NCT04808505招募中3 期
An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 Years
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 36
- 试验地点
- 14
- 主要终点
- Proportion of subjects with infusion-associated reactions (IARs)
研究概览
简要总结
This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.
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
- — 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female subjects who are aged 6 months to < 18 years on Day 1
- •Subject must have documentation of IOPD genotype
- •Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
- •Subject must have received ERT for at least 6 months immediately before enrollment. For subjects whose ERT dosage has been modified, the subject must have been on the modified dosage and regimen for at least 3 months before enrollment
- •Subjects aged ≥ 12 to < 18 years must perform one valid 6-minute walk test (6MWT) (≥ 75 meters) at screening; Subjects aged ≥ 5 to < 12 years must perform one valid 6MWT (≥ 40 meters) at screening; Subjects aged 18 months to < 5 years must be ambulatory and assessed to be likely to be able to perform 6MWT (≥ 40 meters) when they turn 5 years old
- •Subjects must have experienced a clinical decline on their current rhGAA dose and frequency
- •Male or female subjects who are aged 0 to <6 months at Day 1
- •Subject must have documentation of IOPD genotype
- •Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
- •Subject is ERT-naïve
- •Long-term Extension (Cohort 1 or Cohort 2):
- •1. Subject must have, in the opinion of the investigator, benefited from therapy with cipaglucosidase alfa/miglustat during the 104-week primary treatment period with no significant safety concerns.
排除标准
- •Cohort 1 and Cohort 2, unless specified
- •Subject requires invasive ventilation (eg, tracheostomy)
- •Subject is CRIM negative and has not received prophylactic immunomodulation (Cohort 1); Subject is CRIM negative and will not be receiving prophylactic immunomodulation (Cohort 2)
- •Subject has a history of life-threatening IARs/hypersensitivity (eg, anaphylaxis and severe cutaneous reactions) to ERT (eg, alglucosidase alfa, cipaglucosidase alfa, miglustat) or other iminosugars, or to any of the excipients, where rechallenge was unsuccessful
- •Subject has prior history of illness or condition known to affect motor function
- •Female subject is pregnant (or intends to get pregnant) or breastfeeding at screening (Cohort 1)
研究组 & 干预措施
Cohort 1: Cipaglucosidase Alfa/Miglustat in ERT-experienced pediatric IOPD subjects
Experimental
Pediatric IOPD subjects 6 months to <18 years experiencing clinical decline
干预措施: Cipaglucosidase alfa (Biological)
Cohort 1: Cipaglucosidase Alfa/Miglustat in ERT-experienced pediatric IOPD subjects
Experimental
Pediatric IOPD subjects 6 months to <18 years experiencing clinical decline
干预措施: Miglustat (Drug)
Cohort 2: Cipaglucosidase Alfa/Miglustat in ERT-naïve pediatric IOPD subjects
Experimental
Pediatric IOPD subjects <6 months
干预措施: Cipaglucosidase alfa (Biological)
Cohort 2: Cipaglucosidase Alfa/Miglustat in ERT-naïve pediatric IOPD subjects
Experimental
Pediatric IOPD subjects <6 months
干预措施: Miglustat (Drug)
结局指标
主要结局
Proportion of subjects with infusion-associated reactions (IARs)
时间窗: 104 weeks
次要结局
未报告次要终点
研究者
研究点 (14)
Loading locations...
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