A Randomized, Placebo-Controlled Phase II Clinical Trial of 0.01% or 0.02% Cyclosporin A with 3% Trehalose in Patients with Dry Eye Disease.

Purpose: To compare the efficacy and safety of HU00701 (0.01% cyclosporin A + 3% trehalose), HU007 (0.02% cyclosporin A + 3% trehalose) (all w/v), and placebo in patients with moderate to severe dry eye disease (DED). Methods: This was a multicenter, randomized, double-masked, parallel, placebo-controlled phase II study. In total, 114 patients were randomly assigned to the HU00701, HU007, placebo, or reference group. There was a 2-week run-in period before the 12-week intervention. Efficacy and safety were evaluated every 4 weeks. Results: The primary endpoint, change in corneal staining score from baseline to week 12, did not differ significantly among the control, HU00701, and HU007 groups in the full analysis. Of the secondary endpoints, only the tear film breakup time differed significantly at week 12 between the placebo and HU00701 groups. Twenty adverse events were reported by 15 patients, but the rate did not differ significantly among the 4 groups. The laboratory test, vital signs, and physical examination data showed no significant changes during the study. Conclusions: HU00701 and HU007 are safe, and HU007 effectively reduces the corneal staining score in patients with moderate-to-severe DED (NCT02917512).