The purpose of this Phase 2b study is to evaluate the 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. This is a multicenter Double-masked, Randomized, Sham-controlled study which will enroll 81 subjects.
A total of eighty-one (81) RP subjects will be enrolled in this study, randomized in a 2:1 ratio to either ultevursen or sham procedure, respectively. Subjects randomized to the active treatment group will receive therapy with ultevursen administered via intravitreal (IVT) injection to the treatment eye (TE) on Day 1 and at Months 6, 12, and 18. Subjects randomized to sham will undergo a sham procedure in the TE at the corresponding timepoints.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
81
Up to 4 doses over a 24-month period
Sham-procedure (no experimental drug administered)
The University of California, San Francisco
San Francisco, California, United States
RECRUITINGBascom Palmer Eye Institute/University of Miami
Miami, Florida, United States
RECRUITINGEmory University
Atlanta, Georgia, United States
RECRUITINGMassachusetts Eye and Ear
Boston, Massachusetts, United States
To evaluate efficacy after 24 months of treatment
Annualized percent change from baseline in ellipsoid zone (EZ) width as measured by spectral-domain optical coherence tomography (SD-OCT) up to Month 24.
Time frame: 24 Months
Annualized change from baseline in static perimetry (SP) mean sensitivity
Time frame: Month 24
Annualized change from baseline in microperimetry (MP) mean sensitivity
Time frame: Month 24
Change from baseline in low luminance visual acuity (LLVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) chart
Time frame: Month 24
Change from baseline in best-corrected visual acuity (BCVA) using the ETDRS chart
Time frame: Month 24
Percent change from baseline in EZ area by SD-OCT
Time frame: Month 24
Percent change from baseline in EZ width by SD-OCT
Time frame: Month 24
Change from baseline in the Michigan Retinal Degeneration Questionnaire (MRDQ) for subjects ≥13 years of age
Michigan Retinal Degeneration Questionnaire (MRDQ) scores in central vision, color vision, contrast sensitivity, scotopic function, photopic peripheral vision, mesopic peripheral vision, and photosensitivity Unabbreviated scale title: Michigan Retinal Degeneration Questionnaire Minimum and Maximum values: -3 and +3 Higher scores indicate a worse outcome.
Time frame: Month 24
Change from baseline in the Michigan Vision-Related Anxiety Questionnaire (MVAQ) for subjects ≥13 years of age
Michigan Vision-Related Anxiety Questionnaire (MVAQ) scores in rod-function anxiety and cone-function anxiety. Unabbreviated scale title: Michigan Vision-Related Anxiety Questionnaire Minimum and Maximum values: -3 and +3 Higher scores indicate a worse outcome.
Time frame: Month 24
Frequency and severity of ocular and non-ocular adverse events (AEs)
Time frame: Up to month 24
Systemic Exposure
Systemic serum concentration of ultevursen
Time frame: Up to month 24
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University of Michigan- Kellogg Eye Center
Ann Arbor, Michigan, United States
RECRUITINGCasey Eye Institute, Oregon Health & Science University
Portland, Oregon, United States
RECRUITINGUniversity of Pennsylvania, Scheie Eye Institute
Philadelphia, Pennsylvania, United States
RECRUITINGRetina Foundation of the Southwest
Dallas, Texas, United States
RECRUITINGBaylor College of Medicine
Houston, Texas, United States
RECRUITINGUniversity of Wisconsin- Madison
Madison, Wisconsin, United States
RECRUITING...and 16 more locations