Development and Evaluation of Functional Visual Field and Navigation Endpoints in Moderate to Profound Inherited Retinal Disease (DEFINE-IRD) (DEFINE-IRD)

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: Retinitis Pigmentosa; Retinal Dystrophies; Stargardt Disease; Stargardt Macular Dystrophy; X-linked Retinoschisis; Geographic Atrophy From Age-related Macular Degeneration

Detailed Description

This observational, non-interventional study is designed to assess both the operational feasibility and the measurement properties of selected visual function assessments in this population. The study will emphasize outcome measures that are sensitive to peripheral visual function and that reflect real-world functional abilities, including spatial orientation, mobility, and wayfinding.

• Study Type: Observational
• Enrollment (Estimated): 25

Contacts and Locations

• Study Contact: Name: Reem Jaber, MS, CGC; Phone Number: 949-264-3793; Email: info@vr-ai.com

Study Locations

• United States
  • California
    • Irvine, California, United States, 92614
      • Recruiting
      • Vision Research and Assessment Institute
      • Contact: Reem Jaber, MS, CGC; Phone Number: 949-264-3793; Email: info@vr-ai.com

Participation Criteria

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 or other retinal dystrophies.

Description

Inclusion Criteria:

• Diagnosis of bilateral retinitis pigmentosa (RP) or other retinal dystrophies impacting peripheral vision as confirmed from previous eye examination records
• Best-corrected visual acuity between 20/200 to HM in at least one eye.
• Reasonably fluent in English or Spanish

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

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
Retinitis Pigmentosa; Up to 25 patients with Retinitis Pigmentosa
Other Retinal Dystrophies; Up to 25 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	Determining how many patients in the visual range 20/200 - 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 assessor-based measurement properties of each test modality (for the purposes of endpoint qualification)	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
To quantify patient-based measurement properties of each test modality (for the purposes of endpoint qualification)	Test-rest reliability on the same visit or on two different testing sessions on different days including determination of significant learning effects of other scoring variables that impact variability.	2 years

Collaborators and Investigators

• Sponsor: Ray Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): February 19, 2026
• Primary Completion (Estimated): February 1, 2027
• Study Completion (Estimated): February 1, 2027

Study Registration Dates

• First Submitted: March 24, 2026
• First Submitted That Met QC Criteria: March 24, 2026
• First Posted (Actual): March 31, 2026

Study Record Updates

• Last Update Posted (Actual): March 31, 2026
• Last Update Submitted That Met QC Criteria: March 24, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Eye Diseases; Eye Diseases, Hereditary; Retinal Diseases; Retinal Degeneration; Macular Degeneration; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Stargardt Disease; Retinitis Pigmentosa; Retinal Dystrophies; Retinoschisis
• Other Study ID Numbers: RTx-VRAI-NHS02 (DEFINE-IRD)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No