Trial results for a long-term safety study of tofacitinib (NCT01500551) in patients with juvenile idiopathic arthritis were posted on ClinicalTrials.gov on 2026-03-23. The study, which enrolled 302 participants, reported that 294 experienced laboratory test abnormalities and 268 had treatment-emergent adverse events (TEAEs).
Background
The study aimed to evaluate the long-term safety and tolerability of tofacitinib in patients with juvenile idiopathic arthritis (JIA) who had previously participated in other tofacitinib JIA studies.
Trial design
The study (NCT01500551) was a Phase 2/Phase 3, open-label, long-term safety study that enrolled 302 participants with juvenile idiopathic arthritis. All participants received tofacitinib 5 mg BID. The trial's primary objective was to evaluate the long-term safety and tolerability of tofacitinib in this patient population.
Key results
The long-term safety and tolerability outcomes for the tofacitinib 5 mg BID (Open-label) group were reported as follows:
- The number of participants with laboratory test abnormalities was 294.
- The number of participants with treatment-emergent adverse events (TEAEs) was 268.
Body weight measurements were also collected over the study period:
- Absolute body weight at baseline was a mean of 41.60 Kilograms (Standard Deviation: 19.35).
- At Month 12, absolute body weight was a mean of 49.16 Kilograms (Standard Deviation: 20.59), with a mean change from baseline of 6.52 Kilograms (Standard Deviation: 6.71).
- At Month 24, absolute body weight was a mean of 52.42 Kilograms (Standard Deviation: 20.15), with a mean change from baseline of 9.42 Kilograms (Standard Deviation: 8.08).
- At Month 36, absolute body weight was a mean of 56.96 Kilograms (Standard Deviation: 19.96), with a mean change from baseline of 12.44 Kilograms (Standard Deviation: 10.34).
- At Month 48, absolute body weight was a mean of 56.72 Kilograms (Standard Deviation: 17.69), with a mean change from baseline of 13.99 Kilograms (Standard Deviation: 11.06).
- At Month 60, absolute body weight was a mean of 58.07 Kilograms (Standard Deviation: 18.28).
What this means
The results from this long-term safety study provide important data on the safety and tolerability profile of tofacitinib in pediatric patients with juvenile idiopathic arthritis. The reported numbers of participants experiencing laboratory test abnormalities and treatment-emergent adverse events highlight the need for continued monitoring and careful consideration of the risk-benefit profile when using tofacitinib in this population. The observed increases in body weight over time are consistent with growth in a pediatric cohort.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT01500551, titled "Long-Term Safety Study Of Tofacitinib In Patients With Juvenile Idiopathic Arthritis," were posted on 2026-03-23 on clinicaltrials.gov.