, 8, 12, 16, 20, 24: Open-Label Phase Part 2 |
-4.54; -4.53; -4.65; -4.89; -5.62; -6.17 | — |
| SECONDARY Change From Open-Label Baseline in ESR at Part 1 Day 7, Weeks 2, 4, 8, 12, 16: Open-Label Phase Part 1 |
-11.00; -18.60; -24.70; -30.59; -31.74; -27.65 | — |
| SECONDARY Change From Open-Label Baseline in ESR at Part 2 Weeks 4, 8, 12, 16, 20, 24: Open-Label Phase Part 2 |
-33.64; -30.73; -27.52; -23.48; -37.54; -24.33 | — |
| SECONDARY Change From Open-Label Baseline in CHAQ- Parental Evaluation of Overall Well-being at Part 1 Days 3, 7, Part 1 Weeks 2, 4, 8, 12, 16: Open-Label Phase Part 1 |
-0.73; -1.36; -1.96; -2.65; -3.35; -3.45 | — |
| SECONDARY Change From Open-Label Baseline in CHAQ - Parental Evaluation of Overall Well-being at Part 2 Weeks 4, 8, 12, 16, 20, 24: Open-Label Phase Part 2 |
-4.09; -4.50; -4.73; -5.11; -5.65; -6.33 | — |
| SECONDARY Change From Open-Label Baseline in CHAQ - Disability Index at Part 1 Day 7, Weeks 2, 4, 8, 12, 16: Open-Label Phase Part 1 |
-0.21; -0.38; -0.52; -0.59; -0.51; -0.74 | — |
| SECONDARY Change From Open-Label Baseline in CHAQ-Disability Index at Part 2 Weeks 4, 8, 12, 16, 20, 24: Open-Label Phase Part 2 |
-0.79; -0.78; -0.85; -0.67; -0.93; -0.96 | — |
| SECONDARY Change From Open-Label Baseline in Number of Joints With Active Arthritis at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase |
-6.53; -7.39; -6.36; -7.69; -5.92; -7.36 | — |
| SECONDARY Change From Double-Blind Baseline in Number of Joints With Active Arthritis at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase |
0.38; 0.09; 0.78; -0.23; 1.47; 0.22 | — |
| SECONDARY Change From Open-Label Baseline in CHAQ-Disability Index at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase |
-0.81; -1.03; -0.93; -1.02; -0.99; -1.04 | — |
| SECONDARY Change From Double Blind Baseline in CHAQ-Disability Index at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase |
0.04; -0.02; -0.03; -0.01; -0.06; -0.05 | — |
| SECONDARY Change From Open-Label Baseline in Number of Joints With Limited Range of Motion at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase |
-2.47; -2.69; -2.31; -2.89; -2.09; -3.29 | — |
| SECONDARY Change From Double-Blind Baseline in Number of Joints With Limited Range of Motion at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase |
0.08; 0.21; 0.45; 0.06; 0.48; -0.23 | — |
| SECONDARY Change From Open-Label Baseline in Physician Global Evaluation of Disease Activity at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase |
-4.22; -5.22; -4.23; -5.55; -4.89; -5.42 | — |
| SECONDARY Change From Double-Blind Baseline in Physician Global Evaluation of Disease Activity at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase |
0.16; 0.21; 0.45; -0.14; 0.10; -0.02 | — |
| SECONDARY Change From Open-Label Baseline in ESR at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase |
-32.65; -31.19; -36.55; -29.32; -38.30; -31.69 | — |
| SECONDARY Change From Double-Blind Baseline in ESR at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase |
3.35; 9.41; 1.94; 12.39; 1.42; 12.28 | — |
| SECONDARY Change From Open-Label Baseline in CHAQ -Parental Evaluation of Overall Well-being at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase |
-4.31; -5.52; -4.56; -5.38; -4.97; -5.28 | — |
| SECONDARY Change From Double-Blind Baseline in CHAQ -Parental Evaluation of Overall Well-being at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase |
0.46; -0.03; 0.60; 0.12; 0.25; 0.22 | — |
| SECONDARY Change From Open-Label Baseline in Child Health Questionnaire (CHQ) Responses at End of Open-Label Phase Part 1 |
26.12; 28.95; 27.51; 31.15; 32.00; 10.98 | — |
| SECONDARY Change From Open-Label Baseline in CHQ Responses at End of Open-Label Phase Part 2 |
25.52; 33.33; 28.17; 38.10; 34.48; 13.02 | — |
| SECONDARY Change From Double-Blind Baseline in CHQ Responses at DB Weeks 24, 48: Double-Blind Phase |
4.02; -1.98; 4.09; 1.24; 2.51; 6.70 | — |
| SECONDARY Change From Open-Label Baseline in CHAQ-Discomfort Index at Part 1 Day 7, Weeks 2, 4, 8, 12, 16: Open-Label Phase Part 1 |
-1.20; -1.87; -2.57; -3.30; -3.57; -3.69 | — |
| SECONDARY Change From Open-label Baseline in CHAQ-Discomfort Index at Part 2 Weeks 4, 8, 12, 16, 20, 24: Open-Label Phase Part 2 |
-4.23; -4.38; -4.42; -4.48; -5.15; -6.00 | — |
| SECONDARY Change From Double-Blind Baseline in CHAQ-Discomfort Index at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase |
0.13; 0.37; 0.56; 0.02; 0.04; 0.09 | — |
| SECONDARY Percentage of Participants With Minimum Disease Activity Calculated From JADAS-27 CRP Score at Part 1 Day 7, Weeks 2, 4, 8, 12 and 16: Open-Label Phase Part 1 |
3.09; 5.21; 6.19; 13.75; 13.64; 25.00 | — |
| SECONDARY Percentage of Participants With Minimum Disease Activity Calculated From JADAS-27 ESR Score at Part 1 Day 7, Weeks 2, 4, 8, 12 and 16: Open-label Phase Part 1 |
1.02; 4.08; 7.22; 14.81; 15.91; 20.83 | — |
| SECONDARY Percentage of Participants With Minimum Disease Activity Calculated From JADAS-27 CRP Score at Part 2 Weeks 4, 8, 12, 16, 20, 24: Open-Label Phase Part 2 |
36.00; 30.00; 32.26; 36.36; 53.85; 33.33 | — |
| SECONDARY Percentage of Participants With Minimum Disease Activity Calculated From JADAS-27 ESR Score at Part 2 Weeks 4, 8, 12, 16, 20, 24: Open-Label Phase Part 2 |
32.00; 27.50; 29.03; 34.78; 53.85; 33.33 | — |
| SECONDARY Percentage of Participants With Inactive Disease Status Calculated From JADAS-27 CRP Score at Part 1 Day 7, Weeks 2, 4, 8, 12 and 16: Open-label Phase Part 1 |
0; 3.13; 3.09; 5.00; 4.55; 4.17 | — |
| SECONDARY Percentage of Participants With Inactive Disease Status Calculated From JADAS-27 ESR Score at Part 1 Day 7, Weeks 2, 4, 8, 12 and 16: Open-Label Phase Part 1 |
0; 2.04; 1.03; 3.70; 6.82; 4.17 | — |
| SECONDARY Percentage of Participants With Inactive Disease Status Calculated From JADAS-27 CRP Score at Part 2 Weeks 4, 8, 12, 16, 20, 24: Open-Label Phase Part 2 |
16.00; 20.00; 9.68; 18.18; 38.46; 33.33 | — |
| SECONDARY Percentage of Participants With Inactive Disease Status Calculated From JADAS-27 ESR Score at Part 2 Weeks 4, 8, 12, 16, 20, 24: Open-Label Phase Part 2 |
16.00; 17.50; 9.68; 17.39; 46.15; 33.33 | — |
| SECONDARY Percentage of Participants With Minimum Disease Activity Calculated From JADAS-27 CRP Score at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase |
21.43; 51.61; 25.00; 48.39; 32.14; 32.26 | — |
| SECONDARY Percentage of Participants With Minimum Disease Activity Calculated From JADAS-27 ESR Score at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase |
25.00; 51.61; 25.00; 45.16; 32.14; 41.94 | — |
| SECONDARY Percentage of Participants With Inactive Disease Status Calculated From JADAS-27 CRP Score at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-blind Phase |
10.71; 25.81; 7.14; 19.35; 7.14; 16.13 | — |
| SECONDARY Percentage of Participants With Inactive Disease Status Calculated From JADAS-27 ESR Score at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase |
7.14; 19.35; 14.29; 29.03; 14.29; 22.58 | — |
| SECONDARY Percentage of Participants With JIA ACR Inactive Disease Status at Part 1 Days 3, 7, Weeks 2, 4, 8, 12, 16: Open-Label Phase Part 1 |
0; 0; 0; 2.02; 3.09; 4.82 | — |
| SECONDARY Percentage of Participants With JIA ACR Inactive Disease Status at Part 2 Weeks 4, 8, 12, 16, 20, 24: Open-Label Phase Part 2 |
14.00; 10.26; 6.45; 13.04; 15.38; 33.33 | — |
| SECONDARY Percentage of Participants With JIA ACR Inactive Disease Status at DB Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52: Double-Blind Phase |
7.14; 16.13; 3.57; 22.58; 7.14; 22.58 | — |
Summary
A randomized withdrawal study in which responders to open-label treatment with tofacitinib will be randomized in a 1:1 ratio to tofacitinib or placebo in a double-blind phase. In the double-blind phase "time to sJIA flare" will be evaluated as primary endpoint and subjects will be discontinued once they experience sJIA flare. An interim analysis for efficacy and futility will be conducted when at least 20 flares have been observed. If either criterion is met, the study will be stopped. If neither criterion is met, the study will continue until the requisite number of flares are observed as determined by the number of flares included in the interim analysis and a statistical penalty for conducting the interim analysis.
Eligibility Criteria
Inclusion Criteria
- active sJIA disease according to ILAR criteria before screening and at baseline (Day 1);
- Treatment with stable doses of methotrexate (MTX) ≤25 mg/week or ≤20 mg/m2/week, whichever is lower, is permitted;
- Treatment with a stable dose of oral prednisone ≤1 mg/kg/day up to a maximum of 30 mg/day, or equivalent, for at least 1 week before the first study drug dose is permitted.
Exclusion Criteria
- Previous juvenile idiopathic arthritis (JIA) treatment with tofacitinib.
- Current symptoms or findings of myocarditis, endocarditis or more than minimal pericardial effusion associated with systemic juvenile idiopathic arthritis (sJIA). Current symptoms or findings of more than minimal pleuritis with sJIA.
- Current infection or serious infection within 3 months of study enrollment.
Data sourced from ClinicalTrials.gov (NCT03000439). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.