The purpose of this Phase 2 Open-label study is to evaluate the long-term safety and tolerability of Ultevursen administered via intravitreal injection (IVT) in participants with Retinitis Pigmentosa due to mutations in exon 13 of the USH2A gene. This is a multicenter study which will enroll approximately 81 participants, however, the exact number enrolled will depend on the final number completing the 24-month LUNA trial and are eligible and willing to enroll in CAPELLA.
Participants who have completed the LUNA study through Month 24 and, in the Investigator's judgment, may benefit from continued treatment or observational follow-up
Be adults (≥18 years old) who are able to provide informed consent OR minors (8 to <18 years old) providing assent with parental permission
Participant (and/or parent/caregiver) willing and able to comply with the 24-month protocol, attend all study visits, and complete all assessments
Treatment Cohort only: Non-pregnant and non-breastfeeding. Women of childbearing potential (WOCBP) and fertile males must use highly effective contraception
Treatment Cohort only: New ocular condition in the SE (e.g., severe infection, uncontrolled glaucoma, or retinal detachment) that, in the Investigator's opinion, makes the SE unsafe for IVT injection
Additionally, any ocular condition or AE in the fellow eye that, in the opinion of the Investigator, may impact participant safety or confound the assessment of the SE. Eligibility in such cases will be evaluated on a case-by-case basis.
Treatment Cohort only: Known hypersensitivity to antisense oligonucleotides or any constituents of the injection
Treatment Cohort only: Any clinically significant safety finding observed during the LUNA trial that, in the Investigator's opinion, compromises the participant's safety with continued dosing. Participants that have had study drug temporarily withdrawn or elective ocular surgery or AESI's during the LUNA trial may be reviewed on a case by case basis for eligibility into the Treatment Cohort (Note: These participants may still be eligible for the Observation Only Cohort at the Investigator's discretion and following discussion with medical monitor).
Parallel
No intervention, will not receive any active study intervention
Other interventionObservation only (no experimental drug administered)
Intravitreal Injection of Ultevursen
DrugParticipants will receive up to 4 doses over a 24-month period
Incidence and severity of ocular adverse events (AEs), including serious AEs (SAEs) and adverse events of special interest (AESI)
Incidence and severity of non-ocular AEs, including SAEs.
Percent change in EZ area (SD-OCT)
Change in PRO domains (MRDQ)
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.
Change in Best Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study chart (ETDRS)
Change in low luminance visual acuity (LLVA) using the Early Treatment Diabetic Retinopathy Study chart (ETDRS)
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Laboratoires Thea
Lead sponsor
Sepul Bio
Collaborator
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