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A Randomized, Controlled Trial of Cyclosporine A Cationic Emulsion in Pediatric Vernal Keratoconjunctivitis: The
Andrea Leonardi1, Serge Doan2, Mourad Amrane3
1Department of Neuroscience, Ophthalmology Unit, University of Padua, Padua, Italy.
Insights
Investigational cyclosporine A cationic emulsion (CsA CE) eye drops effectively treated severe vernal keratoconjunctivitis (VKC) in children and adolescents. High-dose CsA CE significantly improved corneal health and quality of life in VKC patients.
Area of Science:
- Ophthalmology
- Allergic Eye Disease
- Pediatric Eye Care
Background:
- Vernal keratoconjunctivitis (VKC) is a chronic, severe allergic eye condition affecting children and adolescents.
- VKC can impair quality of life (QoL) and lead to vision loss.
- Conventional cyclosporine A (CsA) formulations have limitations in treating severe VKC.
Purpose of the Study:
- To evaluate the efficacy and safety of a novel cyclosporine A cationic emulsion (CsA CE) for severe VKC.
- To compare CsA CE's effectiveness against a vehicle control in pediatric patients.
- To assess the impact of CsA CE on corneal health, symptoms, and QoL.
Main Methods:
- A phase 3, multicenter, double-masked, vehicle-controlled trial (VEKTIS) was conducted.
- 169 pediatric patients (4-18 years) with severe VKC and keratitis were randomized.
- Participants received CsA CE (high or low dose) or vehicle eye drops for 4 months.
Main Results:
- Both high-dose and low-dose CsA CE significantly reduced the primary composite endpoint, mainly driven by corneal fluorescein staining (CFS) scores.
- Significant reductions in rescue medication (dexamethasone) use were observed in CsA CE groups compared to vehicle.
- Improvements in VKC symptoms and patient QoL were noted across all groups, with greater significant improvements in the high-dose CsA CE group.
Conclusions:
- High-dose CsA CE demonstrated significant efficacy in improving keratitis, symptoms, and QoL in pediatric patients with severe VKC.
- CsA CE was well-tolerated in the study cohort.
- This investigational therapy shows promise for managing severe VKC in children and adolescents.
Purpose:
Vernal keratoconjunctivitis (VKC) is a chronic, allergic, and potentially severe ocular disease affecting children and adolescents that can lead to impaired quality of life (QoL) and loss of vision. This study evaluated the efficacy and safety of an investigational therapy for severe VKC, cyclosporine A (CsA) cationic emulsion (CE), an oil-in-water emulsion with increased bioavailability versus conventional CsA formulations.
Design:
The VErnal KeratoconjunctiviTIs Study (VEKTIS) is a phase 3, multicenter, double-masked, vehicle-controlled trial.
Participants:
Pediatric patients (4 to younger than 18 years) with active severe VKC (grade of 3 or 4 on the Bonini severity scale) and severe keratitis (corneal fluorescein staining [CFS] score of 4 or 5 on the modified Oxford scale).
Methods:
One hundred sixty-nine patients were randomized to CsA CE 0.1% (1 mg/ml) eye drops 4 times daily (high dose), CsA CE twice daily (low dose) plus vehicle twice daily, or vehicle 4 times daily for 4 months.
Main Outcome Measures:
The primary end point was a mean composite score that reflected CFS, rescue medication use (dexamethasone 0.1% 4 times daily), and corneal ulceration over the 4 months.
Results:
Differences in least-squares means versus vehicle for the primary end point were statistically significant for both the high-dose (0.76; P = 0.007) and the low-dose (0.67; P = 0.010) groups, with treatment effect mainly driven by CFS score. Significant differences were found between both active treatment groups and vehicle for use of rescue medication. Vernal keratoconjunctivitis symptoms and patient QoL (assessed by visual analog scale and the Quality of Life in Children with Vernal Keratoconjunctivitis questionnaire) improved in all 3 groups, with significant improvements for high-dose CsA CE versus vehicle.
Conclusions:
The efficacy of high-dose CsA CE in improving keratitis, symptoms, and QoL for those with severe VKC was demonstrated in these study patients. In addition, in this study cohort, CsA CE was well tolerated.
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