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

NCT07796646 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: SB-421a-0052026-527242-45-00
• Study Type: interventional
• Target: 81
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

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

Keywords

Retinitis Pigmentosa; Usher Syndrome Type 2; Deaf Blind; USH2A; RP; Exon 13; RNA Therapies; antisense oligonucleotide; exon skipping; LUNA; Capella; IVT; NSRP

Outcome Measures

Primary Outcomes (2)

• Incidence and severity of ocular adverse events (AEs), including serious AEs (SAEs) and adverse events of special interest (AESI)

  24 Months

• Incidence and severity of non-ocular AEs, including SAEs.

  24 Months

Other Outcomes (8)

• Percent change in EZ area (SD-OCT)

  Months 12 and 24

• Change in PRO domains (MRDQ)

  Months 12 and 24

• Change in Best Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study chart (ETDRS)

  Months 12 and 24

Study Arms (2)

Ultevursen 180/60 μg

EXPERIMENTAL

Participants will receive an intravitreal injection (IVT) of Ultevursen with concentrations of 3.6 mg/mL for initial dose and 1.2 mg/mL for maintenance doses every 6 months thereafter through Month 18 (up to 4 doses). The dose concentrations will be determined by the participant's randomized cohort assignment during the Phase 2 LUNA study.

Drug: Intravitreal Injection of Ultevursen

Observation Only

OTHER

Participants who are assigned to the Observation Only Cohort will not receive active study treatment via intravitreal injection (IVT), but will be asked to visit the trial location at Screening/Baseline, Months 12 and 24 for safety follow up and other study-related tests.

Other: No intervention, will not receive any active study intervention

Interventions

No intervention, will not receive any active study intervention OTHER

Observation only (no experimental drug administered)

Observation Only

Intravitreal Injection of Ultevursen DRUG

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

Ultevursen 180/60 μg

Eligibility Criteria

• Age: 8 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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).
• 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
• Both cohorts: Participation in any other interventional clinical trial involving an investigational drug or device during the course of the CAPELLA study

Sponsors & Collaborators

• Laboratoires Thea: lead
• Sepul Bio: collaborator

MeSH Terms

Conditions

Retinitis Pigmentosa; Usher Syndromes; Retinal Diseases; Eye Diseases, Hereditary; Eye Abnormalities; Vision Disorders

Condition Hierarchy (Ancestors)

Eye Diseases; Retinal Dystrophies; Retinal Degeneration; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Deaf-Blind Disorders; Deafness; Hearing Loss; Hearing Disorders; Ear Diseases; Otorhinolaryngologic Diseases; Hearing Loss, Sensorineural; Sensation Disorders; Neurologic Manifestations; Nervous System Diseases; Blindness; Abnormalities, Multiple; Congenital Abnormalities; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Sepul Bio Advocacy Director

CONTACT

+31 617060791; contact@sepulbio.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Participants will be grouped into either Treatment Cohort (IVT injection) or Observation Only Cohort (no IVT injection), depending on a participant's eligibility for CAPELLA and/or a participant's willingness to continue into CAPELLA after completing the Phase 2 LUNA study.

Study Record Dates

• First Submitted: August 20, 2026
• First Posted: September 1, 2026
• Study Start (Estimated): October 5, 2026
• Primary Completion (Estimated): February 28, 2030
• Study Completion (Estimated): March 1, 2030
• Last Updated: September 1, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share