A Randomized, Double-Blind, Placebo-Controlled, Single Oral Dose Study Evaluating the Safety and Pharmacokinetics of BMN 349 in Homozygous for the Z Mutation of Alpha 1 Antitrypsin Gene (PiZZ) and Heterozygous for the Z Mutation (PiMZ/MASH)
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
- 15
- 主要终点
- Participant Adverse Events, Serious Adverse Events, Dose Limit Toxicities, Adverse Event of Special Interests, abnormal laboratory tests, abnormal pulmonary function tests, and 12-lead ECG parameters
研究概览
简要总结
The goal of this clinical trial is to assess the safety and tolerability of a single oral dose of BMN 349 in participants with PiZZ or PiMZ/MASH.
Primary outcome measures include incidence of any adverse events (including serious adverse events, dose limit toxicities, and adverse events of special interest), incidence of any laboratory test abnormalities, incidence of lung function test abnormalities and 12-lead ECG parameters.
Participants will receive a single dose of either BMN 349 or placebo and then monitored for safety and tolerability.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
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
- •Participants must have confirmation of PiZZ or PiMZ genotype
- •Females and males, of any race, 18 to 75 years of age
- •Nonsmokers, defined as not using tobacco or nicotine-containing products for at least 6 months prior to Screening
排除标准
- •International normalized ratio (INR) > 1.2
- •Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels > 125 U/L
- •Current or recent use of AAT augmentation therapy
- •Participants with recent (last 3 months) diagnosis of pneumonia
研究组 & 干预措施
Group A (PiZZ)
5:1 (349:Placebo)
干预措施: BMN 349 (Drug)
Group A (PiZZ)
5:1 (349:Placebo)
干预措施: Placebo (Drug)
Group B (PiMZ)
5:1 (349:Placebo)
干预措施: BMN 349 (Drug)
Group B (PiMZ)
5:1 (349:Placebo)
干预措施: Placebo (Drug)
结局指标
主要结局
Participant Adverse Events, Serious Adverse Events, Dose Limit Toxicities, Adverse Event of Special Interests, abnormal laboratory tests, abnormal pulmonary function tests, and 12-lead ECG parameters
时间窗: 78 days
Number of participant AEs, SAEs, DLTs, AESIs per physician's assessment, abnormal laboratory tests through whole blood samples, abnormal pulmonary function spirometry tests, and 12-lead ECG parameters changes from baseline following a single oral dose of BMN 349
次要结局
- C max(78 days)
- AUC 0-t(78 days)
- AUC 0-inf(78 days)
- CL/F(78 days)
- T max(78 days)
- t 1/2(78 days)
- Vz/F(78 days)
- Assess functional activity of circulating Alpha1 AntiTrypsin in participants(78 days)