A Phase 1/2, Open-Label Study to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous ATR 1072 for the Treatment of PRKAG2 Syndrome
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 37
- 主要终点
- Incidence of treatment-emergent adverse events (TEAEs)
研究概览
简要总结
ATR 1072 is an investigational medicine being studied in adults with PRKAG2 Syndrome. This study will evaluate the safety of ATR 1072 and how the body processes the drug. Researchers will also assess how ATR 1072 affects PRKAG2 activity and measures of heart disease.
详细描述
ATR 1072 is an antibody-oligonucleotide conjugate (AOC) designed to reduce expression of PRKAG2 through targeted delivery of siRNA to cardiac tissue. This first-in-human, Phase 1/2, open-label, multicenter study will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of multiple doses of ATR 1072 in adults with PRKAG2 Syndrome. The study consists of a multiple ascending dose portion (Part A) followed by a dose-expansion cohort at the dose selected in (Part A). After completion of a 48-week treatment period, participants in Parts A and B enter a 42-week extension period followed by a post-treatment follow-up of up to 18 weeks. Approximately 37 participants will be enrolled globally.
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
盲法说明
None (Open Label)
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adults 18 to 65 years of age.
- •Pathogenic, likely pathogenic, or variant of uncertain significance (VUS) in PRKAG2 confirmed by genetic testing.
- •Clinical manifestations consistent with PRKAG2 Syndrome.
- •Able and willing to comply with study procedures.
- •Able and willing to undergo endomyocardial biopsy procedures (Part A)
排除标准
- •Pregnancy or breastfeeding.
- •Significant hepatic, renal, hematologic, or cardiovascular abnormalities that may increase study risk.
- •NYHA Class IV heart failure or left ventricular ejection fraction <45%.
- •Recent clinically significant cardiovascular events or procedures.
- •Prior treatment with prohibited investigational therapies or oligonucleotide therapies within protocol-defined washout periods.
- •Any condition that, in the investigator's
结局指标
主要结局
Incidence of treatment-emergent adverse events (TEAEs)
时间窗: From first dose through end of study (approximately 48 weeks per participant)
Incidence and severity of treatment-emergent adverse events (TEAEs).
次要结局
- Change from baseline in cardiac muscle tissue concentration of ATR 1072 siRNA(Through Week 24)
- Plasma pharmacokinetic (PK) parameters of ATR 1072(Through Week 24)
- Plasma pharmacokinetic parameters of ATR 1072(Through week 48)