Our study objective is to investigate the reliability of commonly used clinical examinations and endpoints performed in patients with inherited retinal dystrophies in anticipation of future clinical trials.
ID
Source
Brief title
Condition
- Retina, choroid and vitreous haemorrhages and vascular disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The main parameter is the test-retest variability in microperimetry,
demonstrated as the change average threshold (dB) between two repeated
measurements of macular sensitivity.
Secondary outcome
Secondary endpoints include the changes in other functional and structural
outcome measures between repeated measurements. The functional measures include
the test-retest variability of best-corrected visual acuity under different
light intensities, visual field, fixation stability, and reported quality of
life. The structural parameters include test-retest variabilities of parameters
measured on SD-OCT and fundus autofluorescence.
Background summary
Inherited retinal dystrophies (IRDs) encompass a spectrum of severe eye
diseases, characterized by progressive loss of retinal structure and visual
function. No treatment is currently available for the majority of IRDs,
although promising results have been achieved using gene therapy in one
specific type of IRD, paving the way for future treatment. However, there is a
knowledge gap on which clinical investigations and endpoints are most suitable
to test treatment efficacy. Particularly, little is known on test-retest
variability in this patient group for a range of specialized clinical
examinations that appear important for the evaluation of these diseases. This
information appears pivotal for optimal patient selection, and to determine
reliable clinical parameters for upcoming clinical trials focused on
treatments. Therefore, further insight is required into the reliability of
commonly used clinical outcome measures such as microperimetry, full-field
stimulus threshold testing, perimetry, best-corrected visual acuity, and
retinal structures on multimodal imaging. Determining the test-retest
variability of these outcome measures will aid future trials in distinguishing
treatment efficacy from measurement errors.
Study objective
Our study objective is to investigate the reliability of commonly used clinical
examinations and endpoints performed in patients with inherited retinal
dystrophies in anticipation of future clinical trials.
Study design
Investigator-initiated, single-center, prospective, observational study
consisting of two visits over a two-week (± 2 week) interval. During each
visit, the participants will perform several ophthalmological measurements.
Study burden and risks
No interventional therapy or study drug will be used and thus there is no risk
of adverse events. However, patients (especially young children and elderly)
may experience a minimal physical burden of visiting the hospital twice in two
weeks. Additionally, patients may feel confronted with their visual
impairments. Lastly, the use of mydriatics pose a minimal risk of acute angle
closure glaucoma. In order to prevent this, the ocular pressure will be
strictly monitored during the study and potential occurrence of an increase in
pressure will be treated according to standard clinical protocol. These burdens
are expected to be minimal.
Albinusdreef 2
Leiden 2333ZA
NL
Albinusdreef 2
Leiden 2333ZA
NL
Listed location countries
Age
Inclusion criteria
In order to be eligible to participate in this study as a visually-impaired
patient, a subject must meet all of the following criteria:
• Willing and able to provide informed consent (IC) for the participation in
the study;
• Minimum age of 16 years;
• Clinical diagnosis of IRD with BCVA using ETDRS of >=20/50 Snellen equivalent;
• Willing and able to undergo ophthalmic examinations at two separate occasions;
• No ocular or non-ocular disease/disorder that may influence the results of
the measurements.
In order to be eligible to participate in this study as a low-vision patient, a
subject must meet all of the following criteria:
• Willing and able to provide informed consent (IC) for the participation in
the study;
• Minimum age of 16 years;
• Clinical diagnosis of IRD with BCVA using ETDRS of <20/50 Snellen equivalent;
• Willing and able to undergo ophthalmic examinations at two separate occasions;
• No ocular or non-ocular disease/disorder that may influence the results of
the measurements
Exclusion criteria
A potential subject who meets any of the following criteria will be excluded
from participation in this study:
• The last measured BCVA in the better-seeing eye is light-perception;
• (History of) ocular of non-ocular disease/disorder that may influence the
results of the measurements (e.g. amblyopia);
• Known allergy or intolerance for ocular anesthetic eye drops oxybuprocaine
0.4% or mydriatics tropicamide 0.5% and/or phenylephrine 5%;
• Participation in another research study involving an investigational
medicinal product related to their ocular health.
Design
Recruitment
metc-ldd@lumc.nl
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL79646.058.21 |