A Phase II/III, randomized, double-masked, vehicle-controlled, dose-ranging study of the safety and efficacy of OTX-101 in the treatment of dry eye disease
Received 23 May 2018
Accepted for publication 18 August 2018
Published 2 October 2018 Volume 2018:12 Pages 1921—1929
Checked for plagiarism Yes
Review by Single-blind
Peer reviewer comments 5
Editor who approved publication: Dr Scott Fraser
Joseph Tauber,1 Barry A Schechter,2 Jason Bacharach,3 Melissa M Toyos,4 Robert Smyth-Medina,5 Sidney L Weiss,6 Jodi I Luchs7
1Tauber Eye Center, Kansas City, MO, USA; 2Florida Eye Microsurgical Institute, Boynton Beach, FL, USA; 3North Bay Eye Associates, Petaluma, CA, USA; 4Toyos Clinic, Nashville, TN, USA; 5North Valley Eye Medical Group, Mission Hills, CA, USA; 6i-Novion, Inc, Randolph, NJ, USA; 7South Shore Eye Care, Wantagh, NY, USA
Purpose: The aim of this study was to evaluate the safety and efficacy of OTX-101, a clear nanomicellar aqueous solution of cyclosporine, in the treatment of dry eye disease (DED).
Patients and methods: This was a 12-week multicenter, randomized, prospective, double-masked, vehicle-controlled, dose-ranging clinical trial. Subjects were adults aged ≥18 years, with a total conjunctival staining score of ≥3 and ≤9, and global DED symptom score ≥40 (0–100 visual analogue scale). Following a 14-day vehicle run-in, subjects were randomized in a 1:1:1 ratio to twice daily treatment with OTX-101 0.09%, OTX-101 0.05%, or vehicle for 84 days. Co-primary efficacy end points were changes, from baseline to Day 84, in the total lissamine green conjunctival staining score in the designated study eye and in the global symptom score (both eyes). Secondary end points included total corneal fluorescein staining score, tear breakup time, and Schirmer’s test score.
Results: In total, 455 subjects were randomized. Subjects treated with active drug experienced greater improvement in conjunctival staining than vehicle-treated patients (P<0.01 for both concentrations). All groups demonstrated improvements in global symptom score, but there were no differences among groups. Nominally significant differences were found between the active drug arms and vehicle for corneal staining scores and Schirmer’s test scores. Most treatment-emergent adverse events were mild in severity; no serious ocular adverse events were reported.
Conclusions: Both concentrations of OTX-101 met the co-primary sign end point (conjunctival staining) but not the co-primary symptom end point. OTX-101 0.09% demonstrated a notable impact on multiple signs of DED relative to vehicle and was well-tolerated.
Keywords: dry eye disease, cyclosporine, inflammation, Schirmer’s test
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