Phase 1 Open-Label, Dose-Escalation Study of the Safety, Tolerability and Preliminary Efficacy of Intravitreal 4D-110 in Patients With Choroideremia
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 13
- 试验地点
- 2
- 主要终点
- Frequency and severity of ocular and systemic adverse events (AEs)
研究概览
简要总结
This study will evaluate safety, tolerability, and preliminary efficacy of a single intravitreal (IVT) injection of a recombinant adeno-associated virus (AAV) gene therapy, 4D-110, in male patients with genetically-confirmed Choroideremia (CHM).
详细描述
This is an open-label, Phase 1 study to evaluate safety and tolerability as well as preliminary efficacy of a single IVT injection of 4D-110 at three dose levels in male patients with genetically-confirmed CHM.
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
- Male
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of CHM defined as a pathogenic mutation in the CHM gene, confirmed by genetic testing
- •Both eyes must have ≥ 34 ETDRS letters (~20/200)
排除标准
- •Clinically significant, active ocular or peri-ocular infection or inflammation in the study eye
- •Patient has previously received any AAV treatment
- •Ocular conditions or ocular media opacity in either eye that would preclude the planned treatment (i.e. IVT injection) or interfere with the interpretation of study endpoints (e.g. significant lens opacity)
研究组 & 干预措施
4D-110 Dose 1
4D-110 IVT injection
干预措施: 4D-110 (Biological)
4D-110 Dose 2
4D-110 IVT injection
干预措施: 4D-110 (Biological)
4D-110 Dose 3
4D-110 IVT injection
干预措施: 4D-110 (Biological)
结局指标
主要结局
Frequency and severity of ocular and systemic adverse events (AEs)
时间窗: 24 months
Frequency and severity of ocular and systemic AEs including clinically significant changes in ocular evaluations, systemic examinations and laboratory testing.
次要结局
未报告次要终点