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NOT YET RECRUITING

NCT07796646

PHASE2

Study to Evaluate the Long-Term Safety of Ultevursen in Participants With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

Sponsor: Laboratoires Thea

View on ClinicalTrials.gov

Summary

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.

Official title: An Open-Label Extension Study to Evaluate the Long-Term Safety and Tolerability of Ultevursen in Participants With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

Key Details

Gender

All

Age Range

8 Years - Any

Study Type

INTERVENTIONAL

Enrollment

Start Date

2026-10-05

Completion Date

2030-03

Last Updated

2026-09-01

Healthy Volunteers

Conditions

Retinitis Pigmentosa (RP)Usher Syndrome Type 2 Deaf Blind Retinal Disease Eye Diseases, Hereditary Eye Disorders Congenital Vision Disorders

Interventions

OTHER

No intervention, will not receive any active study intervention

Observation only (no experimental drug administered)

DRUG

Intravitreal Injection of Ultevursen

Participants will receive up to 4 doses over a 24-month period

Inclusion Criteria: 1. 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 2. Be adults (≥18 years old) who are able to provide informed consent OR minors (8 to \<18 years old) providing assent with parental permission 3. Participant (and/or parent/caregiver) willing and able to comply with the 24-month protocol, attend all study visits, and complete all assessments 4. Treatment Cohort only: Non-pregnant and non-breastfeeding. Women of childbearing potential (WOCBP) and fertile males must use highly effective contraception Exclusion Criteria: 1. 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. 2. Treatment Cohort only: Known hypersensitivity to antisense oligonucleotides or any constituents of the injection 3. 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). 4. Both cohorts: Presence of any significant ocular or systemic disease, or laboratory abnormality, that would increase participant risk irrespective of treatment or interfere with study assessments 5. Both cohorts: Participation in any other interventional clinical trial involving an investigational drug or device during the course of the CAPELLA study