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Title: Safety and effectiveness of a novel neuroprotectant, KUS121, in patients with non-arteritic central retinal artery occlusion: An open-label, non-randomized, first-in-humans, phase 1/2 trial
Authors: Ohashi Ikeda, Hanako
Muraoka, Yuki
Hata, Masayuki
Sumi, Eriko  kyouindb  KAKEN_id  orcid (unconfirmed)
Ikeda, Takafumi
Nakagawa, Takayuki  kyouindb  KAKEN_id  orcid (unconfirmed)
Abe, Hiroyasu  kyouindb  KAKEN_id
Tada, Harue  kyouindb  KAKEN_id
Morita, Satoshi  kyouindb  KAKEN_id
Kakizuka, Akira  kyouindb  KAKEN_id
Yoshimura, Nagahisa
Tsujikawa, Akitaka
Author's alias: 池田, 華子
村岡, 勇貴
畑, 匡侑
角, 栄里子
池田, 隆文
中川, 貴之
阿部, 寛康
多田, 春江
森田, 智視
垣塚, 彰
吉村, 長久
辻川, 明孝
Issue Date: 13-Feb-2020
Publisher: Public Library of Science (PLoS)
Journal title: PLOS ONE
Volume: 15
Issue: 2
Thesis number: e0229068
Abstract: Kyoto University Substance (KUS) 121, an ATPase inhibitor of valosin-containing protein, is a novel neuroprotectant. We tested the safety and effectiveness of KUS121 in patients with acute central retinal artery occlusion (CRAO). We conducted an investigator-initiated, first-in-humans, phase 1/2 clinical trial. Nine patients with non-arteritic CRAO symptoms lasting for 4–48 h were enrolled. These patients received daily intravitreal injections of KUS121 for 3 days: 25 μg (low-dose) in the first three patients and 50 μg (high-dose) in the next six patients. The primary endpoint was the safety of the drug. As a secondary endpoint, pharmacokinetics was evaluated. Other key secondary endpoints were changes in best-corrected visual acuity (BCVA), measured using the Early Treatment Diabetic Retinopathy Study chart, visual field scores, and retinal sensitivities between baseline and week 12; and decimal BCVA at week 12. Administration of KUS121 did not result in serious adverse events. All nine patients (100%) showed significant improvement of BCVA. Average readable letter counts, visual field scores, and retinal sensitivities also improved. Decimal BCVA at week 12 was better than 0.1 in four patients (44%) and equal to or better than 0.05 in seven patients (78%). This first-in-humans clinical trial provides support for the safety and efficacy of intravitreal KUS121 injection. To substantiate the safety and effectiveness for patients with acute CRAO, further larger scale clinical studies will be needed.
Description: 新規神経保護剤「KUS121」の投与安全性と有効性を医師主導治験にてヒトで初めて確認 --治療薬を網膜中心動脈閉塞症患者へ--. 京都大学プレスリリース. 2020-03-03.
Rights: © 2020 Ikeda et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.
DOI(Published Version): 10.1371/journal.pone.0229068
PubMed ID: 32053676
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