PMID- 31430268
OWN - NLM
STAT- MEDLINE
DCOM- 20200615
LR  - 20200901
IS  - 1536-5166 (Electronic)
IS  - 1070-8022 (Print)
IS  - 1070-8022 (Linking)
VI  - 39
IP  - 3
DP  - 2019 Sep
TI  - Randomized Controlled Phase 2a Study of RPh201 in Previous Nonarteritic Anterior 
      Ischemic Optic Neuropathy.
PG  - 291-298
LID - 10.1097/WNO.0000000000000786 [doi]
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.
FAU - Rath, Eitan Z
AU  - Rath EZ
AD  - Ophthalmology Department (EZR, ZIS), Galilee Medical Center, Nahariya; and 
      Azrieli Faculty of Medicine, Bar-Ilan University, Safed, Israel; Regenera Pharma 
      Ltd (ZH, KA), Israel; Goldschleger Eye Research Institute (AS), Sheba Medical 
      Center, Tel Aviv University, Tel Hashomer, Israel; and Departments of 
      Ophthalmology and Visual Sciences, Neurology and Neurosurgery (LAL), McGill 
      University, Montreal, Canada.
FAU - Hazan, Zadik
AU  - Hazan Z
FAU - Adamsky, Konstantin
AU  - Adamsky K
FAU - Solomon, Arieh
AU  - Solomon A
FAU - Segal, Zvi I
AU  - Segal ZI
FAU - Levin, Leonard A
AU  - Levin LA
LA  - eng
SI  - ClinicalTrials.gov/NCT02045212
GR  - P30 EY016665/EY/NEI NIH HHS/United States
GR  - R21 EY025074/EY/NEI NIH HHS/United States
PT  - Clinical Trial, Phase II
PT  - Journal Article
PT  - Randomized Controlled Trial
PT  - Research Support, N.I.H., Extramural
PT  - Research Support, Non-U.S. Gov't
PL  - United States
TA  - J Neuroophthalmol
JT  - Journal of neuro-ophthalmology : the official journal of the North American 
      Neuro-Ophthalmology Society
JID - 9431308
RN  - 0 (Mastic Resin)
RN  - 0 (Plant Extracts)
SB  - IM
MH  - Aged
MH  - Evoked Potentials, Visual/*drug effects/physiology
MH  - Female
MH  - Humans
MH  - Male
MH  - Mastic Resin/adverse effects/pharmacology/*therapeutic use
MH  - Middle Aged
MH  - Optic Neuropathy, Ischemic/*drug therapy/physiopathology
MH  - Plant Extracts/adverse effects/pharmacology/*therapeutic use
MH  - Retina/drug effects/physiopathology
MH  - Treatment Outcome
MH  - Visual Acuity/*drug effects/physiology
PMC - PMC6705418
MID - NIHMS1522016
COIS- Conflict of interest: Zvi I. Segal, and Ethan Z. Rath were PI's of the study. 
      Zadik Hazan and Konstantin Adamsky are employees of Regenera Pharma Limited, 
      which funded the study. Leonard A. Levin is a consultant to Regenera, as well as 
      to Aerie, Eyevensys, Galimedix, and Quark.
EDAT- 2019/08/21 06:00
MHDA- 2020/06/17 06:00
PMCR- 2020/09/01
CRDT- 2019/08/21 06:00
PHST- 2019/08/21 06:00 [entrez]
PHST- 2019/08/21 06:00 [pubmed]
PHST- 2020/06/17 06:00 [medline]
PHST- 2020/09/01 00:00 [pmc-release]
AID - 00041327-201909000-00001 [pii]
AID - 10.1097/WNO.0000000000000786 [doi]
PST - ppublish
SO  - J Neuroophthalmol. 2019 Sep;39(3):291-298. doi: 10.1097/WNO.0000000000000786.