An Exploratory Clinical Study Evaluating LX111 Gene Therapy in Patients With Diabetic Macular Edema (DME)
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 32
- 试验地点
- 2
- 主要终点
- Adverse events and serious adverse events in the eyes and throughout the body within 364 days after LX111 treatment
研究概览
简要总结
The goal of this study is to evaluate the safety and efficacy of LX111 treatment of DME. This study will enroll participants aged ≥ 18 vears old to receive a single unilateral intravitreal (lVT) injection of LX111 to evaluate its safety and efficacy.
详细描述
This trial is a prospective, multicenter, dose-ranging trial to evaluate the safety and efficacy of LX111 in participants with DME. The trial will be conducted in two parts: Dose Confirmation and Dose Expansion.
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
- •Willing to sign the informed consent, and willing to attend follow-up visits;
- •Type I or Type II diabetes mellitus with macular thickening secondary to DME involving the center of the fovea;
- •CST ≥ 300 μm in the study eye at Screening;
- •BCVA ETDRS letters between 19 and 73;
- •Participants must have received anti-VEGF therapy within 12 months prior to screening and demonstrated a meaningful response;
- •Male subjects whose partner is a fertile female or female subjects who are fertile, agree to take effective contraceptive measures from the screening period until the last follow-up.
排除标准
- •Active proliferative diabetic retinopathy (PDR);
- •Presence of iris neovascularization in the study eye at Screening;
- •Retinal laser photocoagulation in the study eye within 3 months prior to Screening;
- •Prior gene therapy in the study eye;
- •The study eye has been treated with an intravitreal dexamethasone implant (Ozurdex®) within 6 months prior to Screening.
- •Systemic anti-VEGF treatment within 3 months before Screening;
- •Received an investigational drug, agent, device, or therapy (ocular or non-ocular) in the 3 months (or at least 5 half-lives, whichever is longer) prior to Screening;
研究组 & 干预措施
LX111 Dose Escalation up to 2 dose levels
Qualified participants will receive a single unilateral intravitreal injection of LX111 at Day 0.
干预措施: LX111 Injection (Genetic)
LX111 Dose Expansion Dose 1
Qualified participants will receive a single unilateral intravitreal injection of LX111 at Day 0.
干预措施: LX111 Injection (Genetic)
LX111 Dose Expansion Dose 2
Qualified participants will receive a single unilateral intravitreal injection of LX111 at Day 0.
干预措施: LX111 Injection (Genetic)
结局指标
主要结局
Adverse events and serious adverse events in the eyes and throughout the body within 364 days after LX111 treatment
时间窗: 52 weeks
Incidence of adverse events and serious adverse events within 52 weeks of LX111 intravitreal injection in each dose group.
Dose Limiting Toxicity
时间窗: 4 weeks
The incidence of DLT in each dose group.
次要结局
- Mean changes in BCVA from Baseline(12 weeks, 36 weeks, 52 weeks)
- Mean changes in Central Subfield Thickness (CST) from Baseline(12 weeks, 36 weeks, 52 weeks)
- The percentage of participants who received anti-VEGF supplemental injection within 52 weeks after LX111 treatment(52 weeks)
- Proportion of participants achieving an improvement or worsening in DR in the study eye per the ETDRS-DRSS on ultra-wide field fundus photography.(52 weeks)
研究者
Xiaodong Sun
Professor
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine