An Open-label, Multicenter, Multinational, Ascending Dose Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Repeated Biweekly Infusions of neoGAA in naïve and Alglucosidase Alfa Treated Late-onset Pompe Disease Patients.
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 24
- 试验地点
- 17
- 主要终点
- Vital signs
研究概览
简要总结
Primary Objective:
To evaluate the safety and tolerability of neoGAA in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.
Secondary Objective:
To evaluate the pharmacokinetics, pharmacodynamics of neoGAA in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.
To evaluate the effect of neoGAA on exploratory efficacy endpoints in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.
详细描述
Screening: within 90 days Period of treatment: 24 weeks (including 13 bi-weekly infusions) Post treatment evaluation visit: 2 weeks after last neoGAA infusion (at Week 27) End of study visit: 4 weeks after last neoGAA infusion (at Week 29) Total duration: approximately 41 weeks
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 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
- 未提供
排除标准
- 未提供
研究组 & 干预措施
GZ402666 (neoGAA) Group 1 - 5 mg
Intravenous infusion of 5mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks
干预措施: GZ402666 (Drug)
GZ402666 (neoGAA) Group 1 - 10 mg
Intravenous infusion of 10mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks.
干预措施: GZ402666 (Drug)
GZ402666 (neoGAA) Group 1 - 20 mg
Intravenous infusion of 20mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks.
干预措施: GZ402666 (Drug)
GZ402666 (neoGAA) Group 2 - 5 mg
Intravenous infusion of 5mg neoGAA once every other week for a total of 24 weeks to late onset Pompe disease patients previously treated with alglucoside alfa.
干预措施: GZ402666 (Drug)
GZ402666 (neoGAA) Group 2 - 10 mg
Intravenous infusion of 10mg neoGAA once every other week for a total of 24 weeks to late onset Pompe disease patients previously treated with alglucoside alfa.
干预措施: GZ402666 (Drug)
GZ402666 (neoGAA) Group 2 - 20 mg
Intravenous infusion of 20mg neoGAA once every other week for a total of 24 weeks to late onset Pompe disease patients previously treated with alglucoside alfa.
干预措施: GZ402666 (Drug)
结局指标
主要结局
Vital signs
时间窗: screening/baseline to Week 25
Adverse events
时间窗: screening/baseline to Week 25
Laboratory assessments including hematology, biochemistry and urinalysis
时间窗: screening/baseline to Week 25
次要结局
- Skeletal muscle glycogen content(screening/baseline, Week 27)
- Electrocardiogram(screening/baseline, Week 1, Week 13, Week 25)
- Cmax(Week 1, Week 13, Week 25)
- t1/2(Week 1, Week 13, Week 25)
- Skeletal muscle magnetic resonance images for qualitative and quantitative muscle degenerative assessments.(screening/baseline, Week 27)
- Quality of life assessments(screening/baseline, Week 13, Week 25)
- AUC(Week 1, Week 13, Week 25)
- Immunogenicity assessments(screening/baseline to Week 29)
- Functional assessments including 6 Minute Walk Test (6MWT)(screening/baseline, Week 13, Week 25)
- Urinary Hex4(screening/baseline to Week 25)