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: 8938376
• 负责人: Catherine Cukras
• 金额: $ 10.43万
• 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/8938376
• 关键词: Adverse event; Age; Bilateral; Blindness; Categories; Cell Death; Cells; Childhood; Clinical Trials; Cystoid Macular Edema; Development; Disease; Dose; Electroretinography; Enrollment; Etiology; Eye; Hereditary Disease; Immune; Inflammation; Inflammatory; Institutes; Measurable; Measurement; Measures; Microglia; Minocycline; Optical Coherence Tomography; Oral; Outcome; Outcome Measure; Participant; Patients; Peripheral; Phase; Pilot Projects; Process; Production; Recruitment Activity; 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; macula; ocular surface; open label; primary outcome; prospective; 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.

最初将入组5名12岁及以上的受试者，他们患有与视网膜色素变性相关的单侧或双侧黄斑囊样水肿。但是，最多可额外入组5例受试者，以替换可能在第6个月访视前退出研究的受试者。 设计图：这是一项初步、单中心、非对照、开放标签、前瞻性、I/II期临床试验，旨在评价米诺环素作为继发于RP的CME的潜在治疗方法。在开始使用研究药物（IP）之前，将建立持续两个月的治疗前阶段，以评估CME的解剖变异性以及作为疾病自然史一部分的其他可测量参数的变异性。受试者将接受口服剂量为100 mg（或适当的体重调整儿科剂量）的米诺环素，每日两次，持续12个月。将有一个共同的终止日期，即最后一名招募的受试者接受了12个月的IP。在研究早期招募的受试者将继续服用IP，并每两个月进行一次随访，直至共同终止日期。每次就诊时，参与者将接受视力测量，并接受光学相干断层扫描（OCT）测试以测量视网膜厚度。还将收集中央视野敏感度全视野视网膜电图（ERG）和微视野（MP-1）的测量结果。 结果测量：主要结果是与治疗前值相比，6个月时研究眼中基于OCT测量的CME变化。次要结局包括OCT厚度变化、ERG测试明视和暗视反应幅度变化、微视野变化、6个月和12个月时HVF 30-2视野测试测量的视野变化（与治疗前值相比）以及12个月时OCT CME变化（与治疗前值相比）。将分析治疗前测量结果，以测量CME的自然变异性以及功能测试的变异性。安全性结局将包括不良事件（AE）的数量和严重程度。眼部安全性结局将通过视力变化、眼表变化、眼内炎症和与RP自然进展不一致的任何其他眼部变化来指示。