A Two-Year 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, unless otherwise specified: Change in retinal sensitivity measured by static perimetry (SP) with topographic analysis (Hill of Vision [HoV]), Efficacy: The following endpoints will be evaluated at Months 12 and 24, unless otherwise specified: Change in retinal sensitivity measured by microperimetry (MP) with topographic analysis (HoV), Efficacy: The following endpoints will be evaluated at Months 12 and 24, unless otherwise specified: Change from baseline in retinal sensitivity in a pre-specified area with at least 5 loci, Efficacy: The following endpoints will be evaluated at Months 12 and 24, unless otherwise specified: 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, unless otherwise specified: 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, unless otherwise specified: Percent change from baseline in EZ area by SD-OCT, Efficacy: The following endpoints will be evaluated at Months 12 and 24, unless otherwise specified: Percent change from baseline in EZ width by SD-OCT, Efficacy: The following endpoints will be evaluated at Months 12 and 24, unless otherwise specified: Annualized percent change from baseline in EZ area by SD-OCT, Efficacy: The following endpoints will be evaluated at Months 12 and 24, unless otherwise specified: Change from baseline in virtual visual maze performance score using Mobility Standardized Test in Virtual Reality (MOSTVR) (applicable for sites with ability to conduct the assessment), Efficacy: The following endpoints will be evaluated at Months 12 and 24, unless otherwise specified: Change from baseline in dark-adapted full-field stimulus threshold (FST) testing, Efficacy: The following endpoints will be evaluated at Months 12 and 24, unless otherwise specified: 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: The following endpoints will be evaluated at Months 12 and 24, unless otherwise specified: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), Safety and Tolerability: Incidence of anti-drug antibodies (ADAs) against ultevursen, Systemic Exposure: Systemic serum concentration of ultevursen.

No related papers found yet.

France, Germany, Italy, Netherlands