A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease (LOPD)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 119
- 试验地点
- 116
- 主要终点
- Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Discontinuation of Study Drug
研究概览
简要总结
This is a multicenter, international open-label extension study of ATB200/AT2221 in adult subjects with late-onset Pompe disease (LOPD) who completed Study ATB200-03.
详细描述
This is an open-label extension study for subjects who completed the ATB200-03 study. The subjects will stay in this study until regulatory approval or marketing authorization and/or commercialization in the participating subject's country.
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
- •1. Subject must have completed Study ATB200-
- •Note: Subjects who were forced to withdraw from Study ATB200-03 for a logistical reason not related to the efficacy or safety of cipaglucosidase alfa/miglustat (eg, hospitalization for a car accident, COVID-19 pandemic, or emergency surgery) that resulted in several consecutive missed doses may have been eligible to participate in this study upon approval by the Amicus medical monitor.
排除标准
- •Subject plans to receive gene therapy or participate in another interventional study for Pompe disease.
- •Subject, if female, is pregnant or breastfeeding.
- •Subject, whether male or female, is planning to conceive a child during the study.
- •Subject had a hypersensitivity to any of the excipients in cipaglucosidase alfa or miglustat, or had a medical condition or any other extenuating circumstance that may have, in the opinion of the investigator or medical monitor, posed an undue safety risk to the subject or may have compromised his/her ability to comply with or adversely impacted protocol requirements.
研究组 & 干预措施
ATB200/AT2221
Participants received ATB200 (cipaglucosidase alfa) co-administered with AT2221 capsule (miglustat)
干预措施: AT2221 (Drug)
ATB200/AT2221
Participants received ATB200 (cipaglucosidase alfa) co-administered with AT2221 capsule (miglustat)
干预措施: ATB200 (Biological)
结局指标
主要结局
Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and TEAEs Leading to Discontinuation of Study Drug
时间窗: Entire extension study (mean = 40.5 months on treatment)
Number of subjects with TEAE, TESAE, and TEAE leading to discontinuation during this long-term extension study
次要结局
- Change From Baseline in 6-Minute Walk Distance (6MWD)(baseline, Week 208)
- Change From Baseline in Sitting % Predicted Forced Vital Capacity (FVC)(baseline, Week 208)
- Change From Baseline in Manual Muscle Testing (MMT) Lower Extremity Score(baseline, Week 208)
- Change From Baseline in the Total Score for Patient-reported Outcomes Measurement Information System (PROMIS®) - Physical Function(baseline, Week 208)
- Change From Baseline in Gait, Stairs, Gower, Chair (GSGC) Test(baseline, Week 208)
- Change From Baseline in Overall Physical Well-being (Subject's Global Impression of Change [SGIC], Question 1)(baseline, Week 208)
- Change From Baseline in Physician's Global Impression of Change (PGIC) Overall Status(baseline, Week 208)
- Percent Change From Baseline in Creatine Kinase (U/L)(baseline, Week 208)
- Percent Change From Baseline in Urine Hex4 (mmol/Mol Creatinine)(baseline, Week 208)
- Proportion of Subjects With Positive Anti-drug Antibodies at Baseline and Week 208(baseline, Week 208)
- Change From Baseline in the Total Score for PROMIS® - Fatigue(baseline, Week 208)
- Change From Baseline in EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) Responses(baseline, Week 208)