设计中期AMD的下一代临床试验——一个共识驱动的、务实的、概念证明的早期干预研究。

IF 3 2区医学 Q1 OPHTHALMOLOGY

Experimental eye research Pub Date : 2025-03-14 DOI:10.1016/j.exer.2025.110340

Robyn H. Guymer , Philip J. Rosenfeld , Justis P. Ehlers , Mayssa Attar , Hao Chen , Frederick Ferris , James G. Fujimoto , Michael Ip , Maximilian Pfau , Marlene Saßmannshausen , Jie Shen , David N. Zacks , Tavé van Zyl , Zhichao Wu , Glenn J. Jaffe

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引用次数: 0

摘要

对老年性黄斑变性的病理生理学的深入了解已经产生了减缓视力丧失的潜在治疗策略。作为AMD临床试验的替代终点，结构成像生物标志物和新的功能结局都在深入研究中，它们是评估疾病进展和跟踪治疗反应所需的新颗粒工具。这些进步首次创造了在AMD早期干预的可能性，并在临床试验可行的时间框架内衡量成功。在2024年Ryan黄斑研究倡议（RIMR）会议上，达成了一项共识，即设计一项假设的、实用的、概念验证的、为期24个月的早期干预研究，以确定干预对中度AMD （iAMD）患者的疗效。虽然需要进一步的研究，使用自然历史队列和干预试验来验证所提出的试验设计和终点，但本文提出了AMD早期临床试验的专家共识框架，我们希望这对治疗开发人员和监管机构也有帮助。需要可行的临床试验策略来开发治疗方法，在发展为威胁视力的晚期AMD之前减缓AMD的进展；这份协商一致文件应有助于实现这一目标。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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Designing the next generation of clinical trials in intermediate AMD-a consensus driven, pragmatic, proof of concept early intervention study

A greater understanding of the pathophysiology of AMD has yielded potential therapeutic strategies for slowing vision loss. Structural imaging biomarkers and novel functional outcomes, both under intensive study as surrogate endpoints in AMD clinical trials, are new granular tools needed to assess disease progression and follow response to therapy. These advances have created, for the first time, the possibility to intervene early in AMD, and to measure success in a time frame feasible for clinical trials. At the 2024 Ryan Initiative for Macular Research (RIMR) meeting, a consensus was reached for design of a hypothetical, pragmatic, proof of concept, 24-month, early intervention study to determine the efficacy of an intervention in subjects with intermediate AMD (iAMD). Although further research is needed, using natural history cohorts and intervention trials to validate the proposed trial design and endpoints, this paper presents an AMD expert consensus framework for early AMD clinical trials, that we hope will also be useful for therapeutic developers and regulators. Viable clinical trial strategies are needed to develop treatments that slow the progression of AMD prior to the development of vision-threatening late AMD; this consensus document should aid in reaching this goal.

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来源期刊

Experimental eye research 医学-眼科学

CiteScore

6.80

自引率

5.90%

发文量

323

审稿时长

66 days

期刊介绍： The primary goal of Experimental Eye Research is to publish original research papers on all aspects of experimental biology of the eye and ocular tissues that seek to define the mechanisms of normal function and/or disease. Studies of ocular tissues that encompass the disciplines of cell biology, developmental biology, genetics, molecular biology, physiology, biochemistry, biophysics, immunology or microbiology are most welcomed. Manuscripts that are purely clinical or in a surgical area of ophthalmology are not appropriate for submission to Experimental Eye Research and if received will be returned without review.