A Phase 1/2 Intracerebroventricular and Intravitreal Administration of NGN-101 for Treatment of Neuronal Ceroid Lipofuscinosis (NCL) Subtype 5 (CLN5) Disease
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 6
- 试验地点
- 4
- 主要终点
- Incidence of Serious Adverse Events (SAEs)
研究概览
简要总结
This is a prospective, non-randomized, open-label, dose escalation study of a single administration of gene therapy in children who are 3 to 9 years old with Neuronal Ceroid Lipofuscinosis (Batten) Subtype 5 (CLN5) disease.
详细描述
The study is a first in human (FIH) open-label, dose escalation study designed to assess the safety and efficacy of administration of an adeno-associated viral vector serotype 9 (AAV9) carrying the gene encoding human ceroid-lipofuscinosis neuronal protein 5 (CLN5) in subjects with CLN5 Batten disease. The study treatment will be delivered via intracerebroventricular (ICV) and intravitreal (IVT) injection on the same day. Each participant will be followed for safety and efficacy for 5 years after treatment. Efficacy assessments in this study will evaluate motor, language, visual and cognitive function.
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
- 3 Years 至 9 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
结局指标
主要结局
Incidence of Serious Adverse Events (SAEs)
时间窗: 5 years (multiple visits)
Incidence, type, severity, and frequency of SAEs
Incidence of clinical laboratory abnormalities
时间窗: 5 years (multiple visits)
Incidence, type, severity, and frequency of clinical laboratory abnormalities
Incidence of Treatment Emergent Adverse Events (TEAEs)
时间窗: 5 years (multiple visits)
Incidence, type, severity, and frequency of TEAEs
Incidence of new nerve conduction study (NCS) abnormalities
时间窗: 5 years (multiple visits)
Incidence, type, severity, and frequency of new nerve conduction study (NCS) abnormalities
Incidence of new physical and neurologic exam abnormalities
时间窗: 5 years (multiple visits)
Incidence, type, severity, and frequency of new physical and neurologic exam abnormalities
次要结局
- Change in Spectral Domain-Optical Coherence Tomography (SD-OCT)(5 years (multiple visits))
- Change in Unified Batten Diseases Rating Scale (UBDRS)(5 years (multiple visits))
- Change in color vision(5 years (multiple visits))
- Change in Hamburg Scale, Motor and Language domain scores(5 years (multiple visits))
- Change in visual acuity measurements(5 years (multiple visits))
- Change in Caregiver global impression of change(5 years (multiple visits))