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A phase II trial protocol of Tocilizumab in anti-TNF refractory patients with JIA-associated uveitis (the APTITUDE
Athimalaipet V Ramanan1, Andrew D Dick2,3, Ashley P Jones4
11University Hospitals Bristol NHS Foundation Trust & Bristol Medical School, University of Bristol, Bristol, UK.
Insights
This study investigates Tocilizumab for treating severe JIA-associated uveitis in children unresponsive to anti-TNF agents. It assesses the drug's effectiveness and safety, offering hope for improved ocular outcomes.
Area of Science:
- Pediatric Rheumatology
- Ophthalmology
- Immunology
Background:
- Juvenile idiopathic arthritis (JIA) is a common childhood rheumatic disease.
- Children with JIA are at risk of developing uveitis, which can be asymptomatic initially.
- Current treatments like methotrexate and anti-TNF agents are not effective for all severe cases, necessitating alternative therapies.
Purpose of the Study:
- To evaluate the efficacy and safety of Tocilizumab in children with JIA-associated uveitis refractory to anti-tumour necrosis factor (anti-TNF) agents.
- To explore the potential of anti-interleukin-6 (IL-6) agents as an alternative therapy for severe refractory uveitis.
Main Methods:
- A two-stage Simon design clinical trial involving at least 22 patients aged 2-18 years with active JIA-associated uveitis.
- Participants received a stable dose of methotrexate plus Tocilizumab (162 mg every 2 or 3 weeks based on weight) for 6 months.
- The primary outcome was treatment response at 12 weeks, with adverse events monitored.
Main Results:
- This section is not detailed in the provided abstract.
- Further research is needed to determine the role of IL-6 inhibition in managing severe refractory JIA-associated uveitis.
Conclusions:
- This is a novel adaptive design study assessing subcutaneous IL-6 inhibition in anti-TNF refractory JIA-associated uveitis.
- It is the first pediatric clinical trial to evaluate the efficacy and safety of Tocilizumab with methotrexate in JIA-associated uveitis.
- The study aims to determine if further research into Tocilizumab for uveitis is warranted.
Background:
Juvenile idiopathic arthritis (JIA) is the most common rheumatic disease in children. Children with JIA are at risk of intraocular inflammation (uveitis). In the initial stages of mild-moderate inflammation uveitis is asymptomatic. Most children with mild-moderate uveitis are managed on topical steroid drops with or without systemic methotrexate (MTX). When children with moderate-severe uveitis are refractory to MTX, monoclonal anti-tumour necrosis factor agents have been trialled, interim analysis data showed positive results. However, several children with severe recalcitrant disease or non-responsive to anti-tumour necrosis factor agents remain and are at greater risk of significant ocular complications and visual loss. Further evidence of alternative therapies is needed with evidence of a potential role of anti-interleukin-6 agents in the management of severe refractory uveitis.
Methods:
The trial will be conducted following a two-stage Simon design. The trial will register at least 22 patients aged 2 to 18 years with active JIA-associated uveitis, who have taken MTX for at least 12 weeks and have failed an anti-TNF agent. It will take place in 7 centres across the UK. All participants will be treated for 6 months, with follow up of 9 months from registration. Participants will receive a stable dose of MTX and those weighing ≥30 kg will be dosed with 162 mg of Tocilizumab every 2 weeks and participants weighing < 30 kg dosed with 162 mg of Tocilizumab every 3 weeks. Primary outcome is treatment response at 12 weeks. Adverse events will be collected up to 30 calendar days following treatment cessation.
Discussion:
This is a novel adaptive design study of subcutaneous IL-6 inhibition in anti-TNF refractory JIA associated uveitis which will be able to determine if further research should be conducted. This is the first trial to look at ophthalmology outcomes in the efficacy of Tocilizumab in uveitis.This is the first paediatric clinical trial to assess the clinical effectiveness and safety of tocilizumab with MTX in JIA associated uveitis.
Trials Registration:
The Trial is registered on the ISRCTN registry (ISRCTN95363507) on the 10/06/2015 and EU Clinical Trials Register on the 03/07/2015 (EudraCT Number: 2015-001323-23).
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