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NCT Number: NCT07796646

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

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.

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Key information

About this study

Approximately Eighty-One (81) RP participants will be enrolled in this study. These participants would have completed the Phase 2b study, LUNA, and are deemed eligible to participate in the open-label study, CAPELLA. The end of study (EOS) visit for LUNA will also serve as the Screening/Baseline visit for CAPELLA. All eligible participants enrolled in the Treatment Cohort will be given the study medication (Ultevursen) via intravitreal injection (IVT) every 6 months at Screening/Baseline, Month 6, Month 12 and Month 18. The starting dosage of Ultevursen will be determined by the participant's treatment cohort they were assigned in the LUNA study (active or sham cohort). Participants not participating in the CAPELLA Treatment Cohort, either due to Investigator discretion, or participant withdrawal, will be asked to enter the "Observation Only" Cohort. These participants will not receive IVT injection of Ultevursen, but will visit the trial location at Screening/Baseline, Month 12 and Month 24 for safety follow-up and other study related tests.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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

Treatment and study plan

No intervention, will not receive any active study intervention

Other

Observation only (no experimental drug administered)

Intravitreal Injection of Ultevursen

Drug

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

Primary outcomes

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

    Time frame: 24 Months

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

    Time frame: 24 Months

Other outcomes

  1. Percent change in EZ area (SD-OCT)

    Time frame: Months 12 and 24

  2. Change in PRO domains (MRDQ)

    Time frame: Months 12 and 24

    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.

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

    Time frame: Months 12 and 24

  4. Change in low luminance visual acuity (LLVA) using the Early Treatment Diabetic Retinopathy Study chart (ETDRS)

    Time frame: Months 12 and 24

  5. Change in mean sensitivity at ≥5 pre specified MP loci

    Time frame: Months 12 and 24

  6. Change in MP mean sensitivity

    Time frame: Months 12 and 24

  7. Percent change in EZ width (SD-OCT)

    Time frame: Months 12 and 24

  8. Change in PRO Domains (MVAQ)

    Time frame: Month 12 and Month 24

    Unabbreviated scale title: Michigan Vision-Related Anxiety Questionnaire Minimum and Maximum values: -3 and +3 Higher scores indicate a worse outcome.

Study contacts

Contact information is provided by the study sponsor or research team.

Sepul Bio Advocacy Director

CONTACT

[email protected]

+31 617060791

Sponsors and collaborators

Lead sponsor

Laboratoires Thea

Industry

Collaborators

  • Sepul Bio

Registry information

Official study 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

Acronym: CAPELLA

Important dates

Study start
2026
Primary completion
2030
Study completion
2030
First posted
Sep 1, 2026
Registry last updated
Sep 1, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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