[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100654190":3},{"organization":4,"outcomesModule":7,"designInfo":36,"detailedDescription":43,"studyPopulation":11,"armGroups":44,"interventions":57,"overallOfficials":11,"centralContacts":67,"locations":11,"responsibleParty":73,"collaborators":75,"id":78,"slug":79,"hasResults":80,"nctId":81,"briefTitle":82,"officialTitle":83,"acronym":84,"eligibilityCriteria":85,"healthyVolunteers":80,"sex":86,"minAge":87,"maxAge":11,"enrollmentInfo":88,"targetDuration":11,"studyType":91,"phases":92,"briefSummary":94,"conditions":95,"keywords":103,"overallStatus":115,"whyStopped":11,"lastUpdateSubmitDate":116,"lastUpdatePostDateStruct":117,"startDateStruct":120,"completionDateStruct":122,"leadSponsor":124,"locationsCount":11},{"fullName":5,"class":6},"Laboratoires Thea","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":11,"otherOutcomes":15},[9,13],{"measure":10,"description":11,"timeFrame":12},"Incidence and severity of ocular adverse events (AEs), including serious AEs (SAEs) and adverse events of special interest (AESI)",null,"24 Months",{"measure":14,"description":11,"timeFrame":12},"Incidence and severity of non-ocular AEs, including SAEs.",[16,19,22,24,26,28,30,32],{"measure":17,"description":11,"timeFrame":18},"Percent change in EZ area (SD-OCT)","Months 12 and 24",{"measure":20,"description":21,"timeFrame":18},"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.",{"measure":23,"description":11,"timeFrame":18},"Change in Best Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study chart (ETDRS)",{"measure":25,"description":11,"timeFrame":18},"Change in low luminance visual acuity (LLVA) using the Early Treatment Diabetic Retinopathy Study chart (ETDRS)",{"measure":27,"description":11,"timeFrame":18},"Change in mean sensitivity at ≥5 pre specified MP loci",{"measure":29,"description":11,"timeFrame":18},"Change in MP mean sensitivity",{"measure":31,"description":11,"timeFrame":18},"Percent change in EZ width (SD-OCT)",{"measure":33,"description":34,"timeFrame":35},"Change in PRO Domains (MVAQ)","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",{"allocation":37,"interventionModel":38,"interventionModelDescription":39,"primaryPurpose":40,"observationalModel":11,"timePerspective":11,"maskingInfo":41},"NON_RANDOMIZED","PARALLEL","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\u002For a participant's willingness to continue into CAPELLA after completing the Phase 2 LUNA study.","TREATMENT",{"masking":42,"maskingDescription":11,"whoMasked":11},"NONE","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\u002FBaseline 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\u002FBaseline, 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\u002FBaseline, Month 12 and Month 24 for safety follow-up and other study related tests.",[45,51],{"label":46,"type":47,"description":48,"interventionNames":49},"Ultevursen 180\u002F60 μg","EXPERIMENTAL","Participants will receive an intravitreal injection (IVT) of Ultevursen with concentrations of 3.6 mg\u002FmL for initial dose and 1.2 mg\u002FmL 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.",[50],"Drug: Intravitreal Injection of Ultevursen",{"label":52,"type":53,"description":54,"interventionNames":55},"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\u002FBaseline, Months 12 and 24 for safety follow up and other study-related tests.",[56],"Other: No intervention, will not receive any active study intervention",[58,62],{"type":53,"name":59,"description":60,"armGroupLabels":61,"otherNames":11},"No intervention, will not receive any active study intervention","Observation only (no experimental drug administered)",[52],{"type":63,"name":64,"description":65,"armGroupLabels":66,"otherNames":11},"DRUG","Intravitreal Injection of Ultevursen","Participants will receive up to 4 doses over a 24-month period",[46],[68],{"name":69,"role":70,"phone":71,"phoneExt":11,"email":72},"Sepul Bio Advocacy Director","CONTACT","+31 617060791","contact@sepulbio.com",{"type":74,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR",[76],{"name":77,"class":6},"Sepul Bio","100654190","phase-2-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-100654190",false,"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","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","CAPELLA","Inclusion Criteria:\n\n1. 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\n2. Be adults (≥18 years old) who are able to provide informed consent OR minors (8 to \\\u003C18 years old) providing assent with parental permission\n3. Participant (and\u002For parent\u002Fcaregiver) willing and able to comply with the 24-month protocol, attend all study visits, and complete all assessments\n4. Treatment Cohort only: Non-pregnant and non-breastfeeding. Women of childbearing potential (WOCBP) and fertile males must use highly effective contraception\n\nExclusion Criteria:\n\n1. 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\n\n   * 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.\n2. Treatment Cohort only: Known hypersensitivity to antisense oligonucleotides or any constituents of the injection\n3. 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).\n4. 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\n5. Both cohorts: Participation in any other interventional clinical trial involving an investigational drug or device during the course of the CAPELLA study","ALL","8 Years",{"count":89,"type":90},81,"ESTIMATED","INTERVENTIONAL",[93],"PHASE2","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.",[96,97,98,99,100,101,102],"Retinitis Pigmentosa (RP)","Usher Syndrome Type 2","Deaf Blind","Retinal Disease","Eye Diseases, Hereditary","Eye Disorders Congenital","Vision Disorders",[104,97,98,105,106,107,108,109,110,111,112,113,114],"Retinitis Pigmentosa","USH2A","RP","Exon 13","RNA Therapies","antisense oligonucleotide","exon skipping","LUNA","Capella","IVT","NSRP","NOT_YET_RECRUITING","2026-08-31",{"date":118,"type":119},"2026-09-01","ACTUAL",{"date":121,"type":90},"2026-10-05",{"date":123,"type":90},"2030-03",{"name":5,"class":6}]