A Phase 1b Open-label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Tinlarebant in Japanese Subjects With Stargardt Disease and a Phase 2/3 Randomized, Double-masked, and Placebo-controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Tinlarebant in Subjects With Stargardt Disease
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 60
- 试验地点
- 32
- 主要终点
- To measure the annualized rate of change from baseline lesion size in aggregate area of atrophy
研究概览
简要总结
The goal of this clinical trial is to evaluate the safety, tolerability, and efficacy of tinlarebant in subjects with Stargardt Disease
详细描述
This study will be comprised of a Phase 1b part conducted in Japan and a Phase 2/3 part. The Phase 1b part of the study will be open-label and will evaluate the pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of daily doses of 5 mg tinlarebant, administered for 7 days, in Japanese subjects with Stargardt Disease (STGD1). The Phase 2/3 part of the study will be randomized, double masked, and placebo controlled to evaluate the safety, tolerability, and efficacy of daily doses of 5 mg tinlarebant, administered for 24 months, in subjects with STGD1.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 12 Years 至 20 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects must have clinically diagnosed STGD1 with at least one mutation identified in the ABCA4 gene.
- •Subjects must have a defined aggregate atrophic lesion in 1 or both eyes.
- •Minimum BCVA is required in the study eye
排除标准
- •Any ocular disease other than STGD1 that, in the opinion of the investigator, would complicate assessment of a treatment effect.
- •History of ocular surgery in the study eye in the last 3 months.
- •Any prior gene therapy.
研究组 & 干预措施
LBS-008, Tinlarebant
5 mg tablet taken orally once a day
干预措施: Tinlarebant (Drug)
Placebo
Placebo tablets for tinlarebant 5 mg prepared similarly
干预措施: Placebo (Drug)
结局指标
主要结局
To measure the annualized rate of change from baseline lesion size in aggregate area of atrophy
时间窗: From baseline to Month 24
次要结局
- Change in BCVA measured by the ETDRS method(From baseline to Month 24)
- To measure the annualized rate of change in total area of atrophy(From baseline to Month 24)