NCT01515956已完成2 期
A Phase 2, Open-label, Multinational Clinical Study to Evaluate the Safety and Efficacy of BMN 110 in Pediatric Patients Less Than 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 15
- 主要终点
- To Evaluate Safety and Tolerability of Infusions of BMN 110 at a Dose of 2.0 mg/kg/Week Over a 52-week Period in MPS IVA Subjects Less Than 5 Years of Age at Time of First Study Drug Infusion
研究概览
简要总结
This open-label Phase 2 study will evaluate the safety and efficacy of weekly 2.0 mg/kg/wk infusions of BMN 110 in pediatric patients, less than 5 years of age at the time of administration of the first dose of study drug, diagnosed with MPS IVA (Morquio A Syndrome) for up to 208 weeks.
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
- — 至 5 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Less than 5 years of age at the time of the first study drug infusion
- •Documented clinical diagnosis of MPS IVA based on clinical signs and symptoms of MPS IVA and documented reduced fibroblast or leukocyte GALNS enzyme activity or genetic testing confirming diagnosis of MPS IVA
- •Written informed consent provided by parent or legally authorized representative after the nature of the study has been explained and prior to any research-related procedures.
排除标准
- •Previous hematopoietic stem cell transplant (HSCT).
- •Previous treatment with BMN
- •Known hypersensitivity to any of the components of BMN
- •Major surgery within 3 months prior to stuy entry or planned major surgery during the 52-week treatment period.
- •Use of any investigational product or investigational medical device within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments.
- •Concurrent disease or condition, including but not limited to symptomatic cervical spine instability, clinically significant spinal cord compression, or severe cardiac disease that would interfere with study participation or safety as determined by the Investigator.
- •Any condition that, in the view of the Investigator, places the subject at high risk of poor treatment compliance or of not completing the study.
研究组 & 干预措施
BMN 110 Weekly
Experimental
干预措施: BMN 110 (Drug)
结局指标
主要结局
To Evaluate Safety and Tolerability of Infusions of BMN 110 at a Dose of 2.0 mg/kg/Week Over a 52-week Period in MPS IVA Subjects Less Than 5 Years of Age at Time of First Study Drug Infusion
时间窗: 52 weeks
Number of Participants Experiencing Adverse Events
次要结局
- Change From Baseline in Normalized Growth Rate Z-Scores(Baseline to Week 52)
- Percent Change From Baseline to Week 52 in Urinary Keratan Sulfate Measures(Baseline to Week 52)
研究者
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