The purpose of this study is to evaluate the efficacy safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.
The purpose of this study is to evaluate the efficacy safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. The below dose levels of ultevursen 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 60 µg, maintenance dose of 60 µg 2. Loading dose of 180 µg, maintenance dose of 60 µg Dose levels will include subjects randomized to sham-procedure or treatment with ultevursen.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
7
RNA antisense oligonucleotide for intravitreal injection
Sham-procedure (no experimental drug administered)
Shiley Eye Institute - UC San Diego
San Diego, California, United States
University of California, San Francisco
San Francisco, California, United States
University of Miami, Bascom Palmer Eye Institute
Miami, Florida, United States
Emory Eye Center
Atlanta, Georgia, United States
Wilmer Eye Institute, Johns Hopkins Hospital
Baltimore, Maryland, United States
Center for Clinical Research Operations, Massachusetts Eye and Ear
Boston, Massachusetts, United States
University of Michigan, Kellogg Eye Center
Ann Arbor, Michigan, United States
Columbia University
New York, New York, United States
Retina Foundation of the Southwest
Dallas, Texas, United States
University of Wisconsin - Madison
Madison, Wisconsin, United States
...and 5 more locations
Mean Change From Baseline in Best Corrected Visual Acuity (BCVA)
Mean change from baseline in best corrected visual acuity(BCVA) based on the Early Treatment Diabetic Retinopathy Study (ETDRS) chart
Time frame: 18 months of treatment versus sham-procedure
Proportion of Patients Who Maintain Vision Defined by BCVA Loss Less Than 15 Letters (ETDRS)
Proportion of patients who maintain vision defined by BCVA loss less than 15 Letters based on ETDRS
Time frame: 27 months
Change From Baseline in Other Analyses of Best Corrected Visual Acuity (BCVA)
Change from baseline in other analyses of Best Corrected Visual Acuity (BCVA)
Time frame: 27 months
Change From Baseline in Ellipsoid Zone (EZ) Area and Width as Imaged by Spectral Domain Optical Coherence Tomography (SD-OCT)
Change from baseline in ellipsoid zone (EZ) area and width as imaged by spectral domain optical coherence tomography (SD-OCT)
Time frame: 27 months
Change From Baseline in Low Luminance Visual Acuity (LLVA)
Change from baseline in Low Luminance Visual Acuity (LLVA)
Time frame: 27 months
Change From Baseline in Microperimetry
Change from baseline in Microperimetry
Time frame: 27 months
Change From Baseline in Full-field Stimulus Threshold (FST)
Change from baseline in Full-field Stimulus Threshold (FST)
Time frame: 27 months
Change From Baseline in PRO Measures
As assessed by the Veteran Affairs Low Vision Visual Functioning Questionnaire (VA LV VFQ-20), Patient Global Impressions of Severity (PGI-S) and 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 Ultevursen in Serum
Maximum concentration (Cmax) of ultevursen in serum
Time frame: 27 months
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