A Double-masked, Randomized, Sham-Controlled Study to Evaluate the Efficacy, Safety and Tolerability of Ultevursen in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene

Retinitis Pigmentosa

Annualized percent change from baseline in EZ width as measured by spectral-domain optical coherence tomography (SD-OCT) up to Month 24.

Efficacy: The following endpoints will be evaluated at Months 12 and 24, and as annualized change with data up to 24 months, unless otherwise specified: (Annualized) change from baseline in static perimetry (SP) mean sensitivity, Efficacy: The following endpoints will be evaluated at Months 12 and 24, and as annualized change with data up to 24 months, unless otherwise specified: (Annualized) change from baseline in microperimetry (MP) mean sensitivity, Efficacy: The following endpoints will be evaluated at Months 12 and 24, and as annualized change with data up to 24 months, unless otherwise specified: (Annualized) change from baseline in retinal sensitivity in at least 5 pre-specified loci in SP, Efficacy: The following endpoints will be evaluated at Months 12 and 24, and as annualized change with data up to 24 months, unless otherwise specified: (Annualized) change from baseline in retinal sensitivity in at least 5 pre-specified loci in MP, Efficacy: The following endpoints will be evaluated at Months 12 and 24, and as annualized change with data up to 24 months, unless otherwise specified: (Annualized) change from baseline in low luminance visual acuity (LLVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) chart, Efficacy: The following endpoints will be evaluated at Months 12 and 24, and as annualized change with data up to 24 months, unless otherwise specified: (Annualized) change from baseline in best-corrected visual acuity (BCVA) using the ETDRS chart, Efficacy: The following endpoints will be evaluated at Months 12 and 24, and as annualized change with data up to 24 months, unless otherwise specified: (Annualized) percent change from baseline in EZ area by SDOCT, Efficacy: The following endpoints will be evaluated at Months 12 and 24, and as annualized change with data up to 24 months, unless otherwise specified: Percent change from baseline in EZ width by SD-OCT, Efficacy: Evaluated at Months 12 and 24, and as annualized change with data up to 24 months: (Annualized) change from baseline in each domain of the following PROs (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, Efficacy: Evaluated at Months 12 and 24, and as annualized change with data up to 24 months: (Annualized) change from baseline in each domain of the following PROs (for subjects ≥13 years of age): Michigan Vision-Related Anxiety Questionnaire (MVAQ) scores in rod-function anxiety and cone-function anxiety, Safety and Tolerability: Frequency and severity of ocular and non-ocular adverse events (AEs), Systemic Exposure: Systemic serum concentration of ultevursen.

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