A Natural History Study to Evaluate Functional and Anatomical Progression in Retinitis Pigmentosa

Study Overview
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Brief Summary
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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.

Official Title
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A Natural History Study to Evaluate Functional and Anatomical Progression in Retinitis Pigmentosa

Conditions
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Retinitis Pigmentosa

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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 Exclusion Criteria: 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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Retinitis Pigmentosa<br>Patients with Retinitis Pigmentosa | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in mean macular sensitivity (dB) over time as assessed by microperimetry | Microperimetry (MAIA) will be used to test whether there is a change in sensitivity (dB) in the macula | Baseline, every six months up to 2 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Best Corrected Visual Acuity (BVCA) | Scoring will be determined by the number of letters gained or lost per month using Early Treatment Diabetic Retinopathy Study (ETDRS) Letter Score and visual acuity score together with an overall score range of 0 to 20/20 where 0 is the worst vision and 20/20 is the best. | Baseline, every six months up to 2 years | 
| Change in Ellipsoid Zone (EZ) width | This will be assessed by spectral domain optical coherence tomography (SD-OCT) | Baseline, every six months up to 2 years | 
| Change in Quality of Life survey metrics | Scoring will be determined by the National Eye Institute's Visual Function Questionnaire (NEI-VFQ-25). It has 25 question elements each with score ranging from 1(excellent) to 6(very poor), therefore a total minimum score of 25 and maximum score 150. | Baseline, every year up to 2 years | 
| Change in mean retinal sensitivity | Static Octopus Perimetry will be used to test whether there is a change in mean retinal sensitivity over time using its 30-2 program with III target | Baseline and at 2 years | 
| Correlation between change in visual functional and anatomical measures | Change in visual function parameters such as Best Corrected Visual acuity (measured using ETDRS and visual acuity scale), mean macular sensitivity (quantified using MAIA microperimetry), mean retinal sensitivity (quantified using static Octopus perimetry) will be correlated to anatomical parameters such as Ellipsoid width (measurement on Optical Coherence Tomography) | Baseline, every six months up to 2 years | 
| Correlation between change in visual functional measures and Quality of Life survey metrics | Change in Quality of Life survey metrics (Scored using National Eye Institute's Visual Function Questionnaire, NEI-VFQ-25) will be compared to visual function parameters such as Best Corrected Visual acuity (measured using ETDRS and visual acuity scale), mean macular sensitivity (quantified using MAIA microperimetry), mean retinal sensitivity (quantified using static Octopus perimetry) | Baseline, every year up to 2 years |