Ophthalmology · Clinical trials
Uveitis Clinical Trials
Approved therapies, the pivotal-trial endpoints they were judged on, the magnitude of benefit — and the drugs that failed their endpoints, and why.
Indication overview
About uveitis — and why its trials are hard
Non-infectious uveitis is a group of immune-mediated intraocular inflammatory diseases affecting the uveal tract, classified anatomically as anterior, intermediate, posterior or panuveitis. Intermediate and posterior forms threaten sight through macular oedema, vasculitis, retinal damage and secondary glaucoma or cataract, and account for a substantial share of preventable blindness. Corticosteroids, systemic or local, remain first-line, but chronic use carries cataract, glaucoma and systemic toxicity, driving demand for steroid-sparing options. Adalimumab, an anti-TNF-alpha antibody, became the first biologic approved for non-infectious intermediate, posterior and panuveitis (2016) on the strength of the VISUAL-I and VISUAL-II trials, which measured time to treatment failure. Sustained-release corticosteroid implants provide durable local control: the dexamethasone implant (Ozurdex, HURON) for posterior segment inflammation and fluocinolone acetonide implants (Yutiq, Retisert) for chronic disease. Methotrexate, mycophenolate and other immunomodulators are used off-label. Trial endpoints centre on time to treatment failure, control of vitreous haze and inflammatory recurrence, reflecting the relapsing nature of the disease.
Approved therapies & pivotal evidence
What's been approved — and by how much it moved the endpoint
| Drug (brand) | Approved | Setting | Pivotal trial | Primary endpoint | Magnitude of benefit |
|---|---|---|---|---|---|
| adalimumab (Humira) | 2016 | Non-infectious intermediate, posterior and panuveitis | VISUAL-I (active disease) and VISUAL-II (inactive/controlled disease) | Time to treatment failure (composite of inflammation, vision and lesions) | VISUAL-I: median time to failure 24 vs 13 weeks (HR ~0.50); VISUAL-II: HR ~0.57, both favouring adalimumab |
| dexamethasone intravitreal implant (Ozurdex) | 2010 | Non-infectious posterior segment uveitis (corticosteroid) | HURON | Proportion with vitreous haze score of 0 at week 8 | ~47% reached vitreous haze 0 with the 0.7 mg implant vs ~12% sham |
| fluocinolone acetonide implant (Yutiq / Retisert) | 2018 | Chronic non-infectious posterior uveitis (sustained-release) | Yutiq phase 3 (0.18 mg) program; Retisert approved 2005 | Recurrence of uveitis over 6–12 months | Yutiq roughly halved recurrence vs sham over 12 months (~28% vs ~86% recurrence in pivotal data) |
Where trials have failed
Drugs that missed their endpoint — and what contributed
The most instructive lessons in uveitis development come from programmes that failed the endpoint that mattered.
| Drug / trial | Endpoint outcome | What contributed |
|---|---|---|
| gevokizumab (anti-IL-1beta) — EYEGUARD-B | Failed to meet the primary endpoint (time to first acute ocular exacerbation) in Behçet's disease uveitis | No significant reduction in uveitic flares versus placebo on background therapy; IL-1beta blockade insufficient to control the disease, ending the program |
| secukinumab (anti-IL-17A) — SHIELD, INSURE and ENDURE | Intravenous/subcutaneous secukinumab failed to meet primary endpoints for reducing uveitis recurrence and steroid-sparing in non-infectious uveitis | Subcutaneous dosing may have given inadequate exposure; IL-17 blockade did not translate to meaningful control, and the ocular program was discontinued |
Choosing the right endpoint
Primary endpoints that matter in uveitis trials
- Time to treatment failure — Composite regulatory endpoint (VISUAL trials) combining new lesions, vitreous haze, anterior chamber cells and BCVA decline
- Vitreous haze score — Standardised (NEI/SUN) grading of vitreous opacity; a score of 0 signifies quiescence, used in HURON
- Uveitis recurrence rate — Frequency of inflammatory relapse over follow-up; primary measure for sustained-release implants
- Corticosteroid-sparing — Ability to taper systemic steroids while maintaining control; key practical benefit of biologics and implants
How iNGENū runs uveitis trials
Physician-led design, built for FDA submission
Endpoint & biomarker strategy
Board-certified specialists design endpoints and patient selection aligned to current FDA guidance for this indication.
FDA-ready data
Built to ICH-GCP and 21 CFR 312.120, with direct FDA submission — data accepted by the FDA, EMA and MHRA.
Faster, lower-cost delivery
~4-week ethics via the TGA CTN scheme, up to 43.5% R&D rebate, and 80–90% below US CRO cost.
Request a fixed-milestone proposal and a tailored endpoint & feasibility summary for this indication.
Frequently asked questions
Uveitis clinical trials — FAQs
Why is adalimumab important in uveitis management?
When are corticosteroid implants used?
Why have several biologics failed in uveitis?
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