An Open-label, Multicentre Study to Evaluate the Long-Term Safety, Tolerability and Efficacy of AEB1102 in Patients With Arginase 1 Deficiency
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 14
- 试验地点
- 12
- 主要终点
- Number of Participants With Treatment-related Adverse Events
研究概览
简要总结
The purpose of this study is to determine the safety, tolerability, and efficacy of long-term intravenous and subcutaneous administration of pegzilarginase for the treatment of pediatric and adult patients with ARG1-D who complete Study CAEB1102-101A.
详细描述
Purpose of this study is to determine the safety, tolerability, and efficacy of long-term intravenous and subcutaneous administration of pegzilarginase for the treatment of pediatric and adult patients with ARG1-D who complete Study CAEB1102-101A.
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
- •Complete treatment in Study CAEB1102-101A without experiencing any clinically significant adverse event or other unmanageable drug toxicity that would preclude continued dosing
- •Confirmation by the Investigator and the Sponsor determine that it is acceptable for the patient to continue dosing with AEB1102
- •If female and of child-bearing potential, has a negative serum pregnancy test within 7 days before enrollment
- •If sexually active (male or female), must be surgically sterile, post-menopausal (female), or must agree to use a physician-approved method of birth control during the study and for a minimum of 30 days after the last study drug administration
- •Patient or legal guardian is able and willing to provide written informed consent and where required assent, and to comply with all requirements of study participation (including all study procedures and continuation of prescribed diet without modification), prior to any screening procedures
排除标准
- •1. Clinically significant concurrent disease, serious intercurrent illness, or other extenuating circumstances
研究组 & 干预措施
pegzilarginase (AEB1102)
Each patient may receive pegzilarginase administered IV or SC for up to 4 years.
干预措施: AEB1102 (Drug)
结局指标
主要结局
Number of Participants With Treatment-related Adverse Events
时间窗: From start of study up to 262 weeks
Number of Participants With Anti-pegzilarginase and Anti-PEG Antibodies
时间窗: From start of study up to 262 weeks
Incidence of treatment-related adverse events
时间窗: up to 4 years
Incidence of treatment-related adverse events
次要结局
- Plasma Arginine Levels(From start of study up to 240 weeks)
- Plasma GC Levels (Argininic Acid)(From start of study up to 240 weeks)
- Plasma GC Levels (Guanidinoacetic Acid)(From start of study up to 240 weeks)
- Plasma GC Levels (α-keto-δ-guanidinovaleric Acid)(From start of study up to 240 weeks)
- Plasma GC Levels (α-N-acetylarginine)(From start of study up to 240 weeks)
- Six-Minute Walk Test (6MWT)(From start of study up to 262 weeks)
- Gross Motor Function Measure (GMFM) Part D(From start of study up to 262 weeks)
- Gross Motor Function Measure (GMFM) Part E(From start of study up to 262 weeks)
- Modified Ashworth Scale (MAS)(From start of study up to 262 weeks)
- Berg Balance Scale (BBS)(From start of study up to 262 weeks)
- Purdue Pegboard Test (Dominant Hand)(From start of study up to 262 weeks)
- Purdue Pegboard Test (Non-dominant Hand)(From start of study up to 262 weeks)
- Number of Participants With Hyperammonemic Episodes(From study start up to 262 weeks)
- Number of Participants With Seizures(From study start up to 262 weeks)
- Cmax Cmin(up to 4 years)