NCT00884949已完成1 期
A Phase 1/2, Multicenter, Open-label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 20
- 主要终点
- Subject Incidence of Treatment Emergent AEs
研究概览
简要总结
This multicenter, open-label study is designed to assess safety, dose-response using pharmacokinetic (PK) and pharmacodynamic (PD) measures, and clinical efficacy of BMN 110 in subjects between 5 and 18 years of age, diagnosed with Mucopolysaccharidosis IVA (MPS IVA).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 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
- •Documented history of reduced GALNS activity relative to the normal range of the laboratory performing the assay, or documented result of molecular genetic testing confirming diagnosis of MPS IVA.
- •Willing and able to provide written, signed informed consent, or in the case of subjects under the age of 16 years, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
- •Between 5 and 18 years of age, inclusive.
- •Sexually active subjects must be willing to use an acceptable method of contraception while participating in the study.
- •Females of childbearing potential must have a negative pregnancy test at Screening and be willing to have additional pregnancy tests during the study.
- •Willing to perform all study procedures as physically possible.
排除标准
- •Previous hematopoietic stem cell transplant (HSCT).
- •Has known hypersensitivity to BMN 110 or its excipients.
- •Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study.
- •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 that would interfere with study participation or safety, including, but not limited to, symptomatic cervical spine instability.
- •Any condition that, in the view of the Principal Investigator (PI), 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
Experimental
Within-patient Dose-Escalation
干预措施: BMN 110 (Drug)
结局指标
主要结局
Subject Incidence of Treatment Emergent AEs
时间窗: Entire Study, through week 84
The primary objective of the study was to evaluate the safety of weekly infusions of BMN 110 administered in escalating doses to subjects with MPS IVA. The safety variable incidence of TEAE is summarized.
次要结局
- Change From Baseline in 6MWT(Baseline to Weeks 12, 24, 36, 48, 72)
- Change From Baseline in 3MSCT(Baseline to Weeks 12, 24, 36, 48, 72)
- Percent Change From Baseline in uKS(Baseline to Weeks 12, 24, 36, 72)
- Percent Change From Baseline in MVV(Baseline to Weeks 12, 24, 36, 72)
- Percent Change From Baseline in FVC(Baseline to Weeks 12, 24, 36, 72)
研究者
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