NCT00225615已完成3 期
A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
适应症
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 100
- 试验地点
- 14
- 主要终点
- Primary objective:
研究概览
简要总结
The primary objective of this trial is to evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 8 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •8 years of age and older
- •Prior successful participation in Study PKU-003
- •Willing and able to provide written informed consent or assent and written informed consent (if required) by a parent or legal guardian
- •For females of child-bearing potential only: Negative urine pregnancy test within 24 hours prior to enrollment. Women using acceptable birth control measures must agree to continue to use those measures while participating in the study
- •Willing and able to comply with study procedures
- •Willing to continue current diet unchanged while participating in the study
排除标准
- •Perceived to be unreliable or unavailable for study participation or, if under the age of 18, have parents or legal guardians who are perceived to be unreliable or unavailable
- •Withdrew from, or otherwise did not successfully complete, study PKU-003, except for subjects who were removed from the study because their blood Phe exceeded the alert level
- •Expected to require any investigational agent or vaccine prior to completion of all scheduled study assessments
- •Pregnant or breastfeeding, or planning pregnancy
- •Concurrent disease or condition that would interfere with study participation or safety (e.g., seizure disorder, oral steroid-dependent asthma or other condition requiring oral or parenteral corticosteroid administration, or insulin-dependent diabetes)
- •Requirement for concomitant treatment with any drug known to inhibit folate synthesis (e.g., methotrexate)
- •Concurrent use of levodopa
结局指标
主要结局
Primary objective:
- To evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.
次要结局
- Secondary objectives:
- To compare the safety and tolerability of three different doses of Phenoptin treatment in subjects with PKU.
- To determine the effect of various doses of Phenoptin on blood phenylalanine (Phe) levels.
- To evaluate the population pharmacokinetics of Phenoptin.
- To evaluate the ability of Phenoptin to reduce phenylalanine (Phe) levels over a 24-hour period.
- To evaluate the persistence of benefit of Phenoptin treatment in the subject population as evidenced by long-term control of blood Phe levels.
研究者
研究点 (14)
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