PURPOSE: To investigate the safety and preliminary efficacy of OT-551, a disubstituted hydroxylamine with antioxidant properties, for the treatment of geographic atrophy (GA), the advanced atrophic form of age-related macular degeneration (AMD). METHODS: The study was a single-center, open-label phase II trial, enrolling 10 participants with bilateral GA. Topical 0.45% OT-551 was administered in one randomly assigned eye three times daily for 2 years. Safety measures were assessed by complete ophthalmic examination, fundus photography, and review of symptoms. The primary efficacy outcome measure was the change in best corrected visual acuity at 24 months. Secondary efficacy measures included changes in area of GA, contrast sensitivity, microperimetry measurements, and total drusen area from baseline. RESULTS: Study drug was well tolerated and was associated with few adverse events. The mean change in BCVA at 2 years was +0.2 ± 13.3 letters in the study eyes and -11.3 ± 7.6 letters in fellow eyes (P = 0.0259). However, no statistically significant differences were found between the study and fellow eyes for all other secondary outcome measures. CONCLUSIONS:OT-551 was well tolerated by study participants and was not associated with any serious adverse effects. Efficacy measurements in this small study indicate a possible effect in maintaining visual acuity. However, the absence of significant effects on other outcomes measures in this study suggests that OT-551, in the current concentration and mode of delivery, may have limited or no benefit as a treatment for GA (ClinicalTrials.gov number, NCT00306488).
RCT Entities:
PURPOSE: To investigate the safety and preliminary efficacy of OT-551, a disubstituted hydroxylamine with antioxidant properties, for the treatment of geographic atrophy (GA), the advanced atrophic form of age-related macular degeneration (AMD). METHODS: The study was a single-center, open-label phase II trial, enrolling 10 participants with bilateral GA. Topical 0.45% OT-551 was administered in one randomly assigned eye three times daily for 2 years. Safety measures were assessed by complete ophthalmic examination, fundus photography, and review of symptoms. The primary efficacy outcome measure was the change in best corrected visual acuity at 24 months. Secondary efficacy measures included changes in area of GA, contrast sensitivity, microperimetry measurements, and total drusen area from baseline. RESULTS: Study drug was well tolerated and was associated with few adverse events. The mean change in BCVA at 2 years was +0.2 ± 13.3 letters in the study eyes and -11.3 ± 7.6 letters in fellow eyes (P = 0.0259). However, no statistically significant differences were found between the study and fellow eyes for all other secondary outcome measures. CONCLUSIONS:OT-551 was well tolerated by study participants and was not associated with any serious adverse effects. Efficacy measurements in this small study indicate a possible effect in maintaining visual acuity. However, the absence of significant effects on other outcomes measures in this study suggests that OT-551, in the current concentration and mode of delivery, may have limited or no benefit as a treatment for GA (ClinicalTrials.gov number, NCT00306488).
Authors: Janet S Sunness; Eyal Margalit; Divya Srikumaran; Carol A Applegate; Yan Tian; Daniel Perry; Barbara S Hawkins; Neil M Bressler Journal: Ophthalmology Date: 2007-02 Impact factor: 12.079
Authors: Ronald Klein; Barbara E K Klein; Michael D Knudtson; Stacy M Meuer; Maria Swift; Ronald E Gangnon Journal: Ophthalmology Date: 2007-02 Impact factor: 12.079
Authors: Qinghua Liang; Amanda D Smith; Stephen Pan; Vladimir A Tyurin; Valerian E Kagan; Teresa G Hastings; Nina Felice Schor Journal: Biochem Pharmacol Date: 2005-11-01 Impact factor: 5.858
Authors: Masaki Tanito; Feng Li; Michael H Elliott; Mark Dittmar; Robert E Anderson Journal: Invest Ophthalmol Vis Sci Date: 2007-04 Impact factor: 4.799
Authors: Michael L Klein; Frederick L Ferris; Jane Armstrong; Thomas S Hwang; Emily Y Chew; Susan B Bressler; Suresh R Chandra Journal: Ophthalmology Date: 2007-11-05 Impact factor: 12.079
Authors: Joe G Hollyfield; Vera L Bonilha; Mary E Rayborn; Xiaoping Yang; Karen G Shadrach; Liang Lu; Rafael L Ufret; Robert G Salomon; Victor L Perez Journal: Nat Med Date: 2008-01-27 Impact factor: 53.440
Authors: Annal D Meleth; Pradeep Mettu; Elvira Agrón; Emily Y Chew; Srinivas R Sadda; Frederick L Ferris; Wai T Wong Journal: Invest Ophthalmol Vis Sci Date: 2011-02-28 Impact factor: 4.799
Authors: Etienne M Schönbach; Rupert W Strauss; Beatriz Muñoz; Yulia Wolfson; Mohamed A Ibrahim; David G Birch; Eberhart Zrenner; Janet S Sunness; Michael S Ip; SriniVas R Sadda; Sheila K West; Hendrik P N Scholl Journal: JAMA Ophthalmol Date: 2020-07-01 Impact factor: 7.389
Authors: SriniVas R Sadda; Usha Chakravarthy; David G Birch; Giovanni Staurenghi; Erin C Henry; Christopher Brittain Journal: Retina Date: 2016-10 Impact factor: 4.256