{"title":"Safety and efficacy of ripasudil eye drops in preterm infants with retinopathy of prematurity: phase 1/2, open label, single-arm trial.","authors":"Mitsuru Arima, Hirosuke Inoue, Akiko Misumi, Shoko Tsukamoto, Itsuka Matsushita, Shunsuke Araki, Manami Ohta, Kazumasa Takahashi, Miyuki Imazato, Tomoko Goto, Yoshinori Aoki, Koshiro Tagawa, Masayuki Hirose, Yuito Fujita, Noriko Yoshida, Shintaro Nakao, Hiroyuki Kondo, Koichi Kusuhara, Kazuhiro Kimura, Shunji Hasegawa, Yasuhiro Ikeda, Yuki Kodama, Hiroshi Moritake, Masayuki Ochiai, Shouichi Ohga, Junji Kishimoto, Koji Todaka, Ichiro Ieiri, Koh-Hei Sonoda","doi":"10.1007/s10384-024-01100-3","DOIUrl":null,"url":null,"abstract":"<p><strong>Purpose: </strong>To assess the safety and efficacy of ripasudil for retinopathy of prematurity (ROP).</p><p><strong>Study design: </strong>Phase 1/2, multicenter, open-label, single-arm, 12-week clinical trial.</p><p><strong>Methods: </strong>Infants born with gestational age (GA) of ≤ 32 weeks or weight of ≤ 1500 g with zone I or II, ≥ stage 1, ROP in both eyes were enrolled. Ripasudil eye drops were administered to patients in both eyes. Phase 1 was a dose-escalation study (once daily for 1 week, then twice daily for 2 weeks); an additional dosing up to 9 weeks was allowed if no safety issues occurred. In phase 2, ripasudil was administered twice daily for up to 12 weeks. Adverse events were assessed. The proportion of patients with type 1 ROP progression, number of days for type 1 ROP progression, and progression to the most advanced ROP stage were estimated.</p><p><strong>Results: </strong>Twenty-four infants were enrolled (phase 1, n = 3; phase 2, n = 21). Nineteen and four patients experienced systemic and ocular adverse events, respectively. Efficacy endpoints were not different between the ripasudil and historical control groups. However, in the GA ≤ 27 weeks subgroup, fewer patients progressed to type 1 ROP in the ripasudil than in the historical control group (P = 0.09). In the GA ≤ 27 weeks subgroups, the 25th percentile for the number of days for type 1 ROP progression was 22 days in the historical control group and 44 days in the ripasudil group.</p><p><strong>Conclusion: </strong>Ripasudil was safe and inhibited/delayed type 1 ROP progression, especially in infants with short GA.</p>","PeriodicalId":14563,"journal":{"name":"Japanese Journal of Ophthalmology","volume":null,"pages":null},"PeriodicalIF":2.1000,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Japanese Journal of Ophthalmology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s10384-024-01100-3","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/7/26 0:00:00","PubModel":"Epub","JCR":"Q2","JCRName":"OPHTHALMOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
Purpose: To assess the safety and efficacy of ripasudil for retinopathy of prematurity (ROP).
Study design: Phase 1/2, multicenter, open-label, single-arm, 12-week clinical trial.
Methods: Infants born with gestational age (GA) of ≤ 32 weeks or weight of ≤ 1500 g with zone I or II, ≥ stage 1, ROP in both eyes were enrolled. Ripasudil eye drops were administered to patients in both eyes. Phase 1 was a dose-escalation study (once daily for 1 week, then twice daily for 2 weeks); an additional dosing up to 9 weeks was allowed if no safety issues occurred. In phase 2, ripasudil was administered twice daily for up to 12 weeks. Adverse events were assessed. The proportion of patients with type 1 ROP progression, number of days for type 1 ROP progression, and progression to the most advanced ROP stage were estimated.
Results: Twenty-four infants were enrolled (phase 1, n = 3; phase 2, n = 21). Nineteen and four patients experienced systemic and ocular adverse events, respectively. Efficacy endpoints were not different between the ripasudil and historical control groups. However, in the GA ≤ 27 weeks subgroup, fewer patients progressed to type 1 ROP in the ripasudil than in the historical control group (P = 0.09). In the GA ≤ 27 weeks subgroups, the 25th percentile for the number of days for type 1 ROP progression was 22 days in the historical control group and 44 days in the ripasudil group.
Conclusion: Ripasudil was safe and inhibited/delayed type 1 ROP progression, especially in infants with short GA.
期刊介绍:
The Japanese Journal of Ophthalmology (JJO) was inaugurated in 1957 as a quarterly journal published in English by the Ophthalmology Department of the University of Tokyo, with the aim of disseminating the achievements of Japanese ophthalmologists worldwide. JJO remains the only Japanese ophthalmology journal published in English. In 1997, the Japanese Ophthalmological Society assumed the responsibility for publishing the Japanese Journal of Ophthalmology as its official English-language publication.
Currently the journal is published bimonthly and accepts papers from authors worldwide. JJO has become an international interdisciplinary forum for the publication of basic science and clinical research papers.