Retinitis PigmentosaNCT06375239
Observational Study to Assess Endpoint Operational Feasibility & Measurement Properties in Patients with Retinal Degeneration
An observational study of Retinitis Pigmentosa and Choroideremia, run by Ray Therapeutics, Inc. at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Retinitis Pigmentosa, Choroideremia, Stargardt Macular Dystrophy
- How many people
- 120 participants
- Where
- 1 siteUnited States
- Study dates
- 5 Apr 2024to 30 Apr 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Vision Research and Assessment Institute
Irvine, California, 92612
Shawn Yu, OD, 949-264-3793, info@vr-ai.com
About this study
Official title: 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.
Read the 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.
- Conditions
- Retinitis Pigmentosa, Choroideremia, Stargardt Macular Dystrophy, Stargardt Disease, Geographic Atrophy from Age-related Macular Degeneration, X-lined Retinoschisis
- Medical subject headings
- Retinitis Pigmentosa, Choroideremia, Stargardt Disease, Retinal Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 3 months
Secondary outcomes (2)
To quantify patient-based measurement properties of each test modality (for the purposes of endpoint qualification)
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.
Measured: 2 years
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.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Apr 2024
- Study start5 Apr 2024
- Primary completion30 Apr 2026 (estimated)
- Study completion30 Apr 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis PigmentosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ray Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- The Vision Research and Assessment Institute (VRAI)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06375239
- Sponsor's protocol ID
- RTx-VRAI-NHS01
- Status last verified
- Jan 2025
- First submitted
- 15 Apr 2024
- Last updated
- 8 Jan 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Observational, Non-Interventional Study to Determine the Operational Feasibility and Measurement Properties of Endpoints in Patients with Retinal Degeneration
- ClinicalTrials.gov ID
- NCT06375239
- Acronym / sponsor ID
- RTx-VRAI-NHS01
- Conditions
- Retinitis Pigmentosa, Choroideremia, Stargardt Macular Dystrophy, Stargardt Disease, Geographic Atrophy from Age-related Macular Degeneration, X-lined Retinoschisis
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- Ray Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- The Vision Research and Assessment Institute (VRAI)
- Study start
- 5 Apr 2024 (actual)
- Primary completion
- 30 Apr 2026 (estimated)
- Study completion
- 30 Apr 2026 (estimated)
- First posted
- 19 Apr 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| 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 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
2 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jan 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Retinitis Pigmentosa and Choroideremia, run by Ray Therapeutics, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 8 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Shawn Yu, OD (949-264-3793, info@vr-ai.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Operational feasibility of testing moderate to profound vision impaired patients with various retinal dystrophies on a battery of visual assessments (time frame: 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jan 2025.