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

Retinitis Pigmentosa Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04558983
• Other study ID #: IRB00227603
• Status: Recruiting
• Start date: June 11, 2020
• Est. completion date: January 2025

Study information

• Verified date: March 2024
• Source: Johns Hopkins University
• Contact: Gulnar Hafiz, M.D., M.P.H.
• Phone: 4105020768
• Email: ghafiz[@]jhmi.edu
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 130
• Est. completion date: January 2025
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Retinitis
• Retinitis Pigmentosa

Locations

Country	Name	City	State
United States	Wilmer Eye Institute at Johns Hopkins University	Baltimore	Maryland

Sponsors (1)

Lead Sponsor	Collaborator
Johns Hopkins University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Correlation between baseline functional and anatomical measures	Visual function parameters at baseline 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 at baseline such as Ellipsoid width (measurement on Optical Coherence Tomography)	Baseline, up to 2 years
Other	Correlation between baseline functional measures and Quality of Life survey metrics	Visual function parameters at baseline 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 baseline Quality of Life survey metrics (Scored using National Eye Institute's Visual Function Questionnaire, NEI-VFQ-25)	Baseline, up to 2 years
Other	Correlation between functional, anatomic and Quality of Life measures	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), anatomical parameters such as Ellipsoid width (measurement on Optical Coherence Tomography) Quality of Life survey metrics (Scored using National Eye Institute's Visual Function Questionnaire, NEI-VFQ-25) will be correlated.	Baseline, up to 2 years
Other	Proportion of eyes with = 5 loci that show = 6 decibels (dB) decline in mean macular sensitivity from baseline	This will be measured using MAIA microperimetry	Baseline, every six months up to 2 years
Other	Proportion of eyes with = 5 loci that show = 7 decibels (dB) decline in mean retinal sensitivity from baseline	This will be measured by static Octopus perimetry using 30-2 program with III target	Baseline and at 2 years
Primary	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	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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