A Natural History Study to Evaluate Functional and Anatomical Progression in Retinitis Pigmentosa
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 32
- 试验地点
- 1
- 主要终点
- Change in mean macular sensitivity (dB) over time as assessed by microperimetry
研究概览
简要总结
This study will assess the progression of RP as seen on newer modalities including spectral-domain optical coherence (SD-OCT) and macular assessment integrity (MAIA) microperimetry to evaluate disease status. Understanding the natural history of the disease is not only essential to monitoring and comparing patient populations in clinical trials. It is also fundamental in the predevelopment phase in order to optimize the study duration needed to observe a statistically significant outcome. Furthermore, since the progression of RP is usually slow, relying on traditional tests can take an unfeasible length of time to observe any meaningful changes and assess therapeutic efficacy for new drugs. Therefore, the results of this study will be beneficial in establishing reliable endpoints and outcome measures for future clinical trials. Such outcome measures may be able to detect treatment response with more precision. More importantly, investigators may be able to detect changes early enough to prevent irreversible vision loss.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —(Adult, Older Adult)
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 18 years or older
- •Patients diagnosed with Retinitis Pigmentosa
- •Ability to provide informed consent
- •Ability to authorize use and disclosure of protected health information
排除标准
- •Concomitant ocular pathology that limits central macular function, including but not limited to age-related macular degeneration, diabetic retinopathy, and retinal vein occlusion
- •If EZ width ≤200µm
结局指标
主要结局
Change in mean macular sensitivity (dB) over time as assessed by microperimetry
时间窗: Baseline, every six months up to 2 years
Microperimetry (MAIA) will be used to test whether there is a change in sensitivity (dB) in the macula
次要结局
- Change in mean retinal sensitivity(Baseline and at 2 years)
- Correlation between change in visual functional measures and Quality of Life survey metrics(Baseline, every year up to 2 years)
- Change in Best Corrected Visual Acuity (BVCA)(Baseline, every six months up to 2 years)
- Change in Ellipsoid Zone (EZ) width(Baseline, every six months up to 2 years)
- Change in Quality of Life survey metrics(Baseline, every year up to 2 years)
- Correlation between change in visual functional and anatomical measures(Baseline, every six months up to 2 years)
