Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

Retinitis Pigmentosa Clinical Measures and Repeatability Testing of Potential Outcome Measures

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03845218
Recruitment Status : Recruiting
First Posted : February 19, 2019
Last Update Posted : May 24, 2019
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Eye Institute (NEI) )

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, 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


Condition or disease
Retinitis Pigmentosa

Detailed Description:

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.


Layout table for study information
Study Type : Observational
Estimated Enrollment : 120 participants
Observational Model: Case-Only
Time Perspective: Cross-Sectional
Official Title: A Survey Study of Retinitis Pigmentosa (RP) Clinical Measures and Repeatability Testing of Potential Outcome Measures
Estimated Study Start Date : May 29, 2019
Estimated Primary Completion Date : March 30, 2021
Estimated Study Completion Date : July 30, 2021

Resource links provided by the National Library of Medicine


Group/Cohort
1
Patients with photoreceptor dysfunction in retinitis pigmentosa (RP)



Primary Outcome Measures :
  1. Macular thicknes as measured by OCT, EZ band length; functional testing including phototopic/scotopic perimetry and kinetics, mfERG [ Time Frame: ongoing ]
    The primary outcome measure will be the limits of agreement in repeatedly calculations of the test performed.


Secondary Outcome Measures :
  1. Secondary outcome measures will include analysis of parameter testing based on absolute measures to categorize testing results into severity groups [ Time Frame: ongoing ]
    Information about which tests did not yield quantifiable measures in which participants will also be collected.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   12 Years to 100 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Up to 120 participants aged 12 or older with a diagnosis of RP.
Criteria
  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03845218


Contacts
Layout table for location contacts
Contact: Angel H Garced, R.N. (301) 594-3141 garceda@nei.nih.gov

Locations
Layout table for location information
United States, Maryland
National Institutes of Health Clinical Center Recruiting
Bethesda, Maryland, United States, 20892
Contact: For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)    800-411-1222 ext TTY8664111010    prpl@cc.nih.gov   
Sponsors and Collaborators
National Eye Institute (NEI)
Investigators
Layout table for investigator information
Principal Investigator: Catherine A Cukras, M.D. National Eye Institute (NEI)

Additional Information:
Layout table for additonal information
Responsible Party: National Eye Institute (NEI)
ClinicalTrials.gov Identifier: NCT03845218     History of Changes
Other Study ID Numbers: 190056
19-EI-0056
First Posted: February 19, 2019    Key Record Dates
Last Update Posted: May 24, 2019
Last Verified: February 8, 2019

Keywords provided by National Institutes of Health Clinical Center (CC) ( National Eye Institute (NEI) ):
Retina
Imaging
Psychophysics
Functional Outcome Measures

Additional relevant MeSH terms:
Layout table for MeSH terms
Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn