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

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，伴有视网膜色素变性的单侧或双侧囊样黄斑水肿的受试者将被纳入研究。然而，最多可以额外招募5名参与者，以取代可能在第6个月访问之前退出研究的参与者。