Pilot Study to Evaluate Oral Minocycline in the Treatment of Cystoid Macular Edema Associated with Retinitis Pigmentosa

评估口服米诺环素治疗与视网膜色素变性相关的囊样黄斑水肿的初步研究

• 批准号: 9796729
• 负责人: Catherine Cukras
• 金额: $ 5.71万
• 依托单位: NATIONAL EYE INSTITUTE
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/9796729
• 关键词: Adverse event; Age; Anatomy; Bilateral; Blindness; Categories; Cell Death; Cells; Childhood; Clinical Trials; Cystoid Macular Edema; Development; Disease; Dose; Electroretinography; Enrollment; Etiology; Eye; Genetic Diseases; Immune; Inflammation; Inflammatory; Institutes; Investigation; Measurable; Measurement; Measures; Microglia; Minocycline; Optical Coherence Tomography; Oral; Outcome; Outcome Measure; Participant; Patient Recruitments; Patients; Peripheral; Phase; Pilot Projects; Process; Production; Retina; Retinal; Retinitis Pigmentosa; Safety; Secondary to; Severities; Testing; Thick; Vision; Visit; Visual Acuity; Visual Fields; Weight; base; central visual field; constriction; design; disease natural history; field study; macula; ocular surface; open label; primary outcome; prospective; recruit; response; secondary outcome

Five participants, ages 12 and older, with unilateral or bilateral cystoid macular edema associated with retinitis pigmentosa will be enrolled initially. However, up to an additional five participants may be enrolled to replace participants who may withdraw from the study prior to reaching the Month 6 visit. Design: This is a pilot, single-center, uncontrolled, open-label, prospective, Phase 1/2 clinical trial to evaluate minocycline as a potential treatment for CME secondary to RP. A pre-treatment phase lasting two months will be instituted prior to investigational product (IP) initiation to assess the anatomical variability of CME as well as variability of other measurable parameters as part of the natural history of the disease. Participants will receive an oral dose of 100 mg (or appropriate weight adjusted pediatric dose) of minocycline twice daily for 12 months. There will be a common termination date, which will take place when the last recruited participant has received 12 months of IP. Participants who were recruited in the earlier part of the study will continue taking IP and be followed every two months until the common termination date. At each visit, participants will have visual acuity measured and will undergo optical coherence tomography (OCT) testing to measure retinal thickness. Measures of central visual field sensitivity full-field electroretinograms (ERG) and microperimetry (MP-1) will also be collected. Outcome Measures: The primary outcome is the change in CME based on OCT measurements in the study eye at 6 months compared to pre-treatment values. Secondary outcomes include changes in OCT thickness, changes in amplitude of photopic and scotopic responses on ERG testing, changes in microperimetry, and changes in visual field as measured by HVF 30-2 visual field testing at 6 months and 12 months compared to pre-treatment values, as well as CME changes on OCT at 12 months compared to pre-treatment values. Pre-treatment measurements will be analyzed to measure the natural variability of the CME as well as to measure the variability of the functional testing. Safety outcomes will include the number and severity of adverse events (AEs). Ocular safety outcomes will be indicated by changes in visual acuity, ocular surface changes, intraocular inflammation and any other ocular changes not consistent with the natural progression of RP.

5名年龄在12岁及以上，单侧或双侧黄斑囊样水肿伴视网膜色素变性的参与者最初将被纳入研究。然而，最多可以再招募5名参与者，以取代可能在6个月访问之前退出研究的参与者。 设计：这是一项先导性、单中心、非对照、开放标签、前瞻性的1/2期临床试验，旨在评估米诺环素作为继发于RP的CME的潜在治疗方法。为期两个月的治疗前阶段将在研究产品(IP)开始之前启动，以评估CME的解剖可变性以及作为疾病自然历史的一部分的其他可测量参数的可变性。参与者将在12个月内每天两次口服100毫克的米诺环素(或适当的儿童体重调整剂量)。将有一个共同的终止日期，该日期将发生在最后一名受聘参与者收到12个月知识产权的时候。在研究早期招募的参与者将继续接受IP治疗，并每两个月进行一次跟踪，直到共同终止日期。每次就诊时，参与者都将接受视力测量，并接受光学相干断层扫描(OCT)测试，以测量视网膜厚度。中心视野敏感度、全场视网膜电图(ERG)和微视野计(MP-1)的测量也将被收集。 观察指标：主要结果是与治疗前相比，研究眼在6个月时基于OCT测量的CME的变化。次要结果包括治疗6个月和12个月时OCT厚度的变化、ERG测试中明视和暗视反应幅度的变化、微视野的变化和由HVF 30-2视野测试测量的视野变化，以及与治疗前相比OCT 12个月时CME的变化。将对前处理测量进行分析，以测量CME的自然可变性以及功能测试的可变性。安全结果将包括不良事件的数量和严重程度。视力的变化、眼表的变化、眼内炎症和任何其他与RP自然发展不一致的眼部变化将表明眼的安全性结果。