Ophthalmology · Clinical trials
Retinal Vein Occlusion 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 retinal vein occlusion — and why its trials are hard
Retinal vein occlusion (RVO) is the second most common retinal vascular disorder after diabetic retinopathy, arising when a retinal vein is blocked, causing haemorrhage, ischaemia and macular oedema. It is classified as branch (BRVO) or central (CRVO) occlusion, with macular oedema the main driver of vision loss. VEGF and inflammatory cytokines released from ischaemic retina increase vascular permeability. Before anti-VEGF therapy, grid laser (for BRVO) and observation were standard, and CRVO carried a poor prognosis. Intravitreal anti-VEGF agents transformed outcomes: ranibizumab (CRUISE for CRVO, BRAVO for BRVO) and aflibercept (COPERNICUS and GALILEO for CRVO, VIBRANT for BRVO) produced rapid, large gains in best-corrected visual acuity. The dexamethasone implant (Ozurdex, GENEVA) offers a steroid alternative, and faricimab (BALATON/COMINO) has extended the class. Endpoints are mean BCVA letter change and the proportion gaining ≥15 letters at the primary (often six-month) timepoint. Prompt treatment and ischaemia monitoring, including for neovascular glaucoma, are essential.
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 |
|---|---|---|---|---|---|
| ranibizumab (Lucentis) | 2010 | Macular oedema following CRVO and BRVO | CRUISE (CRVO) and BRAVO (BRVO) | Mean change in BCVA at month 6 vs sham | CRUISE +14.9 letters vs +0.8 sham; BRAVO +18.3 letters vs +7.3 sham (0.5 mg) |
| aflibercept (Eylea) | 2012 | Macular oedema following CRVO (BRVO added 2014) | COPERNICUS and GALILEO (CRVO); VIBRANT (BRVO) | Proportion gaining ≥15 letters at week 24 | COPERNICUS ~56% gained ≥15 letters vs ~12% sham; mean ~+17.3 letters |
| dexamethasone intravitreal implant (Ozurdex) | 2009 | Macular oedema following BRVO or CRVO (corticosteroid) | GENEVA | Time to ≥15-letter BCVA improvement over 6 months | Faster and higher proportion reaching ≥15-letter gain vs sham (peak ~29% at day 60); benefit waned by ~6 months |
| faricimab (Vabysmo) | 2024 | Macular oedema following RVO | BALATON (BRVO) and COMINO (CRVO) | Mean BCVA change at week 24; non-inferiority vs aflibercept | Non-inferior to aflibercept, ~+16.9 letters (BALATON) and ~+16.9–17 letters (COMINO) |
Where trials have failed
Drugs that missed their endpoint — and what contributed
The most instructive lessons in retinal vein occlusion development come from programmes that failed the endpoint that mattered.
| Drug / trial | Endpoint outcome | What contributed |
|---|---|---|
| triamcinolone acetonide (intravitreal) — SCORE-BRVO | In branch RVO, intravitreal triamcinolone showed no visual-acuity benefit over standard grid laser, while causing more cataract and raised intraocular pressure | Steroid side-effect burden without efficacy advantage in BRVO (unlike SCORE-CRVO, where triamcinolone beat observation) discouraged its use as first-line therapy |
Choosing the right endpoint
Primary endpoints that matter in retinal vein occlusion trials
- Mean BCVA change (ETDRS letters) — Primary functional endpoint at the 6-month timepoint in CRUISE, BRAVO and COPERNICUS
- Proportion gaining ≥15 letters — Responder measure emphasising clinically meaningful three-line vision improvement
- Central retinal/subfield thickness (OCT) — Tracks resolution of macular oedema and informs retreatment intervals
- Ischaemia / neovascularisation conversion — Safety and prognostic measure; CRVO non-perfusion can progress to neovascular glaucoma
How iNGENū runs retinal vein occlusion 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
Retinal Vein Occlusion clinical trials — FAQs
Is CRVO or BRVO more responsive to treatment?
How do steroid implants compare with anti-VEGF in RVO?
How quickly must RVO macular oedema be treated?
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