Rate of Progression of PCDH15-Related Retinal Degeneration in Usher Syndrome 1F (NCT04765345) | Clinical Trial Compass
Active ā Not RecruitingNot Applicable
Rate of Progression of PCDH15-Related Retinal Degeneration in Usher Syndrome 1F
United States, Canada, France44 participantsStarted 2021-06-08
Plain-language summary
The overall goal of this project, co-funded by the Foundation Fighting Blindness and the USHER 1F Collaborative is to characterize the natural history of disease progression in patients with PCDH15 mutations in order to accelerate the development of outcome measures for clinical trials.
Who can participate
Age range8 Years
SexALL
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Inclusion criteria
ā. Willing to participate in the study and able to communicate consent during the consent process
ā. Ability to return for all study visits over 48 months
ā. Age ā„ 8 years
ā. Not planning to enroll in an experimental clinical trial for the treatment of PCDH15 for the duration of this study
ā. Must meet one of the Genetic Screening Criteria, defined below:
ā. Clinical diagnosis of retinal dystrophy
ā. Clear ocular media and adequate pupil dilation to permit good quality photographic imaging
Exclusion criteria
ā. Mutations in genes that cause autosomal dominant retinitis pigmentosa (ADRP), X-linked retinitis pigmentosa (RP), or presence of biallelic mutations in autosomal recessive RP/retinal dystrophy genes other than PCDH15
ā. Expected to enter experimental treatment trial at any time during this study
ā. History of more than 1 year of cumulative treatment, at any time, with an agent associated with pigmentary retinopathy (including hydroxychloroquine, chloroquine, thioridazine, and deferoxamine)
ā. Current vitreous hemorrhage
ā. Current or any history of tractional or rhegmatogenous retinal detachment
What they're measuring
1
Change in Visual Field Sensitivity
Timeframe: Baseline (all Vision Cohort 1 and 2 participants will complete two tests at baseline. The results will be compared according to the visual field criteria to determine if a third test is needed), 12Month, 24Month, 36Month, and 48Month
2
Change in Best Corrected Visual Acuity
Timeframe: Screening, Baseline, 12Month, 24Month, 36Month, and 48Month
3
Change in Mean Retinal Sensitivity
Timeframe: Baseline (all Vision Cohort 1 and 2 participants will complete two tests at baseline. The results will be compared according to the visual field criteria to determine if a third test is needed), 12Month, 24Month, 36Month, and 48Month
4
Change in Full-field Retinal Sensitivity
Timeframe: Baseline, 12Month, 24Month, 36Month, and 48Month
5
Change in Best Corrected Low Luminance Visual Acuity (LLVA)
Timeframe: Screening, Baseline, 12Month, 24Month, 36Month, and 48Month
6
Change in Contrast Sensitivity Function (CSF)
Timeframe: Baseline, 12Month, 24Month, 36Month, and 48Month
7
Change in ellipsoid zone (EZ) area
Timeframe: Baseline, 12Month, 24Month, 36Month, and 48Month
. Current or any history of (e.g., prior to cataract or refractive surgery) spherical equivalent of the refractive error worse than -8 Diopters of myopia
ā. History of intraocular surgery (e.g., cataract surgery, vitrectomy, penetrating keratoplasty, or LASIK) within the last 3 months
ā. Current or any history of confirmed diagnosis of glaucoma (e.g., based on glaucomatous VF changes or nerve changes, or history of glaucoma filtering surgery)