Retinitis Pigmentosa Clinical Measures and Repeatability Testing of Potential Outcome Measures
| Status: | Recruiting |
|---|---|
| Conditions: | Ocular |
| Therapuetic Areas: | Ophthalmology |
| Healthy: | No |
| Age Range: | 12 - 100 |
| Updated: | 4/6/2019 |
| Start Date: | April 10, 2019 |
| End Date: | July 30, 2021 |
| Contact: | Angel H Garced, R.N. |
| Email: | garceda@nei.nih.gov |
| Phone: | (301) 594-3141 |
A Survey Study of Retinitis Pigmentosa (RP) Clinical Measures and Repeatability Testing of Potential Outcome Measures
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, butut 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
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.
Sponsoring Institute: National Eye Institute
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, butut 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
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.
Sponsoring Institute: National Eye Institute
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.
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.
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.
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.
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.
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.
- INCLUSION CRITERIA:
To be eligible, the following inclusion criteria must be met, where applicable.
- Participant must be 12 years of age or older.
- Participant (or legal guardian) must understand and sign the protocol s informed
consent document.
- Participant must have evidence of RP as defined by characteristic ERG responses,
visual fields, clinical exam and /or genetic testing.
EXCLUSION CRITERIA:
A participant is not eligible if any of the following exclusion criteria are present.
- Participant is actively receiving study therapy in another investigational study.
- Participant is started on (or changed dosage of) topical or systemic carbonic
anhydrase inhibitor (CAI) treatment in the 3 months prior to enrollment.
- Participant is expected to be unable to comply with study procedures or follow-up
visits.
We found this trial at
1
site
9000 Rockville Pike
Bethesda, Maryland 20892
Bethesda, Maryland 20892
301-496-2563
Phone: 800-411-1222
National Institutes of Health Clinical Center The National Institutes of Health (NIH) Clinical Center in...
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