Clinical trial NCT04558983: A Natural History Study to Evaluate Functional and Anatomical Progression in Retinitis Pigmentosa
Official title: A Natural History Study to Evaluate Functional and Anatomical Progression in Retinitis Pigmentosa
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Retinitis Pigmentosa
Lead sponsor: Johns Hopkins University
Sponsor class: OTHER
First posted: 2020-09-22
Enrollment: 32
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients diagnosed with Retinitis Pigmentosa

Brief 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.


Eligibility criteria: 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

Primary outcomes:
measure: Change in mean macular sensitivity (dB) over time as assessed by microperimetry
timeFrame: Baseline, every six months up to 2 years
Secondary outcomes:
measure: Change in Best Corrected Visual Acuity (BVCA)
timeFrame: Baseline, every six months up to 2 years
measure: Change in Ellipsoid Zone (EZ) width
timeFrame: Baseline, every six months up to 2 years
measure: Change in Quality of Life survey metrics
timeFrame: Baseline, every year up to 2 years
measure: Change in mean retinal sensitivity
timeFrame: Baseline and at 2 years
measure: Correlation between change in visual functional and anatomical measures
timeFrame: Baseline, every six months up to 2 years
measure: Correlation between change in visual functional measures and Quality of Life survey metrics
timeFrame: Baseline, every year up to 2 years
Other outcomes:
measure: Correlation between baseline functional and anatomical measures
timeFrame: Baseline, up to 2 years
measure: Correlation between baseline functional measures and Quality of Life survey metrics
timeFrame: Baseline, up to 2 years
measure: Correlation between functional, anatomic and Quality of Life measures
timeFrame: Baseline, up to 2 years
measure: Proportion of eyes with ≥ 5 loci that show ≥ 6 decibels (dB) decline in mean macular sensitivity from baseline
timeFrame: Baseline, every six months up to 2 years
measure: Proportion of eyes with ≥ 5 loci that show ≥ 7 decibels (dB) decline in mean retinal sensitivity from baseline
timeFrame: Baseline and at 2 years
Locations:
city: Baltimore
country: United States
facility: Wilmer Eye Institute at Johns Hopkins University
state: Maryland
Study officials:
affiliation: Johns Hopkins University
name: Peter A Campochiaro, M.D.
role: PRINCIPAL_INVESTIGATOR
References: