Study to Evaluate the Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU
赞助:
BioMarin Pharmaceutical
合作者:
信息的提供 (责任方):
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| 追踪信息 | |||
|---|---|---|---|
| 首次提交日期 ICMJE | September 4, 2018 | ||
| 首次发布日期e ICMJE | October 3, 2018 | ||
| 最后更新发布日期 | October 3, 2018 | ||
| 预计研究开始日期 ICMJE | September 13, 2018 | ||
| 预计主要完成日期 | December 31, 2019 (主要结果测量的最终数据收集日期) | ||
| 目前主要观察指标 ICMJE |
Incidence of treatment-emergent adverse events as assessed by CTCAE v5.0[ Time Frame: Week 0 - Week 61 ] Change in blood Phe concentration[ Time Frame: Week 0 - Week 61 ] |
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| 原始主要观察测量 ICMJE | 与当前相同 | ||
| 目前的二级观察 ICMJE |
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| 描述性信息 | |||
| 简略标题 ICMJE | Study to Evaluate the Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU | ||
| 正式标题 ICMJE | An Open-label Extension Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase (> 40 mg/Day Dose) in Adults With Phenylketonuria | ||
| 简要概况 | This is a Phase 3 open-label extension study enrolling approximately 40 adult patients with PKU who were previously treated with pegvaliase in Studies PAL-003 or 165-302. The study is designed to evaluate the long-term safety and efficacy of pegvaliase administered at doses > 40 mg/day to 60 mg/day |
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| 详细说明 | |||
| 研究类型 ICMJE | Interventional | ||
| 研究阶段 | Phase 3 | ||
| 研究设计 ICMJE | 分配: 干预模型: Single Group Assignment 干预模型描述: 盲法: Interventional 盲法描述: 主要目的: Treatment |
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| 适用条件 ICMJE | |||
| 干预项目 ICMJE |
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| 研究工具 |
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| 招募信息 | |||
| 招募状态 ICMJE | Active, not recruiting | ||
| 预计入组 ICMJE |
40 | ||
| 原始预计入组 ICMJE | 与当前相同 | ||
| 预计研究完成日期 | December 31, 2019 | ||
| 预计主要完成日期 | December 31, 2019 (主要结果测量的最终数据收集日期) | ||
| 合格标准 ICMJE | Inclusion criteria: - Must be enrolled in PAL-003 or 165-302 Part 4 at the time of screening for 165-304 and most recently receiving pegvaliase at a dose > 40 mg/day - Has identified a competent person or persons ≥ 18 years of age who can observe the subject during study drug administration and for a minimum of 1 hour following administration in situations required per protocol - For females with childbearing potential, must have negative pregnancy test at screening and be willing to have additional pregnancy tests during the study. - If sexually active and not planning to become pregnant (self or partner), must be willing to use 2 acceptable methods of contraception while participating in the study and for 4 weeks after the study. Exclusion criteria: - Use of any investigational product (except pegvaliase) or investigational medical device within 30 days prior to screening or requirement for any investigational agent prior to completion of all scheduled study assessments. - Use of any medication (except pegvaliase) intended to treat PKU, including the use of large neutral amino acids, within 2 days prior to the administration of pegvaliase (Day 1). - Use or planned use of any injectable drugs containing PEG (other than pegvaliase), including medroxyprogesterone injection, within 3 months prior to screening and during study participation. - Current participation in the Kuvan® registry study (PKU Demographics, Outcomes and Safety [PKUDOS]) - A history of organ transplantation or on chronic immunosuppressive therapy | ||
| 性别 |
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| 年龄 | 最小年龄：18 Years ，最大年龄：70 Years | ||
| 接受健康的志愿者 | 没有 | ||
| 可入组国家 ICMJE | United States | ||
| 管理信息 | 数据检测委员会 | ||
| 研究涉及美国FDA监管的产品 |
研究美国FDA监管的药品: Yes 研究涉及美国FDA监管的设备产品: No |
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| IPD 共享声明 |
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| 责任方 | ， | ||
| 研究赞助商 ICMJE | BioMarin Pharmaceutical | ||
| 合作者 ICMJE | |||
| 研究员 ICMJE |
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| PRS 账户 | |||
| 验证日期 | October 2018 | ||
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ICMJE 国际医学期刊编辑委员会和 世界卫生组织 ICTRP 要求的元素 |
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