Efficacy Study Of Tofacitinib In Pediatric JIA Population

Summary

Evaluate efficacy, safety and tolerability of tofacitinib in pediatric JIA patients.

Timeline

Start

2016-06-10

Primary completion

2019-05-16

Completion

2019-05-16

Outcome

Met primary endpoint

paper Flare rate by week 44 was significantly lower with tofacitinib (21 [29%] of 72 patients) than with placebo (37 [53%] of 70 patients; PMID 34767764 ↗

registry analysis (superiority test); Tofacitinib: Double Blind Phase vs Placebo; p = 0.0031; Difference in percentage -23.69 (95% CI -39.41 to -7.97) NCT02592434 ↗

release “The trial met its primary endpoint showing that in patients with polyarticular JIA, the occurrence of disease flare” pfizer.com ↗

release “The trial met its primary endpoint showing that in patients with pa-JIA, the occurrence of disease flare in patients treated with tofacitinib was significantly lower” pfizer.com ↗

release “The trial met its primary endpoint showing that in patients with pcJIA” pfizer.com ↗

release “The trial met its primary endpoint showing that in patients with polyarticular JIA who achieved a JIA American College of Rheumatology (ACR) 30 response” pfizer.com ↗

Publications

• Consolaro A, Ruperto N, Lovell DJ, Synoverska O, Abud-Mendoza C, Spindler A, Vyzhga Y, Alexeeva E, Chaitow J, Chiraseveenuprapund P, Lazariciu I, Stockert L, Cadatal MJ, Diehl A, Brunner HI; Paediatric Rheumatology International Trials Organisation (PRINTO) and the Pediatric Rheumatology Collaborative Study Group (PRCSG). Clinically Inactive Disease and Remission in Patients With Juvenile Idiopathic Arthritis Receiving Tofacitinib: Post Hoc Analysis of a Phase III Trial. J Rheumatol. 2025 Sep 1;52(9):919-926. doi: 10.3899/jrheum.2024-0536.
• Chang C, Vong C, Wang X, Hazra A, Diehl A, Nicholas T, Mukherjee A. Tofacitinib pharmacokinetics in children and adolescents with juvenile idiopathic arthritis. CPT Pharmacometrics Syst Pharmacol. 2024 Apr;13(4):599-611. doi: 10.1002/psp4.13104. Epub 2024 Jan 31.
• Ruperto N, Brunner HI, Synoverska O, Ting TV, Mendoza CA, Spindler A, Vyzhga Y, Marzan K, Grebenkina L, Tirosh I, Imundo L, Jerath R, Kingsbury DJ, Sozeri B, Vora SS, Prahalad S, Zholobova E, Butbul Aviel Y, Chasnyk V, Lerman M, Nanda K, Schmeling H, Tory H, Uziel Y, Viola DO, Posner HB, Kanik KS, Wouters A, Chang C, Zhang R, Lazariciu I, Hsu MA, Suehiro RM, Martini A, Lovell DJ; Paediatric Rheumatology International Trials Organisation (PRINTO) and Pediatric Rheumatology Collaborative Study Group (PRCSG). Tofacitinib in juvenile idiopathic arthritis: a double-blind, placebo-controlled, withdrawal phase 3 randomised trial. Lancet. 2021 Nov 27;398(10315):1984-1996. doi: 10.1016/S0140-6736(21)01255-1. Epub 2021 Nov 9.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Tofacitinib	Small molecule	2 mg	Oral
Subject	Tofacitinib	Small molecule	2.5 mg	Oral
Subject	Tofacitinib	Small molecule	3 mg	Oral
Subject	Tofacitinib	Small molecule	3.5 mg	Oral
Subject	Tofacitinib	Small molecule	4 mg	Oral
Subject	Tofacitinib	Small molecule	5 mg	Oral

Indications