A Safety, Efficacy And Pharmacokinetics Study Of Tofacitinib In Pediatric Patients With sJIA

Pfizer

Status and phase: Completed; Phase 3

Conditions: Arthritis Juvenile Idiopathic

Treatments: Drug: In double-blind phase: treatment with tofacitinib or placebo in 1:1 ratio; Drug: In open-label phase: treatment with tofacitinib

Study type: Interventional

Funder types: Industry

Identifiers

NCT03000439

A3921165

2017-002018-29 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 100 patients

Sex: All

Ages: 2 to 17 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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100 participants in 2 patient groups, including a placebo group

Tofacitinib 5 mg BID

Experimental group

Description:

oral, twice daily, tablet or solution.

Treatment:

Drug: In double-blind phase: treatment with tofacitinib or placebo in 1:1 ratio

Drug: In open-label phase: treatment with tofacitinib

Placebo

Placebo Comparator group

Treatment:

Drug: In double-blind phase: treatment with tofacitinib or placebo in 1:1 ratio