A Cohort Dose-Escalation Phase 1 Study of Intramuscular Injection of Uricase-PEG 20
试验速览
- 阶段
- 1 期
- 发起方
- 入组人数
- 28
- 试验地点
- 4
- 主要终点
- Safety (assessment of signs and symptoms and clinical laboratory measurements following administration of Uricase-PEG 20, graded according to the Common Toxicity Criteria for Rheumatology, version 2.0)
研究概览
简要总结
The purpose of this study is to assess the safety, pharmacokinetics and pharmacodynamics of single and multiple intramuscular doses of Uricase-PEG 20
详细描述
Uricase is an enzyme (found in most mammals but not humans) that converts poorly soluble uric acid into highly soluble allantoin. Because humans lack uricase, they are prone to develop elevated levels of uric acid, which can form crystals in the joints and soft tissues. In those which chronically elevated uric acid, gout may develop. In the setting of acute rises in uric acid, seen for example in tumor lysis syndrome, uric acid crystals can damage the renal tubules. Uricase-PEG 20 is a recombinant uricase conjugated with multiple PEG molecules designed to prolong the half-life and decrease the immunogenicity of uricase. This study will characterize the safety, pharmacokinetics and pharmacodynamics of intramuscular Uricase-PEG 20, the anticipated route of administration to be used in future clinical development in gout.
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
- 40 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Serum uric acid > 6 mg/dL (men) or > 5 mg/dL (women)
- •Clinical laboratory values within normal limits or not clinically significant
- •Women should be menopausal or peri-menopausal
排除标准
- •Prior exposure to uricase
- •History of severe allergic reactions, or any allergy to PEG or pegylated products
- •G6PD or catalase deficiency
- •Medical condition that may interfere with ability to complete the study (e.g., uncontrolled diabetes or hypertension, congestive heart failure NYHA Class III or IV, history of myocardial infarction, immunosuppression, pregnancy)
结局指标
主要结局
Safety (assessment of signs and symptoms and clinical laboratory measurements following administration of Uricase-PEG 20, graded according to the Common Toxicity Criteria for Rheumatology, version 2.0)
时间窗: Up to 35 days after dosing
次要结局
- Pharmacokinetics (Uricase-PEG 20 serum concentration)(Up to 35 days after dosing)
- Pharmacodynamics (plasma uric acid concentration)(Up to 35 days after dosing)
- Immunogenicity(Up to 35 days after dosing)