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

NCT06375239 | Recruiting

• 1 other identifier: RTx-VRAI-NHS01
• Study Type: observational
• Target: 120
• Locations: 1 country
• Sites: 1

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.

Conditions

Retinitis Pigmentosa; Choroideremia; Stargardt Macular Dystrophy; Stargardt Disease; Geographic Atrophy from Age-related Macular Degeneration; X-lined Retinoschisis; Retinal Dystrophies

Outcome Measures

Primary Outcomes (1)

• 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 20/70 - HM (depending upon indication) can perform specific tests of visual function with a measurable score.

  3 months

Secondary Outcomes (2)

• To quantify patient-based measurement properties of each test modality (for the purposes of endpoint qualification)

  2 years

• To quantify assessor-based measurement properties of each test modality (for the purposes of endpoint qualification)

  2 years

Study Arms (6)

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

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Ray Therapeutics, Inc.: lead
• The Vision Research and Assessment Institute (VRAI): collaborator

Study Sites (1)

Vision Research and Assessment Institute

Irvine, California, 92612, United States

RECRUITING

MeSH Terms

Conditions

Retinitis Pigmentosa; Choroideremia; Stargardt Disease; Retinal Dystrophies

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Choroid Diseases; Uveal Diseases; Genetic Diseases, X-Linked; Macular Degeneration

Central Study Contacts

Shawn Yu, OD

CONTACT

949-264-3793; info@vr-ai.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 15, 2024
• First Posted: April 19, 2024
• Study Start: April 5, 2024
• Primary Completion: April 30, 2026
• Study Completion: April 30, 2026
• Last Updated: January 8, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)