A Phase 2, Open-label, Multicenter, Extension Study to Investigate the Long-term Safety and Efficacy of S-606001 in Patients With Late-onset Pompe Disease
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 60
- 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 long-term safety and tolerability profile of S-606001 in participants with LOPD.
Participants who completed other S-606001 studies (for example, NCT07123155), including participants in a S-606001 study whose allocated arm was terminated by the sponsor and who are willing to participate in this study, will be enrolled globally.
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
- •Participants who previously completed a S-606001 Pompe treatment study, including participants whose assigned treatment arm was terminated by the sponsor
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
- •Current enrollment in any other clinical study involving an investigational intervention or any other type of medical research
- •Participant, if female, is pregnant or breastfeeding at visit 1
- •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
S-606001
Participants will receive S-606001. Participants who were previously using enzyme replacement therapy (ERT) at the time of enrollment will receive the same regimen of ERT as standard of care.
干预措施: ERT (Drug)
S-606001
Participants will receive S-606001. Participants who were previously using enzyme replacement therapy (ERT) at the time of enrollment will receive the same regimen of ERT as standard of care.
干预措施: S-606001 (Drug)
结局指标
主要结局
Number of Participants With Treatment-emergent Adverse Events
时间窗: Baseline through Week 96
次要结局
- Change from Baseline in Patient-Reported Outcomes Measurement Information System v2.0 Pain Intensity 3a (PROMIS v2.0 Pain)(Baseline, Week 96)
- Change from Baseline in Daily Living Activity Levels as Assessed by the Investigator(Baseline, Week 96)
- Change from Baseline in Patient-Reported Outcomes Measurement Information System Fatigue Short Form 8a (PROMIS Fatigue-8a)(Baseline, Week 96)
- Change from Baseline in Patient-Reported Outcomes Measurement Information System Item Bank v2.0 - Physical Function - Short Form 20a (PROMIS-Item Bank-PF-20a)(Baseline, Week 96)
- Change from Baseline in Patient-Reported Outcomes Measurement Information System Dyspnea Characteristics (PROMIS-Dyspnea)(Baseline, Week 96)
- Change from Baseline in Percent Forced Vital Capacity (%FVC)(Baseline, Week 96)
- Change from Baseline in 6-minute Walk Test (6MWT)(Baseline, Week 96)
- Change from Baseline in Gait, Stair, Gower's Maneuver, and Chair (GSGC) Score(Baseline, Week 96)
- Change from Baseline in 36-item Short Form Health Survey (SF-36)(Baseline, Week 96)
- Change from Baseline in Patient Global Impression of Severity (PGI-S)(Baseline, Week 96)