Golimumab in Juvenile Idiopathic Arthritis-associated Uveitis Failing Adalimumab
Conditions: JIA Associated Uveitis
Sponsor: Medical University of Graz

Study protocol Golimumab in JIA-associated uveits                                                         Version 2.0                                 
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Golimumab in juvenile idiopathic arthritis (JIA) - associated uveitis 
unresponsive to adalimumab    
 
NCT ID: 31-301ex18/19 
NCT04200833 
 
 

Study protocol Golimumab in JIA-associated uveits                                                         Version 2.0                                 
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Index 
1 
Background ........................................................................................................................ 3 
2 
Aim ..................................................................................................................................... 4 
The aim oft he study ist o evaluate the effectiveness of golimumab in patients with JIA 
associated Uveitis that experienced a treatment failure to ADA ............................................... 4 
3 
Patients ............................................................................................................................... 4 
4 
Outcome measures ............................................................................................................. 4 
4.1 Main outcome measures ................................................................................................... 4 
4.2 Secondary Outcomes ........................................................................................................ 5 
5 
Methods .............................................................................................................................. 5 
6 
Statistics ............................................................................................................................. 5 
6.1 Scheduled Evaluations ..................................................................................................... 5 
6.1 Sample size ....................................................................................................................... 6 
7 
Data protection ................................................................................................................... 6 
8 
Benefit-Risk ratio ............................................................................................................... 6 
9 
References .......................................................................................................................... 6 
 
 

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1 
Background 
 
 
Uveitis is a potentially blinding complication of juvenile idiopathic arthritis (JIA) [1–3]. 
Early and complete control of ocular inflammation in JIA is important. Treatment 
moves step-by step, starting with topical and/or systemic steroids and followed by 
conventional disease-modifying anti-rheumatic drugs (cDMARDs), usually 
methotrexate (MTX). In refractory cases treatment with anti-tumor necrosis factor 
(TNF)-α agents is recommended [4, 5]. Among these adalimumab (ADA), a fully 
humanised antibody against TNF-α and the only TNF-α inhibitor approved for uveitis 
as of 2021, has become the drug of choice. ADA shows efficacy in around 75% of 
patients in the first 18 months of treatment [6, 7]. In case of no response or loss of 
response to ADA, a switch to another biological agent is recommended [5, 8, 9]. 
However, the choice of biological drug is not clearly defined. The question remains: 
Is persistence in an attack on the TNF- α pathway promising in these patients? In this 
regard data from small numbers of patients provide evidence that switching to a 
second anti-TNF agent may be beneficial [8, 10]. As such golimumab (GLM), another 
fully humanised anti-TNF-α monoclonal antibody approved for the treatment of 
polyarticular JIA, has shown promising results in small heterogeneous case series 
[11–13]. 
 
Abbreviations:  
• JIA 
  Juvenile idiopathic arthritis 
• Anti-TNF  anti- tumor necrosis factor 
• ADA         Adalimumab 
• GLM        Golimumab 
• SUN      Standardization of Uveitis Nomenclature    
 

Study protocol Golimumab in JIA-associated uveits                                                         Version 2.0                                 
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2 
Aim 
 The aim oft he study ist o evaluate the effectiveness of golimumab in patients with 
JIA associated Uveitis that experienced a treatment failure to ADA 
. 
3 
Patients 
Patients that suffered a loss of response to ADA and were subsequently 
switched to GLM from November 2012 to February 2019 will be included. 
Patients diagnosed and treated in the department of pediatric rheumatology and 
the uveitis clinic of the Medical University of Graz are eligible. Due to the 
retrospective nature and the classification as orphan disease no sample size 
calculation is feasible; a sample size of around 10 patients is to be expected. 
Inclusion Criteria:  
• Patients diagnosed with JIA according tot he ILAR-criteria and associated 
Uveitis 
• Golimumab treatment following treatment with  
• Age between 1 and 25 years 
      Exclusion Criteria: 
• Patient not fulfilling diagnostic criteria of a JIA associated  
• Patients that were not previously treated with ADA 
 
4 
Outcome measures 
4.1 Main outcome measures  
 The main outcome measures are assessed at predefined time points  month 1, 
month 3 and every 3 months thereafter) including intraocular inflammation as 
determined by anterior chamber cell count, best-corrected visual acuity (BCVA), 
corticosteroid-sparing potential, and ocular complications. Results of slit-lamp 
examination, applanation tonometry, ophthalmoscopy, fluorescein angiography, and 
spectral-domain optical coherence tomography were evaluated. Anterior chamber 

Study protocol Golimumab in JIA-associated uveits                                                         Version 2.0                                 
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(AC) cells and vitreous haze (VH) are graded by SUN criteria [16]. BCVA is 
determined using a Snellen chart. 
 
4.2 Secondary Outcomes 
- Demographic data of patients 
- JIA-subtype  
- anatomically localization of uveitis 
- Uveitis complication 
- medication 
- opthalmological investigations, including visual acuity, intraocular 
pressure, fundoscopic findings  
- Laboratory parameters under treatment ( whole blood count, lever and 
kidney enzymes, CRP, autoimmune lab (ANA, ds-DNA,  
  anti-drug-Ab, RF, HLA-B27. 
 
5 
Methods 
A single-centre, retrospective study in patients with JIA-associated uveitis who were treated 
with GLM for active uveitis that had proved refractory to at least one cDMARD and to ADA. 
 
6 
Statistics 
6.1 Scheduled Evaluations 
Descriptive statistics will be used. Continuous data will be evaluated via median and 
quartiles or mean and standard deviation, with respect to the data distribiution. 
Categorical data will be given as absolute and relative proportions. Differences 
between groups will be evaluated with chi-square or fisher exact test. Time to 
treatment failure will be evaluated via Kaplan-Meier plots. 

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6.1 Sample size 
Due to scarce data (JIA associated uveitis is an orphan disease) no sample size 
calculation is feasible. A number of 10 patients ist o be expected. The nature oft he 
study is explorative. 
 
7 
Data protection 
All patients will be given a numerical code. The data files will be saved in a 
secure computer at the department of pediatrics and analyzed pseudo-
anonymously. 
8 
Benefit-Risk ratio 
Therapeutic options after treatment failure of ADA for JIA associated uveitis are 
limited and the risk of ocular complications is high. Thus, these patients are in 
high need for effective treatment options. The data of the present study can help 
guide physicians in their choice of treatment in such cases. 
A better informed treatment might reduce the burden of systemic steroids and 
reduce uveitis complications with their associated vision loss. In addition, data 
form this study might serve as a pilot for further studies. 
As this is a retrospective study no risk of harm to the study population is to be 
expected. All data have been collected in advance and patients have been 
treated according to international guidelines. Thus the risk-benefit ratio is to be 
judged favourably. 
9 
References 
 
1. 
Petty R.E, Southwood T.R., Baum, et al. Revision of the proposed 
classification 
           criteria for juvenile idiopathic arthritis: Durban,1997, 
J.Rheumatol.25(1998)199-1994 J 
 
2.         Heiligenhaus et al. Interdisziplinäre Leitlinie zur Diagnostik und 
antientzündlichen 

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            Therapie der Uveitis bei juveniler idiopathischer Arthritis. AWMF online 
1/2018 
 
3.        Palmou-Fontana et al. Golimumab in refractory uveitis associated to juvenile 
           idiopathic arthritis: multicentre study of 7 cases and literature review:, Clin Exp 
           Rheumatol. 2018 
 
4.         Cordero-Coma et al. Anti-tumort necrosis factor-α therapy in uveitis. 
Survophthal. 
            2015