NCT03547297终止不适用
INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHP
Alnylam Pharmaceuticals1 个研究点 分布在 1 个国家目标入组 212 人开始时间: 2018年5月29日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- 入组人数
- 212
- 试验地点
- 1
- 主要终点
- Proportion of participants with current or past clinically symptomatic AHP with urinary aminolevulinic acid (ALA) or porphobilinogen (PBG) values above the upper limit of normal (ULN)
研究概览
简要总结
This study will use specific diagnostic tests on a group of patients who are experiencing symptoms typical of acute hepatic porphyria (AHP) to determine how many have the condition, and to potentially help improve the diagnostic process for patients in the future.
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
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •≥ 16 years of age
- •Recurrent and severe episodes of abdominal pain that last for at least 24 hours
- •At least 1 episode of severe abdominal pain that required an urgent healthcare visit in the past 12 months
- •Other symptoms related to AHP that your doctor will discuss with you
- •Willing and able to comply with protocol required assessments and provide written informed consent
排除标准
- •Known diagnosis of AHP
- •Alternative explanation for abdominal pain with clinical response to treatment specific for alternative diagnosis
结局指标
主要结局
Proportion of participants with current or past clinically symptomatic AHP with urinary aminolevulinic acid (ALA) or porphobilinogen (PBG) values above the upper limit of normal (ULN)
时间窗: 12 months
次要结局
- Proportion of participants with AHPs that contain likely pathogenic mutation(s) in the presence of urinary ALA or PBG values above the ULN(12 months)
- Proportion of patients with variants of unknown significance in the presence of urinary ALA and PBG values above the ULN(12 months)
- Correlation of biochemical and genetic test results with severity of disease measured by symptom frequency and severity in the participant questionnaire(12 months)
- Correlation of biochemical and genetic test results with severity of disease measured by healthcare utilization in the participant questionnaire(12 months)
- Proportion of patients with pathogenic mutation(s) in the presence of urinary ALA and PBG values above the ULN(12 months)
- Proportion of patients with negative genetic tests in the presence of urinary ALA or PBG values above the ULN(12 months)
研究者
研究点 (1)
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