Observational Study to Assess Endpoint Operational Feasibility & Measurement Properties in Patients with Retinal Degeneration

January 6, 2025 updated by: Ray Therapeutics, Inc.

Observational, Non-Interventional Study to Determine the Operational Feasibility and Measurement Properties of Endpoints in Patients with Retinal Degeneration

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.

Study Overview

Status

Recruiting

Conditions

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.

Study Type

Observational

Enrollment (Estimated)

120

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • California
      • Irvine, California, United States, 92612
        • Recruiting
        • Vision Research and Assessment Institute
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

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

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Retinitis Pigmentosa
Up to 40 patients with Retinitis Pigmentosa
Choroideremia
Up to 40 patients with Choroideremia
Stargardt Macular Dystrophy (Stargardt Disease)
Up to 40 patients with Stargardt Macular Dystrophy also known as Stargardt Disease
Geographic Atrophy from Age-related Macular Degeneration
Up to 40 patients with Geographic Atrophy from Age-related Macular Degeneration
X-linked Retinoschisis
Up to 40 patients with X-linked Retinoschisis
Other Retinal Dystrophies
Up to 40 patients with other retinal dystrophies

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Operational feasibility of testing moderate to profound vision impaired patients with various retinal dystrophies on a battery of visual assessments
Time Frame: 3 months
Determining how many patients in the visual range 20/70 - HM (depending upon indication) can perform specific tests of visual function with a measurable score.
3 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To quantify patient-based measurement properties of each test modality (for the purposes of endpoint qualification)
Time Frame: 2 years
Test-retest reliability on the same visit or on two different testing sessions on different days including determination of significant learning effects or other scoring variables that impact variability.
2 years
To quantify assessor-based measurement properties of each test modality (for the purposes of endpoint qualification)
Time Frame: 2 years
Intra- and inter-grader variability for scoring of tests by test graders/raters. Assessments incorporating independent graders/raters will be assessed for within session and across session scoring reliability to ensure consistent methods.
2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ray Therapeutics, Inc.

Collaborators

The Vision Research and Assessment Institute (VRAI)

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 5, 2024

Primary Completion (Estimated)

April 30, 2026

Study Completion (Estimated)

April 30, 2026

Study Registration Dates

First Submitted

April 15, 2024

First Submitted That Met QC Criteria

April 16, 2024

First Posted (Actual)

April 19, 2024

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

January 6, 2025

Last Verified

January 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RTx-VRAI-NHS01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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