Observational Study to Assess Endpoint Operational Feasibility & Measurement Properties in Patients with Retinal Degeneration
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Retinitis Pigmentosa, Choroideremia, Stargardt Macular Dystrophy, Stargardt Disease. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Observational, Non-Interventional Study to Determine the Operational Feasibility and Measurement Properties of Endpoints in Patients with Retinal Degeneration
Overview
The Vision Research and Assessment Institute (VRAI) was established with the purpose of serving as a testing facility for efficacy endpoints for patients with Low Vision. The mission of the VRAI is to enable the highest quality, standardized efficacy testing of patients with visual impairment. The VRAI facilitates the development and refinement of existing endpoints specifically for testing patients with Low Vision.
Detailed description
The VRAI is a state-of-the-art research institute specifically for patients with low or very low vision due to diseases of the retina of the eye. The Institute is staffed by highly experienced Low Vision specialists and provides a comfortable, familiar environment for patient testing.
The study is a non-interventional prospective cohort study of up to 40 patients for each disease condition (Retinitis Pigmentosa, Choroideremia, Stargardt Macular Dystrophy, Geographic Atrophy from Age-related Macular Degeneration, X-linked Retinoschisis or other retinal dystrophies ).
Patients are scheduled for an initial visit where they are tested on a series of efficacy tests. Thereafter, patients will be invited to attend for a further visit up to two weeks and then periodically for up to one year. The objectives of the study are to evaluate how well patients use their vision, how the tests relate to activities of daily living, establish clinical meaningfulness, assess the impact of disease progression and the precision and accuracy of each test in the context of their eye condition.
Primary outcome measures
- Operational feasibility of testing moderate to profound vision impaired patients with various retinal dystrophies on a battery of visual assessments [Time frame: 3 months]
Secondary outcome measures (2)
- To quantify patient-based measurement properties of each test modality (for the purposes of endpoint qualification) [Time frame: 2 years]
- To quantify assessor-based measurement properties of each test modality (for the purposes of endpoint qualification) [Time frame: 2 years]
Eligibility criteria
Inclusion criteria
- Diagnosis of bilateral retinitis pigmentosa, choroideremia, Stargardt macular dystrophy, geographic atrophy from age-related macular degeneration, X-linked retinoschisis or other retinal dystrophies confirmed from previous eye examination records
- Best-corrected visual acuity between 20/70 and HM in at least one eye as tested with clinic-based visual acuity method
- Reasonably fluent in English
Exclusion criteria
- Cognitive impairment, memory loss or dementia sufficient in severity to preclude informed consent or in the opinion of the investigator would prevent satisfactory completion of some or all of the testing.
- Any circumstance that in the opinion of the investigator, would interfere with participation in, or compliance with the study protocol
- Current pregnancy as reported by patient
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 1 center
- Vision Research and Assessment Institute — Irvine
Identifiers
NCT: NCT06375239 · RTx-VRAI-NHS01