NCT06405633进行中（未招募）1 期
A Phase 1b/2a Open-label Single Ascending Doses (SAD) and Multiple Ascending Doses (MAD) Research Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Participants With AATD Pi*ZZ on WVE-006 (RestorAATion-2)
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 24
- 试验地点
- 17
- 主要终点
- The proportion of participants with adverse events
研究概览
简要总结
The purpose of this open-label study is to assess the safety, tolerability, pharmacodynamics, and pharmacokinetics of WVE-006 in participants with alpha-1 antitrypsin deficiency (AATD) following Period 1 single ascending dose (SAD) and Period 2 multiple ascending doses (MAD), respectively.
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 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Healthy as determined by the Investigator, based on a medical evaluation, or has mild to moderate AATD-induced lung disease (FEV1 ≥50%) and/or stable mild AATD-induced liver disease (≤F2 (≤10 kPa) on FibroScan.
- •Genetic testing confirming Pi*ZZ.
- •Participant has been a non-smoker for at least 1 year prior to screening.
排除标准
- •• Participant has a history of multiple drug allergies or of allergic reaction to an oligonucleotide or to N-acetylgalactosamine (GalNAc).
- •Participant has a history of intolerance or any medical condition that might interfere with subcutaneous injections.
- •Any ongoing or recent infections.
- •Any recent or planned vaccinations during the study.
- •Participant has a history of regular alcohol consumption exceeding 14 standard drinks/week.
- •Unwilling to abstain from alcohol for 48 hours prior to dosing at each of the dosing visits.
- •Any recent or planned major surgery during the study.
- •Participant has any medical condition or social circumstance that, in the opinion of the Investigator, would make the participant unsuitable for participation in the study or for dosing on Day 1, or could interfere with the assessments of safety, pharmacodynamics, or pharmacokinetics, or completion of the study.
- •Participant currently on AAT augmentation therapy, planned to be on augmentation therapy anytime during the study, or has been on augmentation therapy within 30 days prior to Screening Visit.
- •Donation of blood or blood products in excess of 500 mL within 12 weeks prior to Screening Visit and/or unwilling to refrain from blood donation for the duration of the study.
- •Participant has received an investigational agent within 3 months of the Screening Visit.
研究组 & 干预措施
WVE-006 (Dose 3)
Experimental
干预措施: WVE-006 (Drug)
WVE-006 (Dose 2)
Experimental
干预措施: WVE-006 (Drug)
WVE-006 (Dose 1)
Experimental
干预措施: WVE-006 (Drug)
结局指标
主要结局
The proportion of participants with adverse events
时间窗: Up to 36 Weeks
次要结局
- Single Ascending Dose - Change from baseline in levels of serum M-AAT protein.(Up to 12 Weeks)
- Multiple Ascending Dose - Change from baseline in levels of serum M-AAT protein(Up to 24 Weeks)
- Single Ascending Dose - Area under the plasma concentration time curve for WVE-006 from time of dosing to the last measurable concentration (AUClast)(Up to 12 Weeks)
- Multiple Ascending Doses - Area under the plasma concentration time curve for WVE-006 from time of dosing to the last measurable concentration (AUClast)(Up to 24 Weeks)
- Single Ascending Dose - Maximum concentration of WVE-006 in plasma(Up to 12 Weeks)
- Multiple Ascending Doses - Maximum concentration of WVE-006 in plasma (Cmax)(Up to 24 Weeks)
研究者
研究点 (17)
Loading locations...
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