A Phase 3 Open-Label Study of Safety of Weekly Subcutaneous Pegzilarginase in Subjects With Arginase 1 Deficiency
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- 入组人数
- 3
- 试验地点
- 1
- 主要终点
- Treatment emergent adverse events
研究概览
简要总结
This is an open-label, multicenter study to evaluate the safety of weekly SC administration of pegzilarginase over 12 months in subjects with ARG1-D. The study consists of a screening period of up to 4 weeks, a subsequent 12-month treatment period, and a Safety Follow-Up Visit 2 weeks after the last treatment.
详细描述
Open-label, multicenter study to evaluate the safety of weekly SC administration of pegzilarginase over 12 months in subjects with ARG1-D. The study consists of a screening period of up to 4 weeks, a subsequent 12-month treatment period, and a Safety Follow-Up Visit 2 weeks after the last treatment.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Prior enrollment in pegzilarginase clinical studies, with completion of treatment or continued dosing as of the study termination date
- •Written informed consent by subject/parent/legal guardian, which includes compliance with the requirements and restrictions listed in the informed consent form and in this protocol
- •Negative pregnancy test within 7 days prior to dosing in childbearing subjects if they are sexually active
- •If the subject (male or female) is engaging in sexual activity that could lead to pregnancy, must be surgically sterile, post-menopausal (female), or must agree to use a highly effective method of birth control during the study and for a minimum of 30 days after the last study drug administration. Highly effective methods of contraception include combined (estrogen and progestogen-containing) hormonal contraception associated with inhibition of ovulation, progesterone-only hormonal contraception associated with inhibition of ovulation, intrauterine device, or intrauterine hormone-releasing system
排除标准
- •Subjects with acute or ongoing hyperammonemia within 6 weeks of the first dose
- •Subjects with medical conditions or underlying issues that, in the opinion of the Investigator, would preclude participation in the study
研究组 & 干预措施
Pegzilarginase
Weekly subcutaneous dosing of pegzilarginase plus individualized disease management for 52 weeks
干预措施: Pegzilarginase (Drug)
结局指标
主要结局
Treatment emergent adverse events
时间窗: Baseline through week 54
To evaluate the safety of long-term subcutaneous pegzilarginase in subjects with arginase 1 deficiency (ARG1-D)
次要结局
未报告次要终点