overview of paediatric investigation plans for uveitis · infectious uveitis in children from 6...
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An agency of the European Union
Richard Veselý, scientific coordinator,Paediatric medicinesHuman Medicines Special AreasEuropean Medicines Agency
Overview of Paediatric Investigation Plans for uveitis
© European Medicines Agency, 2011. Reproduction is authorised provided the source is acknowledged.
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Voclosporin
Active substance(s):
Voclosporin
Condition(s):
Chronic non-infectious uveitis
Pharmaceutical form(s):
Soft capsules
Oral liquid
Route(s) of administration:
Oral use
Name/corporate name of the PIP applicant:
Lux Biosciences GmbH
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Voclosporin - agreed measures
Development of an oral liquid formulation Juvenile animal toxicity study Open-label, multi-centre, dose-titration study of voclosporin for the treatment of non-infectious uveitis in children from 6 years to less than 18 years of age. Open-label, multi-centre study to evaluate safety and efficacy of voclosporin for the treatment of non-infectious uveitis in children from 6 years to less than 18 years of age. Multi-centre study to assess the safety and efficacy of voclosporin in the treatment of non-infectious uveitis in children from 2 to less than 6 years of age.
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AIN457
Active substance(s):
Recombinant human monoclonal antibody to human Interleukin 17A (AIN457)
Condition(s):
Treatment of chronic non-infectious uveitis
Pharmaceutical form(s):
Powder for solution for injection
Route(s) of administration:
Subcutaneous use
Name/corporate name of the PIP applicant:
Novartis Europharm Limited
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AIN457 – agreed measure
A double-blind, randomised, placebo-controlled, multicentre, dose
ranging trial with AIN457 in paediatric population with active chronic
non-infectious uveitis
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JIA uveitis
• No separate studies agreed yet for biologics
• Uveitis exclusion from JIA studies?
• Conclusion of paediatric rheumatology expert meetings (no exclusion)
• Inclusion into JIA studies or separate studies for JIA uveitis?
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Treatment response -activity of uveitis
Inactive
Grade 0 cells
Worsening activity
Two-step increase in level of inflammation (e.g. anterior chamber cells, vitreous haze) or increase from grade 3+ to 4+
Improved activity
Two-step decrease in level of inflammation (e.g. anterior chamber cells, vitreous haze) or decrease to grade 0
Remission
Inactive disease for >3 months after discontinuing all treatments for eye disease
(Standardisation of uveitis nomenclature, SUN, 2005)
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Potential endpoints for clinical studies
• Change in either anterior chamber cells or vitreous haze
• Achieving quiescence (cell grade or haze)
• Combination of achieving quiescence, visual acuity change and change in concomitant medication
• Combination of SUN criteria, concomitant medication and qualityof life
• In JIA combination of local findings with JIA core set criteria