NCT00778206已完成不适用
PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
适应症
干预措施
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 1,887
- 试验地点
- 45
- 主要终点
- Observational Data Only
研究概览
简要总结
The objective of this study is to evaluate the safety of long-term treatment with Kuvan.
详细描述
The PKUDOS program is a voluntary, multicenter, strictly observational program for patients with PKU who have either received Kuvan therapy, or currently receive Kuvan, or intend to begin receiving Kuvan therapy within 90 days of entering the registry.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient has confirmed diagnosis of PKU with hyperphenylalaninemia documented by a Phenylalanine level of greater than or equal to 360 umol/L (6 mg/dL)
- •Patient has previously received Kuvan
- •Patient is currently receiving Kuvan
- •Patient intends to receive Kuvan therapy within 90 days of enrollment into the registry
- •The Patient is being followed at a PKUDOS participating center
- •Willing and able to provide written authorization or, if under the age of 18 years, provide written assent (if required) and written patient authorization by a parent or legal guardian
- •Willing to provide personal health information
排除标准
- •Patients are not eligible to participate in PKUDOS if they are participating in a BioMarin-sponsored clinical study of Kuvan
- •Patients not previously treated with Kuvan and patients that are unwilling to begin Kuvan therapy within 90 days of entry into the registry
- •PKU MOMS Subregistry
- •Inclusion Criteria:
- •Willing to enroll in (or are already enrolled in) PKUDOS
- •Agree to follow the standard of care for pregnant women with PKU in the United States (NIH, 200, NIH Consensus Statement)
- •Agree to be followed by a hospital or PKU clinic offering the standard of care for maternal PKU
- •Are within 10 weeks of their last menstrual period
- •Exclusion Criteria:
- •Patients who have not adhered to the standard of care for pregnant women with PKU in the United States are not eligible to participate in PKU MOMS
研究组 & 干预措施
2. PKU MOMS Subregistry
Patients with PKU who are pregnant at enrollment in the registry or who become pregnant while participating in the registry.
干预措施: Kuvan (Drug)
结局指标
主要结局
Observational Data Only
时间窗: 15 years
Registry data including demographic and baseline characteristics
次要结局
未报告次要终点
研究者
研究点 (45)
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