Ignite Creation Date:
2024-05-06 @ 12:47 PM
Last Modification Date:
2024-10-26 @ 1:04 PM
Study NCT ID:
NCT03845218
Status:
COMPLETED
Last Update Posted:
2023-11-15
First Post:
2019-02-16
Brief Title:
Retinitis Pigmentosa Clinical Measures and Repeatability Testing of Potential Outcome Measures
Sponsor:
National Eye Institute NEI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
A Survey Study of Retinitis Pigmentosa RP Clinical Measures and Repeatability Testing of Potential Outcome Measures
Status:
COMPLETED
Status Verified Date:
2024-08-15
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Background
Retinitis pigmentosa RP is a group of blinding eye diseases It is caused mostly by mutations in photoreceptor-expressed genes RP affects about 2 million people globally There is no cure but treatment and diagnosis can be guided by certain tests Researchers want to see how well these tests capture stages of RP
Objectives
To find out how well certain tests track changes in retinitis pigmentosa
Eligibility
People ages 12 and older with RP
Healthy volunteers ages 18 and older with no evidence of RP
Design
Participants will be screened in another protocol
Participants will have 2 visits about 6 weeks apart Both will include all the tests below Each visit will last 5-6 hours or a visit can be split into 2 days
Participants will give their medical and eye history
Participants will have an eye exam Their pupils will be dilated with eye drops
Participants will give blood samples
Pictures of participants retinas will be taken Their retinas will be measured
Participants will take several eye tests They will
Sit in a dark room and press a button when they see lights
View a bright background then press a button when they see lights
Look into a bowl and press a button when they see lights
Sit in the dark with their eyes patched Then they will take eye-numbing drops and wear contacts as lights flash A small electrode taped to their forehead will record signals from their retinas
Minors will give written consent to stay in the study when they turn 18 After the study ends they may also be asked to give consent for researchers to continue to use their study information
Retinitis pigmentosa RP is a group of blinding eye diseases It is caused mostly by mutations in photoreceptor-expressed genes RP affects about 2 million people globally There is no cure but treatment and diagnosis can be guided by certain tests Researchers want to see how well these tests capture stages of RP
Objectives
To find out how well certain tests track changes in retinitis pigmentosa
Eligibility
People ages 12 and older with RP
Healthy volunteers ages 18 and older with no evidence of RP
Design
Participants will be screened in another protocol
Participants will have 2 visits about 6 weeks apart Both will include all the tests below Each visit will last 5-6 hours or a visit can be split into 2 days
Participants will give their medical and eye history
Participants will have an eye exam Their pupils will be dilated with eye drops
Participants will give blood samples
Pictures of participants retinas will be taken Their retinas will be measured
Participants will take several eye tests They will
Sit in a dark room and press a button when they see lights
View a bright background then press a button when they see lights
Look into a bowl and press a button when they see lights
Sit in the dark with their eyes patched Then they will take eye-numbing drops and wear contacts as lights flash A small electrode taped to their forehead will record signals from their retinas
Minors will give written consent to stay in the study when they turn 18 After the study ends they may also be asked to give consent for researchers to continue to use their study information
Detailed Description:
Precis
Objective The objective of this study is to investigate the nature of photoreceptor dysfunction in retinitis pigmentosa RP patients using focal static and kinetic psychophysical tests to develop functional outcome measures for the clinical trial study in RP Correlation of novel spatial functional maps with other functional measures such as visual acuity and multifocal electroretinography will help provide a full description of functional change Employing new imaging methods to visualize and analyze structural changes in the retina will allow for the evaluation of structural changes that underlie disease progression Developing these measures has the potential to advance the field by elucidating the process of photoreceptor degeneration as well as being a scaffold for which candidate therapies could be trialed
Study Population Up to 120 participants with a diagnosis of RP will be enrolled Up to
30 healthy volunteers will also be enrolled for a total accrual ceiling of 150
Design This is a single center observational cross-sectional repeatability study of patients with retinitis pigmentosa The goal of Aim 1 is to identify measures that could be used in future studies to track the extent of functional retina over time The goal of Aim 2 is to evaluate structural measures for RP The goal of Aim 3 is to assess the participant s performance on questionnaires assessing visual function The goal of Aim 4 is to survey the cytokinelymphokine profile
Outcome Measures The primary outcome measure will be the limits of agreement in repeatability calculations of the tests performed Secondary outcome measures will include analysis of parameter testing based on severity groups Macular thickness as measured by optical coherence tomography OCT as well as ellipsoid zone band length will be quantified Functional testing with photopic perimetry and scoptopic perimetry and kinetics will be quantified Multifocal electroretinography mfERG will be analyzed by subfield and possible ring analyses Correlations of questionnaire scores with objective measures will be analyzed
Objective The objective of this study is to investigate the nature of photoreceptor dysfunction in retinitis pigmentosa RP patients using focal static and kinetic psychophysical tests to develop functional outcome measures for the clinical trial study in RP Correlation of novel spatial functional maps with other functional measures such as visual acuity and multifocal electroretinography will help provide a full description of functional change Employing new imaging methods to visualize and analyze structural changes in the retina will allow for the evaluation of structural changes that underlie disease progression Developing these measures has the potential to advance the field by elucidating the process of photoreceptor degeneration as well as being a scaffold for which candidate therapies could be trialed
Study Population Up to 120 participants with a diagnosis of RP will be enrolled Up to
30 healthy volunteers will also be enrolled for a total accrual ceiling of 150
Design This is a single center observational cross-sectional repeatability study of patients with retinitis pigmentosa The goal of Aim 1 is to identify measures that could be used in future studies to track the extent of functional retina over time The goal of Aim 2 is to evaluate structural measures for RP The goal of Aim 3 is to assess the participant s performance on questionnaires assessing visual function The goal of Aim 4 is to survey the cytokinelymphokine profile
Outcome Measures The primary outcome measure will be the limits of agreement in repeatability calculations of the tests performed Secondary outcome measures will include analysis of parameter testing based on severity groups Macular thickness as measured by optical coherence tomography OCT as well as ellipsoid zone band length will be quantified Functional testing with photopic perimetry and scoptopic perimetry and kinetics will be quantified Multifocal electroretinography mfERG will be analyzed by subfield and possible ring analyses Correlations of questionnaire scores with objective measures will be analyzed
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 19-EI-0056 | None | None | None |