A Phase III, Single Arm, Cross-over, Multicenter Clinical Trial to Compare Efficacy and Safety of YW17(Laronidase; CinnaGen) Versus Laronidase (Aldurazyme®; Genzyme, BioMarin) in Patients With Mucopolysaccharidosis Type I (MPS I)"
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Cinnagen
- 入组人数
- 12
- 试验地点
- 4
- 主要终点
- mean uGAG
研究概览
简要总结
The purpose of this phase III study is to assess the efficacy and safety of YW17 produced by CinnaGen Company compared to Aldurazyme® in mucopolysaccharidosis type I (MPS I) patients.
All patients receive Aldurazyme® for 12 weeks, followed by YW17 for another 12 weeks.
The primary outcome is the assessment of the maintenance of the mean uGAG levels at the end of each medication administration. The secondary outcomes are the assessment of 6-minute walking test (6MWT), predicted forced vital capacity (FVC), enzyme activity assay, and adverse events (AEs).
详细描述
This is a phase III, single-sequence, cross-over study to assess the efficacy and safety of YW17 produced by CinnaGen Company in comparison with Aldurazyme® in MPS I patients.
All patients receive 0.58 mg/kg of Aldurazyme® for 12 weeks and then receive 0.58 mg/kg of YW17 for another 12 weeks.
Premedication with antipyretics and/or antihistamines is administered for all patients one hour before the infusion.
The primary outcome is to compare the mean uGAG levels at weeks 8, 10, and 12 (related to Aldurazyme®) with the mean uGAG levels at weeks 20, 22, and 24 (related to YW17).
The secondary outcomes, including 6MWT and FVC are assessed at the beginning and the end of each medication administration. Enzyme activity is assessed at the end of each medication administration. Safety assessments are performed during the study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 5 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with MPS I
- •Signing informed consent form
排除标准
- •Prior bone marrow transplantation or being a candidate for receiving haematopoietic stem cell transplantation (HSCT)
- •Prior tracheotomy
- •Being naïve to laronidase
- •Acute hydrocephalus
- •Abnormal renal function determined by measuring serum creatinine and blood urea nitrogen (BUN) levels
- •Any severe organic disease that is not associated with MPS I
- •Known hypersensitivity to laronidase or components of the laronidase solution
- •Presence of any medical condition or other circumstances that could significantly interfere with study compliance
- •Pregnancy and lactation
- •Administration of any investigational drug within 30 days before study enrollment
研究组 & 干预措施
YW17 (laronidase biosimilar)
YW17 (2.9 mg/5 mL) produced by CinnaGen Company, is administered 0.58 mg/kg weekly.
干预措施: Laronidase (Biological)
YW17 (laronidase biosimilar)
YW17 (2.9 mg/5 mL) produced by CinnaGen Company, is administered 0.58 mg/kg weekly.
干预措施: Antihistamine (Drug)
YW17 (laronidase biosimilar)
YW17 (2.9 mg/5 mL) produced by CinnaGen Company, is administered 0.58 mg/kg weekly.
干预措施: Antipyretic (Drug)
Aldurazyme®
Aldurazyme® (2.9 mg/5 mL), is administered 0.58 mg/kg weekly.
干预措施: Laronidase (Biological)
Aldurazyme®
Aldurazyme® (2.9 mg/5 mL), is administered 0.58 mg/kg weekly.
干预措施: Antihistamine (Drug)
Aldurazyme®
Aldurazyme® (2.9 mg/5 mL), is administered 0.58 mg/kg weekly.
干预措施: Antipyretic (Drug)
结局指标
主要结局
mean uGAG
时间窗: Baseline, weeks 8, 10, 12, 20, 22, and 24
urinary glycosaminoglycan
次要结局
- mean enzyme activity level(Weeks 11 and 23)
- mean 6MWT(Baseline, week 12, week 24)
- mean predicted FVC(Baseline, week 12, week 24)
- Number of participants with adverse events(During the study period (screening visit up to week 24))