NCT05754450招募中3 期
A Phase 3, Open-Label, Extension Study to Assess the Long-term Safety and Efficacy of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II (LAD II)
AUG Therapeutics2 个研究点 分布在 1 个国家目标入组 2 人开始时间: 2023年4月10日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 2
- 试验地点
- 2
- 主要终点
- Incidence of treatment emergent adverse events reported during the study attributable to AVTX-803.
研究概览
简要总结
The primary objective of this extension study is to assess the long-term safety and efficacy of AVTX-803 in subjects with LAD II (SLC35C1-CDG).
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
- 6 Months 至 75 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject must have completed protocol AVTX-803-LAD-301
- •Subject has biochemically and genetically proven LAD II (SLC35C1-CDG)
排除标准
- •Subject has severe anemia defined as hemoglobin <8.0 g/dL (<4.9 mmol/L)
- •Subject has impaired renal function as defined by an eGFR <90 mL/min
- •Subject has known or suspected intolerance or hypersensitivity to fucose or any ingredients of the investigational product
- •In the investigator's opinion, subject has a history of failure to respond to fucose at adequate dosing
研究组 & 干预措施
AVTX-803
Experimental
AVTX-803 at a dose specified by the investigator up to 5 times a day not to exceed 1700 mg/kg/day
干预措施: AVTX-803 (Drug)
结局指标
主要结局
Incidence of treatment emergent adverse events reported during the study attributable to AVTX-803.
时间窗: Through study completion, an average of 1 year.
次要结局
- Clinician Global Impression of Severity (CGI-S)(Through study completion, an average of 1 year.)
- Goal Attainment Score (GAS)(Through study completion, an average of 1 year.)
- Change from Baseline in the individual parameters of the Nijmegen Pediatric Congenital Disorders of Glycosylation (CDG) Rating Scale (NPCRS)(Through study completion, an average of 1 year.)
- Sialyl-Lewis X antigen expression on leukocytes(At 6 months)
- Change from Baseline in the composite Nijmegen Pediatric Congenital Disorders of Glycosylation (CDG) Rating Scale (NPCRS)(Through study completion, an average of 1 year.)
研究者
研究点 (2)
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