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: 9556040
• 负责人: Catherine Cukras
• 金额: $ 5.51万
• 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/9556040
• 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; Hereditary Disease; Immune; Inflammation; Inflammatory; Institutes; Investigation; 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; field study; macula; ocular surface; open label; primary outcome; prospective; response; secondary outcome; Adverse event; Age; Anatomy; 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; Investigation; 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; field study; 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.

最初将招募五名12岁及12岁及12岁的参与者，具有与视网膜炎色素炎相关的单侧或双侧囊性黄斑水肿。但是，最多可能会招募另外五名参与者，以取代可能在进入第6个月访问之前退出研究的参与者。 设计：这是一项飞行员，单中心，不受控制的，开放标签，前瞻性，第1/2期临床试验，可评估米诺环素作为继发于RP的CME的潜在治疗方法。在研究产品（IP）启动之前将建立持续两个月的预处理阶段，以评估CME的解剖学变异性以及其他可测量参数的变异性，这是疾病自然史的一部分。参与者将每天两次接受100 mg（或适当的体重调节小儿剂量）的口服剂量，持续12个月。将会有一个共同的终止日期，这将发生在最后一个招募的参与者收到12个月的IP时发生的。在研究的早期招募的参与者将继续使用IP，并每两个月遵循每两个月的终止日期。每次访问时，参与者将测量视力，并将进行光学相干断层扫描（OCT）测试以测量视网膜厚度。还将收集中央视野灵敏度全场电图（ERG）和微量工（MP-1）的度量。 结果指标：主要结果是基于研究眼中的OCT测量值与治疗前值相比，CME的变化是基于OCT测量值的变化。次要结果包括OCT厚度的变化，ERG测试中的光波器和SCOTOPIC响应幅度的变化，微量工的变化以及与预处理预处理值相比，HVF 30-2在6个月和12个月中通过HVF 30-2的视野测试所测量的视野变化，以及与预处理相比的10个月的CME变化。将分析预处理测量值，以衡量CME的自然变异性以及测量功能测试的可变性。安全结果将包括不良事件（AES）的数量和严重性。视敏度，眼表面变化，眼内炎症和任何其他眼睛变化与RP的自然进展不一致，眼部安全结果将表明。