NCT03911505进行中（未招募）3 期
An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 21
- 试验地点
- 34
- 主要终点
- Incidence of treatment-emergent adverse events (TEAEs) from baseline
研究概览
简要总结
This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to < 18 years
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
- 0 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female subjects (ERT-naïve [have never received a dose of rhGAA] or ERT-experienced [have received rhGAA every 2 weeks for at least 6 months immediately before enrollment, and if ERT dosage has been modified, must have been on the modified dosage for at least 3 months before enrollment]) diagnosed with LOPD who are aged 12 to <18 years at screening (Cohort 1 only) or aged 0 months to < 12 years at screening (Cohort 2 only)
- •Subject weighs ≤ 115 kg. (Cohort 1 Only)
- •Subject must have a diagnosis of LOPD based on documentation as defined in study protocol
- •If of reproductive potential and if sexually active, female and male subjects agree to use a highly effective method of contraception throughout the duration of the study and for up to 90 days after their last dose of Cipaglucosidase Alfa/Miglustat
- •Subject has a sitting forced vital capacity (FVC) ≥ 30% of the predicted value for healthy Adolescents at screening (Cohort 1 only)
- •Subject (aged 12 to <18 years; Cohort 1) performs one 6-Minute Walk Test (6MWT) (≥ 75 meters) at screening that is valid, as determined by the clinical evaluator, or subject (aged ≥ 5 to < 12 years; Cohort 2) performs one 6MWT (≥ 40 meters) at screening that is valid, as determined by the clinical evaluator
排除标准
- •Subject has received any investigational/experimental drug, oral anabolic steroid or derivative, biologic, or device within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before screening
- •Subject has received treatment with prohibited medications within 30 days of screening
- •Subject has received any gene therapy at any time
- •Subject has any intercurrent illness or condition at screening or baseline that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator and/or the medical monitor that the potential subject may have an unacceptable risk by participating in this study
- •Subject has a hypersensitivity to any of the excipients in ATB200, approved rhGAA, or AT2221
- •Female subject is pregnant or breast-feeding at screening
- •Subject requires the use of ventilation support for > 6 hours per day while awake
- •Subject has evidence of moderate to severe hypertrophic cardiomyopathy aligning with classic IOPD
- •In the opinion of the investigator, the parent or legally authorized representative is unlikely or unable to comply with the study requirements
- •Subject has any prior history of illness or condition known to affect motor function, such as, but not limited to, Guillain-Barre syndrome, cerebral palsy, etc
- •Subject who is diagnosed with Pompe disease via newborn screening and is asymptomatic (ie, showing no signs and symptoms of Pompe disease (Cohort 2 Only)
研究组 & 干预措施
Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221)
Experimental
Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsule
干预措施: Cipaglucosidase Alfa (Biological)
Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221)
Experimental
Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsule
干预措施: Miglustat (Drug)
结局指标
主要结局
Incidence of treatment-emergent adverse events (TEAEs) from baseline
时间窗: 52 weeks
次要结局
- Assessment of pharmacokinetic parameters(52 weeks)
研究者
研究点 (34)
Loading locations...
相似试验
招募中
3 期
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18Glycogen Storage Disease Type II Infantile OnsetNCT04808505Amicus Therapeutics36
已完成
3 期
Study Evaluating 13-valent Pneumococcal Conjugate Vaccine (13vPnC) in Healthy Children Aged 15 Months to 17 YearsHealthy SubjectsNCT00761631Pfizer1,200
已完成
3 期
Study of Biostate® in Children With Von Willebrand DiseaseVon Willebrand DiseaseNCT01213446CSL Behring17
已完成
3 期
Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of AgeCystic FibrosisNCT05882357Vertex Pharmaceuticals Incorporated70
已完成
3 期
A Study of Ustekinumab or Guselkumab in Pediatric Participants With Active Juvenile Psoriatic ArthritisArthritis, JuvenileNCT05083182Janssen Research & Development, LLC48