Phase 1/2 Multicenter, Open-label Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-3001 in Participants with Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Safety and Tolerability
研究概览
简要总结
This study will be conducted to evaluate the safety, tolerability, clinical activity, pharmacokinetics, and pharmacodynamics of NTLA-3001 in adults with alpha-1 antitrypsin deficiency (AATD) -associated lung disease
详细描述
This study consists of 2 parts: Phase 1 is an open-label, single-arm ascending dose study to characterize the safety and activity of NTLA3001 and identify the dose for evaluation in Phase 2. Phase 2 will follow as an open-label, dose expansion study to further characterize the safety and clinical activity of NTLA-3001 and provide an initial assessment of the effect of NTLA-3001 on clinical measures of pulmonary function.
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
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- •AATD genotypes outside of ZZ or ZNull
- •Participants with total antibodies to adeno-associated virus (AAV) serotype above laboratory assay cut off
- •Participants who have known negative reaction or hypersensitivity to any lipid nanoparticles (LNP) component.
- •Any condition that, in the Investigator's opinion, could adversely affect the safety of the participant.
- •Unwilling to comply with study procedures.
研究组 & 干预措施
Arm 1
干预措施: Biological NTLA-3001 (Biological)
结局指标
主要结局
Safety and Tolerability
时间窗: From NTLA-3001 infusion up to week156 post infusion
To evaluate the safety and tolerability of NTLA-3001 as determined by adverse events (AEs) and dose limiting toxicities (DLTs)
次要结局
- Pharmacodynamics(From NTLA-3001 infusion up to week 156 post infusion)
- Immune Response(From NTLA-3001 infusion up to week 156 post infusion)
- Vector Shedding(From NTLA-3001 infusion up to week 156 post infusion, only during phase 2.)