The purpose of this study is to evaluate the efficacy safety and tolerability of QR-421a administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene with early to moderate vision loss.
The purpose of this study is to evaluate the efficacy safety and tolerability of QR-421a administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene with early to moderate vision loss. The below dose levels of QR-421a will be evaluated with the loading dose administered at Day 1 and maintenance dose administered at Month 3 and every 6 months thereafter: 1. Loading dose of 180 μg, maintenance dose of 60 μg 2. Loading dose of 60 μg, maintenance dose of 60 μg Dose levels will include subjects randomized to sham-procedure or treatment with QR-421a. After the study eye has been treated for at least 12 months, treatment of the fellow eye and cross-over of subjects assigned to sham-procedure may be initiated in eligible eyes based on assessment of benefit-risk.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
5
RNA antisense oligonucleotide for intravitreal injection
Sham-procedure (no experimental drug administered)
Retina Foundation of the Southwest
Dallas, Texas, United States
University of Wisconsin - Madison
Madison, Wisconsin, United States
Moorfields Eye Hospital
London, United Kingdom
Change From Baseline in Mean Sensitivity
Change from baseline in mean sensitivity (based on static perimetry) at 12 months of treatment versus sham-procedure
Time frame: 12 months
Ellipzoid Zone Area (EZ) as Measured by Spectral Domain Optical Coherence Tomography SD-OCT
Ellipzoid Zone area (EZ) as measured by Spectral Domain optical coherence tomography SD-OCT
Time frame: 27 months
Change From Baseline in Best Corrected Visual Acuity (BCVA)
Change from baseline in Best Corrected Visual Acuity (BCVA)
Time frame: 27 months
Change From Baseline in Spectral Domain Optical Coherence Tomography (SD-OCT) (Other Measures)
Change from baseline in Spectral domain optical coherence tomography (SD-OCT) (other measures)
Time frame: 27 months
Change From Baseline in Low Luminance Visual Acuity (LLVA)
Change from baseline in Low Luminance Visual Acuity (LLVA) using the ETDRS vision chart
Time frame: 27 months
Change From Baseline in Other Measures of Static Perimetry
Change from baseline in other measures of static perimetry on the Octopus 900 as assessed by a central reading center
Time frame: 27 months
Change From Baseline in Full-field Stimulus Threshold (FST)
Change from baseline in Full-field Stimulus Threshold (FST) on the Diagnosys FST as assessed by a central reading center
Time frame: 27 months
Change From Baseline in PRO Measure as Assessed by Michigan Retinal Degeneration Questionnaire (MRDQ)
Change from baseline in PRO measure as assessed by Michigan Retinal Degeneration Questionnaire (MRDQ)
Time frame: 27 months
Change From Baseline in PRO Measures as Assessed by Patient Global Impressions of Severity (PGI-S)
Change from baseline in PRO measures as assessed by Patient Global Impressions of Severity (PGI-S)
Time frame: 27 months
Change From Baseline in PRO Measures as Assessed by Patient Global Impressions of Change (PGI-C)
Change from baseline in PRO measures as assessed by Patient Global Impressions of Change (PGI-C)
Time frame: 27 months
Ocular and Non-ocular Adverse Events (AEs)
Ocular and non-ocular adverse events (AEs)
Time frame: 27 months
Cmax of QR-421a in Serum
Maximum concentration (Cmax) of QR-421a in serum
Time frame: 27 months
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