NCT02597114已完成1 期
An Extension Study of AGT-181-102 Evaluating Safety and Glycosaminoglycans (GAGs) in Adult Patients With Hurler-Scheie or Scheie Syndrome Who Have Completed 8-Weeks of Dosing With AGT-181 in Study AGT-181-102
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- ArmaGen, Inc
- 入组人数
- 3
- 主要终点
- Number of participants 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 study is an extension of a safety and dose ranging study to obtain long term safety and exposure data, as well as information on the biological activity of the investigational drug
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
盲法说明
Open Label
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Must have completed clinical trial AGT-181-102
- •Voluntary written consent by patient or legally responsible representative
- •All women of childbearing potential and sexually mature males must be advised to use a medically accepted method of contraception throughout the study.
- •Negative pregnancy test (females)
排除标准
- •Refusal to complete screening evaluations.
- •Any medical condition or other circumstances that may significantly interfere with study compliance
- •Patient is pregnant or lactating
- •Clinically significant spinal cord compression, evidence of cervical instability.
- •Subject developed clinically relevant hypersensitivity to AGT-181
研究组 & 干预措施
AGT-181
Experimental
AGT-181 (fusion protein of anti-human insulin receptor monoclonal antibody and alpha-L-iduronidase) administered once weekly x 26 weeks at same dose level as subject received in core study
干预措施: AGT-181 (Drug)
结局指标
主要结局
Number of participants with adverse events as a measure of safety and tolerability
时间窗: 104 weeks (2 years)
次要结局
- changes in urinary or plasma glycosaminoglycans (GAGs)(104 weeks (2 years))
- change in spleen size(104 weeks (2 years))
- change in levels of heparan sulfate and dermatan sulfate in the cerebrospinal fluid(104 weeks (2 years))
- change in liver size(104 weeks (2 years))
研究者
相似试验
已完成
1 期
Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS IMucopolysaccharidosis INCT03071341ArmaGen, Inc19
已完成
1 期
Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MPS IMucopolysaccharidosis INCT03053089ArmaGen, Inc21
已完成
1 期
Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS IMucopolysaccharidosis INCT02371226ArmaGen, Inc3
进行中（未招募）
2 期
AG-120 in People With IDH1 Mutant ChondrosarcomaChondrosarcoma, Grade 2Chondrosarcoma, Grade 3IDH1 Gene MutationChondrosarcomaNCT04278781Memorial Sloan Kettering Cancer Center6
进行中（未招募）
2 期
Anti-GITR/Anti-PD1/Stereotactic Radiosurgery, in Recurrent GlioblastomaGlioblastomaNCT04225039University of Pennsylvania39