Phase 2, Randomized, Double-blind, Active-controlled, Dose-ranging Study to Evaluate the Pharmacokinetics, Pharmacodynamics and Safety of Idursulfase-beta (GC1111) in Hunter Syndrome (Mucopolysaccharidosis II) Patients
试验速览
- 阶段
- 2 期
- 入组人数
- 20
- 主要终点
- Percent change from baseline in urinary GAG(Glycosaminoglycans) at Week 25
研究概览
简要总结
This study evaluates the efficacy and safety of three doses of GC1111 in patients with Hunter Syndrome. Participants will be randomized to one of three doses of GC1111 or comparator.
详细描述
This is a randomized, double-blind, active-controlled, dose-ranging study, where patient will receive one of the three doses of GC1111 (0.5 mg/kg, 1.0 mg/kg, and 1.5 mg/kg) or ELAPRASE 0.5 mg/kg. Approximately 20 patients will be administrated each study drug once every week as an iv infusion for 24 weeks. Efficacy of GC1111 will be evaluated in Six-Minute Walk Test (6MWT), urine Glycosaminoglycans(uGAG), liver and spleen volume, percent predicted Forced Vital Capacity(FVC), and cardiac size and function. Also immunogenicity, Pharmacokinetics(PK) and safety will be evaluated.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 5 Years 至 35 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male patients between 5 and 35 years of age
- •Informed consent form signed
- •Patients diagnosed with hunter syndrome
- •Previously untreated with an enzyme replacement therapy
排除标准
- •History of tracheostomy, bone marrow transplant, or cord blood transplant
- •Treatment with another investigational product within 30 days prior to the start of study drug
- •Known hypersensitivity of any of the ingredients of study drug
- •Patient with severe hunter syndrome who cannot perform 6MWT
- •Female patients
结局指标
主要结局
Percent change from baseline in urinary GAG(Glycosaminoglycans) at Week 25
时间窗: Baseline to Week 25
次要结局
- Safety changes from baseline in clinical laboratory tests, physical examination and vital signs(Baseline to Week 25)
- Immunogenicity(Baseline to Week 25)
- Pharmacokinetic profile - Maximum observed peak plasma concentration (Cmax)(1 and 17 week)
- Pharmacokinetic profile - Time at which Cmax is observed (Tmax)(1 and 17 week)
- Change from baseline in Six Minute Walk Test at Week 25(Baseline to Week 25)
- Incidence of Adverse Events and Serious Adverse Events(Baseline to Week 25)
- Percent change from baseline in Six Minute Walk Test at Week 25(Baseline to Week 25)
- Change from baseline in Liver volume at Week 25(Baseline to Week 25)
- Change from baseline in Spleen volume at Week 25(Baseline to Week 25)
- Pharmacokinetic profile - Area under the serum concentration time curve (AUClast)(1 and 17 week)
- Change from baseline in urinary GAG at Week 25(Baseline to Week 25)
- Percent change from baseline in Liver volume at Week 25(Baseline to Week 25)
- Percent change from baseline in Spleen volume at Week 25(Baseline to Week 25)