Study to Evaluate the Safety and Preliminary Efficacy of UGX202 Injection in Patients With Advanced Retinitis Pigmentosa
概览
- 阶段
- 早期 1 期
- 状态
- 尚未招募
- 发起方
- Suzhou UgeneX Therapeutics Co., Ltd.
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Incidence of adverse events and serious adverse events
概览
简要总结
The primary objective of this clinical trial is to evaluate the safety and tolerability of a single intravitreal injection of the gene therapy drug UGX202 in patients with advanced RP. The secondary objective is, to assess the preliminary efficacy of a single intravitreal injection of the gene therapy drug UGX202 in treating patients with advanced RP.
详细描述
This study is a non-randomized, open-label investigator-initiated trial (IIT). It plans to enroll approximately 6 subjects with non-syndromic retinitis pigmentosa (RP) who have extremely low vision (the study eye is the eye with lower vision, and the best corrected visual acuity [BCVA] > logMAR 1.9).
The study drug is divided into two dose groups: low dose and high dose. A modified "3+3" dose escalation approach is adopted. The low-dose group (4.2E+10 vg/eye) is planned to include 3 subjects. First, 1 subject (sentinel) will be enrolled and observed for 28 days. If no dose-limiting toxicity (DLT) occurs, 2 more subjects (non-sentinel) will be enrolled and observed for 28 days. The second and third subjects will be enrolled with a 7-day interval.
The high-dose group (1.2E+11 vg/eye) is planned to include 3 subjects. Subjects in the high-dose group will be enrolled and administered the drug in sequence after passing the screening. There will be at least a 1-week interval between each subject. The timing of enrolling the full 3 subjects or stopping enrollment will be determined by the investigator's assessment of safety.All subjects will receive intravitreal injection of the study drug UGX202 after enrollment and will be followed up for 52 weeks to evaluate the safety, tolerability, and preliminary efficacy of UGX202.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Provide written informed consent form (ICF).
- •Age ≥18 years at ICF signing.
- •Diagnosed as non-syndromic RP；
- •BCVA \> logMAR 1.9 (assessed by FrACT) in the study eye.
- •Confirmation of preserved memory of visual experience
- •Spherical equivalent between -9D and +6D.
排除标准
- •Prior gene therapy in either eye.
- •Received any interventional investigational drug within 90 days prior to screening.
- •Any Study eye disease or systemic disease judged by the investigator to affect visual function assessment.
- •Hypersensitivity to corticosteroids, intolerance to corticosteroid regimen, active concurrent infection contraindicating treatment.
- •History or tendency of psychiatric disorders impacting safety and/or efficacy assessment.
- •Any other factor deemed unsuitable by the investigator.
结局指标
主要结局
Incidence of adverse events and serious adverse events
时间窗: baseline to Day 3, Day 7, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 36, Week 52
From the time of administration of UGX202 injection until the 52nd week, based on the topical and systemic safety data, the incidence rates of AEs, TEAEs during treatment, TRAEs related to the study drug, TRAEs related to the study procedures, SAEs, TRSAEs related to the study drug, and TRSAEs related to the study procedures during the study period were summarized by the investigators, and the correlations between AEs and the study drug and study procedures were determined.
The average change in IOP
时间窗: baseline to Day 3, Day 7, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 36, Week 52
The average change in IOP of the study eyes and non-study eyes from after treatment to the 52nd week compared to the baseline. The IOP was measured three times consecutively at each visit and the average value was taken.
次要结局
- The changes in BCVA(baseline to Day 3, Day 7, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 36, Week 52)
- The changes in the average stimulus threshold(baseline to Week 4, Week 12, Week 24, Week 52)
- The changes in the visual function questionnaire (VFQ-25) scores(baseline to Day 3, Day 7, Week 2, Week 4, Week 6, Week 8, Week 12, Week 24, Week 36, Week 52)