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)
2026年8月31日 更新者: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.
研究概览
地位
尚未招聘
详细说明
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.
研究类型
介入性
注册 (估计的)
81
阶段
- 阶段2
联系人和位置
本节提供了进行研究的人员的详细联系信息,以及有关进行该研究的地点的信息。
学习联系方式
- 姓名:Sepul Bio Advocacy Director
- 电话号码:+31 617060791
- 邮箱:contact@sepulbio.com
参与标准
研究人员寻找符合特定描述的人,称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
- 孩子
- 成人
- 年长者
接受健康志愿者
不
描述
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
学习计划
本节提供研究计划的详细信息,包括研究的设计方式和研究的衡量标准。
研究是如何设计的?
设计细节
- 主要用途:治疗
- 分配:非随机化
- 介入模型:并行分配
- 屏蔽:无(打开标签)
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
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实验性的: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.
|
Participants will receive up to 4 doses over a 24-month period
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|
其他: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.
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Observation only (no experimental drug administered)
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研究衡量的是什么?
主要结果指标
结果测量 |
大体时间 |
|---|---|
|
Incidence and severity of ocular adverse events (AEs), including serious AEs (SAEs) and adverse events of special interest (AESI)
大体时间:24 Months
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24 Months
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Incidence and severity of non-ocular AEs, including SAEs.
大体时间:24 Months
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24 Months
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其他结果措施
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Percent change in EZ area (SD-OCT)
大体时间:Months 12 and 24
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Months 12 and 24
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Change in PRO domains (MRDQ)
大体时间: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.
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Months 12 and 24
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Change in Best Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study chart (ETDRS)
大体时间:Months 12 and 24
|
Months 12 and 24
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|
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Change in low luminance visual acuity (LLVA) using the Early Treatment Diabetic Retinopathy Study chart (ETDRS)
大体时间:Months 12 and 24
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Months 12 and 24
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|
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Change in mean sensitivity at ≥5 pre specified MP loci
大体时间:Months 12 and 24
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Months 12 and 24
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Change in MP mean sensitivity
大体时间:Months 12 and 24
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Months 12 and 24
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Percent change in EZ width (SD-OCT)
大体时间:Months 12 and 24
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Months 12 and 24
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Change in PRO Domains (MVAQ)
大体时间: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
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合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
合作者
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查,以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (估计的)
2026年10月5日
初级完成 (估计的)
2030年2月28日
研究完成 (估计的)
2030年3月1日
研究注册日期
首次提交
2026年8月20日
首先提交符合 QC 标准的
2026年8月31日
首次发布 (实际的)
2026年9月1日
研究记录更新
最后更新发布 (实际的)
2026年9月1日
上次提交的符合 QC 标准的更新
2026年8月31日
最后验证
2026年6月1日
更多信息
与本研究相关的术语
其他相关的 MeSH 术语
其他研究编号
- SB-421a-005
- 2026-527242-45-00 (克蒂斯)
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)?
不
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研究美国 FDA 监管的药品
是的
研究美国 FDA 监管的设备产品
不
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