Retinitis PigmentosaNCT04558983
A Natural History Study to Evaluate Functional and Anatomical Progression in Retinitis Pigmentosa
An observational study of Retinitis Pigmentosa, run by Johns Hopkins University at 1 site in United States.
Trial summary
This study will assess the progression of RP as seen on newer modalities including spectral-domain optical coherence (SD-OCT) and macular assessment integrity (MAIA) microperimetry to evaluate disease status. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Retinitis Pigmentosa
- How many people
- 32 participants
- Where
- 1 siteUnited States
- Study dates
- 11 Jun 2020to 20 Dec 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
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 diagnosed with Retinitis Pigmentosa
Who may be able to join
- Age 18 years or older
- Patients diagnosed with Retinitis Pigmentosa
- Ability to provide informed consent
- Ability to authorize use and disclosure of protected health information
Who cannot join
- Concomitant ocular pathology that limits central macular function, including but not limited to age-related macular degeneration, diabetic retinopathy, and retinal vein occlusion
- If EZ width ≤200µm
Trial locations and contacts
1 site in 1 country.
United States1 site
Wilmer Eye Institute at Johns Hopkins University
Baltimore, Maryland, 21287
About this study
This study will assess the progression of RP as seen on newer modalities including spectral-domain optical coherence (SD-OCT) and macular assessment integrity (MAIA) microperimetry to evaluate disease status. Understanding the natural history of the disease is not only essential to monitoring and comparing patient populations in clinical trials. It is also fundamental in the predevelopment phase in order to optimize the study duration needed to observe a statistically significant outcome. Furthermore, since the progression of RP is usually slow, relying on traditional tests can take an unfeasible length of time to observe any meaningful changes and assess therapeutic efficacy for new drugs. Therefore, the results of this study will be beneficial in establishing reliable endpoints and outcome measures for future clinical trials. Such outcome measures may be able to detect treatment response with more precision. More importantly, investigators may be able to detect changes early enough to prevent irreversible vision loss.
- Conditions
- Retinitis Pigmentosa
- Medical subject headings
- Retinitis Pigmentosa
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 | Patients with Retinitis Pigmentosa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in mean macular sensitivity (dB) over time as assessed by microperimetry
Microperimetry (MAIA) will be used to test whether there is a change in sensitivity (dB) in the macula
Measured: Baseline, every six months up to 2 years
Secondary outcomes (6)
Change in Best Corrected Visual Acuity (BVCA)
Scoring will be determined by the number of letters gained or lost per month using Early Treatment Diabetic Retinopathy Study (ETDRS) Letter Score and visual acuity score together with an overall score range of 0 to 20/20 where 0 is the worst vision and 20/20 is the best.
Measured: Baseline, every six months up to 2 years
Change in Ellipsoid Zone (EZ) width
This will be assessed by spectral domain optical coherence tomography (SD-OCT)
Measured: Baseline, every six months up to 2 years
Change in Quality of Life survey metrics
Scoring will be determined by the National Eye Institute's Visual Function Questionnaire (NEI-VFQ-25). It has 25 question elements each with score ranging from 1(excellent) to 6(very poor), therefore a total minimum score of 25 and maximum score 150.
Measured: Baseline, every year up to 2 years
Change in mean retinal sensitivity
Static Octopus Perimetry will be used to test whether there is a change in mean retinal sensitivity over time using its 30-2 program with III target
Measured: Baseline and at 2 years
Correlation between change in visual functional and anatomical measures
Change in visual function parameters such as Best Corrected Visual acuity (measured using ETDRS and visual acuity scale), mean macular sensitivity (quantified using MAIA microperimetry), mean retinal sensitivity (quantified using static Octopus perimetry) will be correlated to anatomical parameters such as Ellipsoid width (measurement on Optical Coherence Tomography)
Measured: Baseline, every six months up to 2 years
Correlation between change in visual functional measures and Quality of Life survey metrics
Change in Quality of Life survey metrics (Scored using National Eye Institute's Visual Function Questionnaire, NEI-VFQ-25) will be compared to visual function parameters such as Best Corrected Visual acuity (measured using ETDRS and visual acuity scale), mean macular sensitivity (quantified using MAIA microperimetry), mean retinal sensitivity (quantified using static Octopus perimetry)
Measured: Baseline, every year up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2020
- Study start11 Jun 2020
- Primary completion20 Dec 2024
- Study completion20 Dec 2024
Why it stopped: The study stopped because patients dropped out of the study to participate in the interventional trials. With interventional trials present there is very little interest in participating in a Natural Histoy study.
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.
- More trials run by Johns Hopkins UniversityThe sponsor's other registered studies.
- 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
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Johns Hopkins University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Peter A Campochiaro, M.D., Principal investigator, Johns Hopkins University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04558983
- Sponsor's protocol ID
- IRB00227603
- Status last verified
- Dec 2024
- First submitted
- 16 Sep 2020
- Last updated
- 27 Dec 2024
- 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 27 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Natural History Study to Evaluate Functional and Anatomical Progression in Retinitis Pigmentosa
- ClinicalTrials.gov ID
- NCT04558983
- Acronym / sponsor ID
- IRB00227603
- Conditions
- Retinitis Pigmentosa
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: The study stopped because patients dropped out of the study to participate in the interventional trials. With interventional trials present there is very little interest in participating in a Natural Histoy study.
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Johns Hopkins UniversityUniversity, hospital or other
- Study start
- 11 Jun 2020 (actual)
- Primary completion
- 20 Dec 2024 (actual)
- Study completion
- 20 Dec 2024 (actual)
- First posted
- 22 Sep 2020 (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.
This study will assess the progression of RP as seen on newer modalities including spectral-domain optical coherence (SD-OCT) and macular assessment integrity (MAIA) microperimetry to evaluate disease status. Understanding the natural history of the disease is not only essential to monitoring and comparing patient populations in clinical trials. It is also fundamental in the predevelopment phase in order to optimize the study duration needed to observe a statistically significant outcome. Furthermore, since the progression of RP is usually slow, relying on traditional tests can take an unfeasible length of time to observe any meaningful changes and assess therapeutic efficacy for new drugs. Therefore, the results of this study will be beneficial in establishing reliable endpoints and outcome measures for future clinical trials. Such outcome measures may be able to detect treatment response with more precision. More importantly, investigators may be able to detect changes early enough to prevent irreversible vision loss.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Retinitis Pigmentosa | – | –Patients with Retinitis Pigmentosa |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients diagnosed with Retinitis Pigmentosa
Key inclusion criteria (4 listed)
- Age 18 years or older
- Patients diagnosed with Retinitis Pigmentosa
- Ability to provide informed consent
- Ability to authorize use and disclosure of protected health information
Key exclusion criteria (2 listed)
- Concomitant ocular pathology that limits central macular function, including but not limited to age-related macular degeneration, diabetic retinopathy, and retinal vein occlusion
- If EZ width ≤200µm
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
Change in mean macular sensitivity (dB) over time as assessed by microperimetry
Time frame: Baseline, every six months up to 2 years
Microperimetry (MAIA) will be used to test whether there is a change in sensitivity (dB) in the macula
6 secondary outcomes
Change in Best Corrected Visual Acuity (BVCA)
Time frame: Baseline, every six months up to 2 years
Change in Ellipsoid Zone (EZ) width
Time frame: Baseline, every six months up to 2 years
Change in Quality of Life survey metrics
Time frame: Baseline, every year up to 2 years
Change in mean retinal sensitivity
Time frame: Baseline and at 2 years
Correlation between change in visual functional and anatomical measures
Time frame: Baseline, every six months up to 2 years
Correlation between change in visual functional measures and Quality of Life survey metrics
Time frame: Baseline, every year up to 2 years
Results and safety
The study was stopped early (reason given: The study stopped because patients dropped out of the study to participate in the interventional trials. With interventional trials present there is very little interest in participating in a Natural Histoy study.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Dec 2024.
- 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. This study is not recruiting at the moment.
Peter A Campochiaro, M.D. principal investigator
Johns Hopkins University
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, run by Johns Hopkins University 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 4 inclusion and 2 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: stopped early, as of the sponsor's last update on 27 Dec 2024. 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?
The study was stopped early (reason given: The study stopped because patients dropped out of the study to participate in the interventional trials. With interventional trials present there is very little interest in participating in a Natural Histoy study.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in mean macular sensitivity (dB) over time as assessed by microperimetry (time frame: Baseline, every six months up to 2 years). 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 27 Dec 2024.