A Phase 1, Multicenter, Open-label, Dose-Finding Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection of INS1202 in Patients With Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 23
- 试验地点
- 7
- 主要终点
- Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)
研究概览
简要总结
The primary objective of this dose-finding study is to evaluate the safety, tolerability and pharmacodynamics of single dose of INS1202 via IT administration in participants ≥ 18 to <80 years of age with ALS who carry superoxide dismutase type 1 (SOD1) mutations or harbor no known ALS-related genetic mutation.
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 79 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant with body mass index (BMI) ≥18 kilograms per square meter (kg/m^2).
- •Participant with symptomatic ALS as diagnosed by Gold Coast diagnostic criteria.
- •Sporadic ALS cohorts: Negative testing for known monogenic mutations associated with familial ALS.
- •SOD1-ALS (Cohorts 2 and 3 only): Confirmed pathogenic SOD1 mutation, with negative testing for other genetic mutations associated with familial ALS.
- •Any polymorphism or mutation in the coding region will require additional review by the Sponsor to determine compatibility with the study intervention.
- •Baseline ALSFRS-R ≥
- •ALS disease duration ≤ 42 months.
排除标准
- •Previous treatment for ALS with cellular or gene therapies.
- •Any investigational medication or treatment (for ALS or other condition).
- •Note: Other protocol-defined inclusion/exclusion criteria may apply.
研究组 & 干预措施
Cohort 3
Participants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 3 on Day 1.
干预措施: INS1202 (Genetic)
Cohort 1
Participants with sporadic amyotrophic lateral sclerosis (sALS) will be administered INS1202, IT injection at dose level 1 on Day 1.
干预措施: INS1202 (Genetic)
Cohort 2
Participants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 2 on Day 1.
干预措施: INS1202 (Genetic)
结局指标
主要结局
Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)
时间窗: Up to 48 weeks
次要结局
- Recommended Phase 2 Dose (RP2D) of INS1202-101(Up to 48 weeks)
- Viral Vector Shedding Following the IT Administration of INS1202 by Droplet Digital Polymerase Chain Reaction (ddPCR)(Baseline, Week 1, Week 2, Week 4, Week 16 and Week 32)