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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 (CAPELLA)

31. august 2026 opdateret af: Laboratoires Thea

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

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.

Studieoversigt

Status

Ikke rekrutterer endnu

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

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.

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

81

Fase

  • Fase 2

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Barn
  • Voksen
  • Ældre voksen

Tager imod sunde frivillige

Ingen

Beskrivelse

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

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Ikke-randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: Ultevursen 180/60 μg
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.
Medicin: Intravitreal Injection of Ultevursen
Participants will receive up to 4 doses over a 24-month period
Andet: Observation Only
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.
Andet: No intervention, will not receive any active study intervention
Observation only (no experimental drug administered)

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Tidsramme
Incidence and severity of ocular adverse events (AEs), including serious AEs (SAEs) and adverse events of special interest (AESI)
Tidsramme: 24 Months
24 Months
Incidence and severity of non-ocular AEs, including SAEs.
Tidsramme: 24 Months
24 Months

Andre resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Percent change in EZ area (SD-OCT)
Tidsramme: Months 12 and 24
Months 12 and 24
Change in PRO domains (MRDQ)
Tidsramme: 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.
Months 12 and 24
Change in Best Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study chart (ETDRS)
Tidsramme: Months 12 and 24
Months 12 and 24
Change in low luminance visual acuity (LLVA) using the Early Treatment Diabetic Retinopathy Study chart (ETDRS)
Tidsramme: Months 12 and 24
Months 12 and 24
Change in mean sensitivity at ≥5 pre specified MP loci
Tidsramme: Months 12 and 24
Months 12 and 24
Change in MP mean sensitivity
Tidsramme: Months 12 and 24
Months 12 and 24
Percent change in EZ width (SD-OCT)
Tidsramme: Months 12 and 24
Months 12 and 24
Change in PRO Domains (MVAQ)
Tidsramme: 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.
Month 12 and Month 24

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Laboratoires Thea

Samarbejdspartnere

Sepul Bio

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Anslået)

5. oktober 2026

Primær færdiggørelse (Anslået)

28. februar 2030

Studieafslutning (Anslået)

1. marts 2030

Datoer for studieregistrering

Først indsendt

20. august 2026

Først indsendt, der opfyldte QC-kriterier

31. august 2026

Først opslået (Faktiske)

1. september 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

1. september 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

31. august 2026

Sidst verificeret

1. juni 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • SB-421a-005
  • 2026-527242-45-00 (Ctis)

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

INGEN

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

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