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
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 81
- 主要终点
- Incidence and severity of ocular adverse events (AEs), including serious AEs (SAEs) and adverse events of special interest (AESI)
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 8 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
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.
干预措施: No intervention, will not receive any active study intervention (Other)
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.
干预措施: Intravitreal Injection of Ultevursen (Drug)
结局指标
主要结局
Incidence and severity of ocular adverse events (AEs), including serious AEs (SAEs) and adverse events of special interest (AESI)
时间窗: 24 Months
Incidence and severity of non-ocular AEs, including SAEs.
时间窗: 24 Months
次要结局
未报告次要终点