TY - JOUR
T1 - Randomized Controlled Phase 2a Study of RPh201 in Previous Nonarteritic Anterior Ischemic Optic Neuropathy
AU - Rath, Eitan Z.
AU - Hazan, Zadik
AU - Adamsky, Konstantin
AU - Solomon, Arieh
AU - Segal, Zvi I.
AU - Levin, Leonard A.
N1 - Publisher Copyright:
© 2019 by North American Neuro-Ophthalmology Society.
PY - 2019/9/1
Y1 - 2019/9/1
N2 - Background: No proven treatment exists for nonarteritic anterior ischemic optic neuropathy (NAION), either in the acute or late phase. Objective: To assess safety and changes in visual function and structure after RPh201/placebo treatment in participants with previous NAION. Design and Setting: Phase 2a, single-site, prospective, randomized, placebo-controlled, double-masked trial (registration NCT02045212). Main Outcomes Measures: Early Treatment Diabetic Retinopathy Study best-corrected visual acuity (BCVA), visual fields, retinal nerve fiber layer, and visual evoked potential at weeks 13, 26, and after a 13-week wash-out (“off-drug”) period; and safety. Study Population: Twenty-two participants aged 18 years or older with previous NAION. Intervention(s): RPh201 (20 mg) or placebo (cottonseed oil vehicle) administered subcutaneously twice weekly at the study site. Results: Thirteen men and 9 women were randomized, of which 20 completed all visits. The mean (±SD) age was 61.0 ± 7.6 years. In a post hoc analysis, after 26 weeks of treatment, BCVA improved by $15 letters in 4/11 (36.4%) eyes with RPh201, compared to 1/8 (12.5%) eyes with placebo (P = 0.24). Overall, 7/11 (63.6%) of participants on RPh201 showed some improvement in BCVA, compared with 3/8 (37.5%) on placebo (P = 0.26). Improvement in BCVA from a calculated baseline was 14.8 ± 15.8 letters for RPh201 and 6.6 ± 15.3 for placebo (P = 0.27). Of the 154 adverse effects (AEs), 52 were considered related to the study procedures/treatment. Across the study and 1,017 injections, the most frequently reported AE was injection site pain (23 events in 5 participants). There were no clinically significant changes in vital signs or laboratory values. Conclusions: This Phase 2a was designed to assess safety, feasibility, and explore potential efficacy signals in treating previous NAION with RPh201. No safety concerns were raised. The results support a larger trial in patients with previous NAION.
AB - Background: No proven treatment exists for nonarteritic anterior ischemic optic neuropathy (NAION), either in the acute or late phase. Objective: To assess safety and changes in visual function and structure after RPh201/placebo treatment in participants with previous NAION. Design and Setting: Phase 2a, single-site, prospective, randomized, placebo-controlled, double-masked trial (registration NCT02045212). Main Outcomes Measures: Early Treatment Diabetic Retinopathy Study best-corrected visual acuity (BCVA), visual fields, retinal nerve fiber layer, and visual evoked potential at weeks 13, 26, and after a 13-week wash-out (“off-drug”) period; and safety. Study Population: Twenty-two participants aged 18 years or older with previous NAION. Intervention(s): RPh201 (20 mg) or placebo (cottonseed oil vehicle) administered subcutaneously twice weekly at the study site. Results: Thirteen men and 9 women were randomized, of which 20 completed all visits. The mean (±SD) age was 61.0 ± 7.6 years. In a post hoc analysis, after 26 weeks of treatment, BCVA improved by $15 letters in 4/11 (36.4%) eyes with RPh201, compared to 1/8 (12.5%) eyes with placebo (P = 0.24). Overall, 7/11 (63.6%) of participants on RPh201 showed some improvement in BCVA, compared with 3/8 (37.5%) on placebo (P = 0.26). Improvement in BCVA from a calculated baseline was 14.8 ± 15.8 letters for RPh201 and 6.6 ± 15.3 for placebo (P = 0.27). Of the 154 adverse effects (AEs), 52 were considered related to the study procedures/treatment. Across the study and 1,017 injections, the most frequently reported AE was injection site pain (23 events in 5 participants). There were no clinically significant changes in vital signs or laboratory values. Conclusions: This Phase 2a was designed to assess safety, feasibility, and explore potential efficacy signals in treating previous NAION with RPh201. No safety concerns were raised. The results support a larger trial in patients with previous NAION.
UR - http://www.scopus.com/inward/record.url?scp=85071571872&partnerID=8YFLogxK
U2 - 10.1097/WNO.0000000000000786
DO - 10.1097/WNO.0000000000000786
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C2 - 31430268
AN - SCOPUS:85071571872
SN - 1070-8022
VL - 39
SP - 291
EP - 298
JO - Journal of Neuro-Ophthalmology
JF - Journal of Neuro-Ophthalmology
IS - 3
ER -
