NCT07787650尚未招募1 期
A Phase 1b, Multicenter, Single-arm, Open-label Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of S-606001 as a Monotherapy in Adult Patients With Late-onset Pompe Disease
Shionogi0 个研究点目标入组 15 人开始时间: 2026年9月30日最近更新:
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 15
- 主要终点
- Number of Participants with Treatment-emergent Adverse Events
研究概览
简要总结
The primary purpose of this study is to evaluate the safety and tolerability profile of S-606001 in participants with LOPD.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant must be ≥40 kilograms of body weight at the time of signing the informed consent form
- •Participant must have a diagnosis of LOPD based on documentation of the following: deficiency of acid alpha-glucosidase (GAA) enzyme; GAA genotype
- •Participant has a %FVC ≥30% and ≤80% in an upright position without mechanical ventilation at screening or participant has >80% FVC in upright position and ≥10% %FVC drop from upright position to supine position and %FVC ≥20% in a supine position
- •Participant performs the 6MWT at screening, as determined by the clinical evaluator, and meets all of the following criteria: screening values of 6-minute walk distance (6MWD) are ≥75 meters; screening values of 6MWD are ≤90% of the predicted value for healthy adults
- •Participant must not have received enzyme replacement therapy (ERT) for at least 3 months prior to providing informed consent and enrollment and agrees not receive any type of ERT while participating in the study
排除标准
- •Has a medical condition or any other extenuating circumstance that may, in the opinion of the investigator or medical monitor, pose an undue safety risk to the participant or may compromise his/her ability to comply with or adversely impact protocol requirements
- •Has active infections at screening
- •Malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years
- •Current or chronic history of liver disease
- •Known biallelic loss of function mutations whether in the muscle glycogen synthase gene or in the glycogen phosphorylase muscle associated gene
- •Has received any investigational therapy or pharmacological treatment for Pompe disease, within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before day 1 or is anticipated to do so during the study
- •Has received gene therapy for Pompe disease within 2 years of screening or small interfering RNA therapy for Pompe disease within 6 months of screening
- •Participant, if female, is pregnant or breastfeeding at screening
- •Participant, whether male or female, is planning to conceive a child during the study
- •Note: Other protocol-specified inclusion and exclusion criteria may apply.
结局指标
主要结局
Number of Participants with Treatment-emergent Adverse Events
时间窗: Baseline (Day 1) through Week 49
次要结局
- Change From Baseline in Percent Forced Vital Capacity (%FVC)(Baseline (Day 1), Week 48)
- Change From Baseline in Maximum Expiratory Capacity (MEP)(Baseline (Day 1), Week 48)
- Change From Baseline in Minimum Inspiratory Capacity (MIP)(Baseline (Day 1), Week 48)
- Change From Baseline in 6-minute Walk Test (6MWT)(Baseline (Day 1), Week 48)
- Change From Baseline in Gait, Stair, Gower's Maneuver, and Chair (GSGC) Score(Baseline (Day 1), Week 48)
- Change from Baseline in Daily Living Activity Levels as Assessed by the Investigator(Baseline (2-4 weeks prior to Day 1), Week 48)
- Plasma Concentration of S-606001 and Metabolites(Up to Week 28)
研究者
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