Sheppard John, Bergmann Mark, Schechter Barry A, Luchs Jodi, Ogundele Abayomi, Karpecki Paul
Virginia Eye Consultants, Norfolk, VA, USA.
Apex Eye, Cincinnati, OH, USA.
Clin Ophthalmol. 2021 Jan 12;15:129-140. doi: 10.2147/OPTH.S279364. eCollection 2021.
OTX-101 is approved for treatment of keratoconjunctivitis sicca (KCS). We present results of a phase 3 worse-eye efficacy analysis and 1-year safety extension.
During the double-masked treatment phase, patients with bilateral KCS were randomized 1:1 to 12 weeks OTX-101 or vehicle 1 drop per eye twice daily. Efficacy assessments included Schirmer's test and corneal and conjunctival staining. All patients who completed the treatment phase were eligible for enrollment in the open-label extension and received 1 drop OTX-101 twice daily for up to 52 weeks. Safety endpoints included adverse event (AE) monitoring, Snellen visual acuity (VA), intraocular pressure (IOP), slit-lamp examination (SLE), and dilated fundoscopy.
Overall, 745 and 258 patients enrolled in the treatment and safety extension phases, respectively. At 12 weeks, number (%) of patients with Schirmer's score increase of ≥10 mm from baseline was 76 (20.5%) vs. 42 (11.3%) for OTX-101 vs. vehicle (=0.0005). OTX-101 significantly improved total conjunctival staining vs. vehicle at week 12 (least squares mean change from baseline -1.65 [0.12] vs. -1.12 [0.12], =0.0013), and number (%) of patients with clear central corneas vs. vehicle at week 12 (222 [64.0%] vs. 199 [55.3%], =0.0179). In the 1-year safety extension, AEs were mostly mild; instillation site pain was most common in 59 (22.9%) patients (17 [13.2%] vs. 42 [32.6%] patients receiving prior OTX-101 and vehicle). No safety concerns were raised by VA, IOP, SLE, and fundoscopy.
OTX-101 efficacy was confirmed in the eye with lower baseline Schirmer's score. OTX-101 was well tolerated long term.
Registered at ClinicalTrials.gov on July 27, 2016. NCT02845674 https://clinicaltrials.gov/ct2/show/NCT02845674?term=OTX-101&draw=2&rank=1.
OTX - 101已获批用于治疗干眼症（KCS）。我们展示了一项3期较差眼疗效分析和1年安全性扩展研究的结果。
在双盲治疗阶段，双侧干眼症患者按1:1随机分组，分别接受为期12周的OTX - 101或赋形剂治疗，每日双眼各滴1次，每次1滴。疗效评估包括泪液分泌试验、角膜和结膜染色。所有完成治疗阶段的患者均有资格进入开放标签扩展研究阶段，接受每日双眼各滴1次OTX - 101，持续长达52周。安全性终点包括不良事件（AE）监测、斯内伦视力（VA）、眼压（IOP）、裂隙灯检查（SLE）和散瞳眼底检查。
总体而言，分别有745例和258例患者进入治疗阶段和安全性扩展阶段。在12周时，泪液分泌试验评分较基线增加≥10 mm的患者数量（%），OTX - 101组为76例（20.5%），赋形剂组为42例（11.3%）（P = 0.0005）。在第12周时，OTX - 101组与赋形剂组相比，结膜总染色显著改善（从基线的最小二乘均值变化 - 1.65 [0.12] 对比 - 1.12 [0.12]，P = 0.0013），且第12周时中央角膜清亮的患者数量（%），OTX - 101组为222例（64.0%），赋形剂组为199例（55.
