Clinical trial NCT06375239: Observational Study to Assess Endpoint Operational Feasibility & Measurement Properties in Patients with Retinal Degeneration
Official title: Observational, Non-Interventional Study to Determine the Operational Feasibility and Measurement Properties of Endpoints in Patients with Retinal Degeneration
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Retinitis Pigmentosa
Choroideremia
Stargardt Macular Dystrophy
Stargardt Disease
Geographic Atrophy from Age-related Macular Degeneration
X-lined Retinoschisis
Retinal Dystrophies
Lead sponsor: Ray Therapeutics, Inc.
Sponsor class: INDUSTRY
Collaborators: name: The Vision Research and Assessment Institute (VRAI)
First posted: 2024-04-19
Enrollment: 120
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 with moderate to profound vision impairment diagnosed with retinitis pigmentosa, choroideremia, or Stargardt macular dystrophy (also known as Stargardt disease), geographic atrophy from age-related macular degeneration, X-linked retinoschisis or other retinal dystrophies

Brief summary: 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.

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

Primary outcomes:
measure: Operational feasibility of testing moderate to profound vision impaired patients with various retinal dystrophies on a battery of visual assessments
timeFrame: 3 months
Secondary outcomes:
measure: To quantify patient-based measurement properties of each test modality (for the purposes of endpoint qualification)
timeFrame: 2 years
measure: To quantify assessor-based measurement properties of each test modality (for the purposes of endpoint qualification)
timeFrame: 2 years
Other outcomes:
Locations:
city: Irvine
country: United States
facility: Vision Research and Assessment Institute
state: California
Study officials:
References: