NCT04348708Enrolling By Invitation不适用
An Open-Label, Long-Term Follow Up Study of Safety and Efficacy in PKU Subjects With PAH Deficiency Previously Administered HMI 102
适应症
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 21
- 试验地点
- 2
- 主要终点
- Incidence and severity of adverse events of special interest (AESIs) and serious adverse events (SAEs) related to HMI 102
研究概览
简要总结
An Open-Label, Long-Term Follow Up Study of Safety and Efficacy in PKU Subjects with PAH Deficiency Previously Administered HMI 102
详细描述
This Long-Term Follow Up Study will evaluate the safety and efficacy of PKU Subjects with PAH Deficiency Previously Administered HMI 102. Subjects will have already received a single dose of HMI-102 administered intravenously
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 55 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject was previously administered HMI
- •Subject is able to understand the purpose and risks of the study and is willing to provide informed consent.
- •Subject is able to comply with all study procedures and long-term follow-up.
排除标准
- •Participation in the study is not in the subject's best interest, in the opinion of the Investigator.
结局指标
主要结局
Incidence and severity of adverse events of special interest (AESIs) and serious adverse events (SAEs) related to HMI 102
时间窗: Baseline to year 5
Subjects with at least one AESI or SAE
次要结局
- Protein intake(Baseline to year 5)
- Plasma Phe Concentration(Baseline to year 5)
- Phe-restricted diet(Baseline to year 5)
- Phenylketonuria Quality of Life Questionnaire (PKU-QOL(Baseline to year 5)
研究者
研究点 (2)
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