Phase 1/2, Open-Label, Dose-Finding Followed by 2-Year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Adolescent Subjects With Stargardt Disease
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- RBP4 Pty Ltd
- 入组人数
- 13
- 试验地点
- 3
- 主要终点
- To evaluate systemic and ocular safety and tolerability of tinlarebant.
研究概览
简要总结
Stargardt disease 1 (STGD1) is the most prevalent form of juvenile macular degeneration. It is caused by a rare, inherited autosomal recessive trait, leading to severe and irreversible blindness by the first or second decade of life. Earlier onset of the disease is related to a rapid vision loss, while patients with a later onset tend to have a better prognosis.
This study will enrol subjects aged 12-18 years old with a confirmed clinical diagnosis of Stargardt disease type 1 (STGD1). This study will include 2 phases, the phase 1b portion is to determine the optimal dose for phase 2 based on the extent of retinol binding protein 4 (RBP4) reduction after 2 cycles of tinlarebant treatment. The phase 2 portion will evaluate the safety and efficacy of a single daily dose of tinlarebant over a 24-month treatment period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject must have clinically diagnosed Stargardt disease with at least one mutation identified in the ABCA4 gene.
排除标准
- •Any ocular disease other than Stargardt disease at baseline that, in the opinion of the PI, would complicate assessment of a treatment effect.
研究组 & 干预措施
tinlarebant
Daily, oral administration of one tinlarebant.
干预措施: tinlarebant (Drug)
结局指标
主要结局
To evaluate systemic and ocular safety and tolerability of tinlarebant.
时间窗: From baseline to 24 months
To evaluate safety and tolerability of daily dosing of tinlarebant assessed by incidence and/or severity of ocular and non-ocular adverse events.
The optimal dose for Phase 2.
时间窗: Up to 24 months
To determine optimal dose of tinlarebant administered orally in adolescent patients with Stargardt Disease.
次要结局
- Maximum Plasma Concentration (Cmax) of tinlarebant in plasma.(Up to 24 months)
- Half-life (t1/2) of tinlarebant in plasma.(Up to 24 months)
- Time to Maximum Plasma Concentration (Tmax) of tinlarebant in plasma.(Up to 24 months)
- Time to minimal plasma RBP4 level (Tmin)(Up to 24 months)
- Change in atrophic lesion size.(From baseline to 24 months.)
- Minimum concentration of RBP4 (Cmin)(Up to 24 months)