Nathan C Steinle1, Ian Pearce2, Jordi Monés3, Ravi Metlapally4, Namrata Saroj5, Mohamed Hamdani4, Ramiro Ribeiro4, Philip J Rosenfeld6, Eleonora M Lad7. 1. From the California Retina Consultants, Santa Barbara, California, USA. 2. St Paul's Eye Unit, Royal Liverpool University Hospital, Liverpool, England, United Kingdom; Department of Eye and Vision Science, University of Liverpool, England, United Kingdom. 3. Barcelona Macula Foundation, Institut de la Màcula, Hospital Quirón Teknon, Barcelona, Spain. 4. Apellis Pharmaceuticals, Waltham, Massachusetts, USA. 5. All Eyes Consulting, LLC, New York, New York, USA. 6. Department of Ophthalmology, Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, Florida, USA. 7. Department of Ophthalmology, Duke University Medical Center, Durham, North Carolina, USA. Electronic address: nora.lad@duke.edu.
Abstract
PURPOSE: To evaluate the effect of select baseline characteristics on geographic atrophy (GA) progression in eyes receiving intravitreal pegcetacoplan or sham. DESIGN: Phase 2 multicenter, randomized, single-masked, sham-controlled trial. METHODS: Patients with GA received 15 mg pegcetacoplan monthly or every other month (EOM), or sham injection monthly or EOM for 12 months. Primary efficacy endpoint was change in GA lesion size (square root) from baseline. Post hoc analysis evaluated the effects of age; gender; lesion size, focality, and location (extrafoveal vs foveal); pseudodrusen status; best-corrected visual acuity (BCVA); and low-luminance deficit (LLD) on GA progression at Month 12. RESULTS: Of 246 randomized patients, 192 with 12-month data were included in this analysis. Overall mean (standard deviation) change in lesion size (mm) was 0.26 (0.17) (P < .01), 0.27 (0.27) (P < .05), and 0.36 (0.21) in the monthly pegcetacoplan (n = 67), EOM pegcetacoplan (n = 58), and sham (n = 67) groups, respectively. In univariate analysis, patients with extrafoveal lesions (P < .001), BCVA ≥20/60 (P = .001), and larger LLD (P = .002) had greater mean changes in lesion size. Multivariate analysis confirmed significant association of extrafoveal lesions (P = .001) and larger LLD (P = .023) with GA progression. Monthly and EOM pegcetacoplan significantly reduced progression (P < .05) when controlling for these risk factors. CONCLUSIONS:Extrafoveal lesions and larger LLD are potential risk factors for GA progression. Pegcetacoplan treatment significantly controlled GA progression even after accounting for these risk factors.
RCT Entities:
PURPOSE: To evaluate the effect of select baseline characteristics on geographic atrophy (GA) progression in eyes receiving intravitreal pegcetacoplan or sham. DESIGN: Phase 2 multicenter, randomized, single-masked, sham-controlled trial. METHODS:Patients with GA received 15 mg pegcetacoplan monthly or every other month (EOM), or sham injection monthly or EOM for 12 months. Primary efficacy endpoint was change in GA lesion size (square root) from baseline. Post hoc analysis evaluated the effects of age; gender; lesion size, focality, and location (extrafoveal vs foveal); pseudodrusen status; best-corrected visual acuity (BCVA); and low-luminance deficit (LLD) on GA progression at Month 12. RESULTS: Of 246 randomized patients, 192 with 12-month data were included in this analysis. Overall mean (standard deviation) change in lesion size (mm) was 0.26 (0.17) (P < .01), 0.27 (0.27) (P < .05), and 0.36 (0.21) in the monthly pegcetacoplan (n = 67), EOM pegcetacoplan (n = 58), and sham (n = 67) groups, respectively. In univariate analysis, patients with extrafoveal lesions (P < .001), BCVA ≥20/60 (P = .001), and larger LLD (P = .002) had greater mean changes in lesion size. Multivariate analysis confirmed significant association of extrafoveal lesions (P = .001) and larger LLD (P = .023) with GA progression. Monthly and EOM pegcetacoplan significantly reduced progression (P < .05) when controlling for these risk factors. CONCLUSIONS: Extrafoveal lesions and larger LLD are potential risk factors for GA progression. Pegcetacoplan treatment significantly controlled GA progression even after accounting for these risk factors.
Authors: Jeremy Liu; Rita Laiginhas; Federico Corvi; Frederick L Ferris; Tock Han Lim; Srinivas R Sadda; Nadia K Waheed; Prashanth G Iyer; Mengxi Shen; Yingying Shi; Omer Trivizki; Liang Wang; Elizabeth A Vanner; William J Feuer; Giovanni Gregori; Philip J Rosenfeld Journal: Ophthalmol Retina Date: 2022-01-31
Authors: Mengxi Shen; Yingying Shi; Liang Wang; Jonathan F Russell; Xiaoshuang Jiang; Rita Laiginhas; Prashanth Iyer; Omer Trivizki; Marie Thulliez; Sonia H Yoo; Terri P Rose; Ranya G Habash; Guillermo Amescua; William J Feuer; Giovanni Gregori; Philip J Rosenfeld Journal: Ophthalmol Sci Date: 2022-05-18