NCT07123155招募中2 期
A Phase 2, Multicenter, Randomized, Placebo-controlled, Double-blind Study to Investigate the Safety, Pharmacodynamics, and Preliminary Efficacy of S-606001 as an Add-on to Enzyme Replacement Therapy in Patients With Late-onset Pompe Disease
Shionogi51 个研究点 分布在 9 个国家目标入组 45 人开始时间: 2025年10月30日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 45
- 试验地点
- 51
- 主要终点
- Change From Baseline in Percent Forced Vital Capacity (%FVC) at Week 52
研究概览
简要总结
The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an add-on to ERT.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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 ≥18 years of age and ≥40 kilograms (kg) of body weight at the time of signing the informed consent.
- •Participant must have a diagnosis of LOPD based on documentation of 1 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 ≥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
- •Participants must be ERT-experienced, defined as currently receiving ERT and having been receiving ERT for ≥24 months, with no regimen change in the last 6 months.
排除标准
- •Has a medical condition or any other extenuating circumstance that may 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 glycogenin gene (GYG) or in glycogen phosphorylase muscle associated gene(PYGM) .
- •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 or small interfering ribonucleic acid (RNA) therapy for Pompe disease.
- •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.
研究组 & 干预措施
Placebo
Placebo Comparator
Participants will receive S-606001 matching placebo BID after a meal for 52 weeks.
干预措施: Placebo (Drug)
S-606001 High Dose
Experimental
Participants will receive S-606001 at a high dose level BID after a meal for 52 weeks.
干预措施: S-606001 (Drug)
S-606001 Low Dose
Experimental
Participants will receive S-606001 at a low dose level twice daily (BID) after a meal for 52 weeks.
干预措施: S-606001 (Drug)
结局指标
主要结局
Change From Baseline in Percent Forced Vital Capacity (%FVC) at Week 52
时间窗: Baseline, Week 52
次要结局
- Number of Participants With Treatment-emergent Adverse Events (TEAEs)(Baseline up to Week 53)
- Change From Baseline in Pulmonary Function Parameter: Maximal Inspiratory Pressure (MIP) at Week 52(Baseline, Week 52)
- Change From Baseline in Pulmonary Function Parameter: Maximal Expiratory Pressure (MEP) at Week 52(Baseline, Week 52)
- Change From Baseline in Motor Function Parameter: Gait, Stair, Gower's Maneuver, Chair (GSGC) Score at Week 52(Baseline, Week 52)
- Change From Baseline in Patient-Reported Outcomes Measurement Information System Fatigue Short Form 8a (PROMIS-Fatigue-8a) Score at Week 52(Baseline, Week 52)
- Change From Baseline in Patient-Reported Outcomes Measurement Information System Physical Function 20-item short form (PROMIS-PF-20) Score at Week 52(Baseline, Week 52)
- Change From Baseline in Patient-Reported Outcomes Measurement Information System v2.0 Pain Intensity 3a (PROMIS v2.0 PAIN) Score at Week 52(Baseline, Week 52)
- Change From Baseline in 36-item Short Form Health Survey (SF-36) Score at Week 52(Baseline, Week 52)
- Change From Baseline in Patient Global Impression of Severity (PGI-S) Score at Week 52(Baseline, Week 52)
- Plasma Concentration of S-606001(Up to Week 12)
- Change From Baseline in Serum Creatine Kinase Levels at Week 52(Baseline, Week 52)
- Change From Baseline in 6-minute Walk Test (6MWT) at Week 52(Baseline, Week 52)
研究者
研究点 (51)
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