An Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Patients With Mucopolysaccharidosis I Who Were Previously Enrolled in Studies With AGT-181
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- ArmaGen, Inc
- 入组人数
- 19
- 试验地点
- 1
- 主要终点
- number of patients with adverse events as a measure of safety and tolerability
研究概览
简要总结
AGT-181 is a fusion protein containing alpha-L-iduronidase that is intended to deliver the enzyme peripherally and to the brain, when administered intravenously.
This is a long term safety and tolerability study of AGT-181 in patients with MPS I who completed the previous 26-week study, AGT-181-101. Information on the biological activity of the investigational drug will also be collected.
详细描述
AGT-181-101E is a safety and tolerability study of AGT-181 in patients with MPS I who completed the previous 26-week study, AGT-181-101. Information on the biological activity of the investigational drug will be collected as well as longitudinal safety information.
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
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •2 years of age or older (and less than 18)
- •Must have been previously enrolled in study AGT-181-101
- •Written consent and assent as required
- •Female patients must not be pregnant, willing to utilize appropriate birth control methods and undergo pregnancy testing during the study
排除标准
- •Refusal to complete all assessments
- •Pregnant or Lactating
- •Received investigational drug within 1 year prior to study enrollment
- •Medical condition or extenuating circumstance that, in the opinion of the investigator, may interfere with study compliance
- •Clinically significant spinal cord compression or evidence of cervical instability (i.e. expected to require intervention during study participation)
- •Has developed clinically relevant hypersensitivity/anaphylactoid reactions to AGT-181
研究组 & 干预措施
AGT-181
Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) fusion protein
干预措施: AGT-181 (Drug)
结局指标
主要结局
number of patients with adverse events as a measure of safety and tolerability
时间窗: 24 months
Incidence and prevalence of adverse events
次要结局
- Plasma heparan sulfate and dermatan sulfate(24 months)
- CSF heparan sulfate and dermatan sulfate(24 months)
- Total urinary glycosaminoglycans (GAGs)(24 months)
- Urinary heparan sulfate and dermatan sulfate(24 months)
- liver and/or spleen volume(24 months)