NCT02240784已完成不适用
A Natural History Study of Acute Hepatic Porphyria (AHP) Patients With Recurrent Attacks
Alnylam Pharmaceuticals1 个研究点 分布在 1 个国家目标入组 136 人开始时间: 2014年8月最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 136
- 试验地点
- 1
- 主要终点
- Medical history of AHP patients (Part A only)
研究概览
简要总结
The purpose of this study is to characterize the natural history and clinical management of Acute Hepatic Porphyria (AHP) patients with recurring attacks.
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
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males and females aged >/= 12 years
- •Diagnosis of AHP [acute intermittent porphyria (AIP), variegate porphyria (VP), hereditary coproporphyria (HCP) and aminolevulinic acid dehydratase (ALAD) deficient porphyria (ADP)]
- •Porphyria attacks in the past 12 months or receiving treatment to prevent attacks
- •Willing to provide written informed consent, medical records, and to comply with study requirements
排除标准
- •Current participation in a clinical trial of an investigational product
结局指标
主要结局
Medical history of AHP patients (Part A only)
时间窗: Baseline to 6-Month Follow-Up Visit
Medication usage of AHP patients (Part A only)
时间窗: Baseline to 6-Month Follow-Up Visit
次要结局
- Porphyria signs and symptoms (Part A only)(0, 2, 4, 6 months)
- Healthcare Utilization (Part A only)(0, 6 months)
- Plasma biomarkers (Part A only)(0, 6 months and During Attacks)
- Change in disease activity as measured by Quality of Life (Part B only)(0, 6, 12, 18, 24, 30, 36 months)
- Change in disease activity as measured by European Organization for Research and Treatment of Cancer (EORTC) Questionnaire (Part B only)(0, 6, 12, 18, 24, 30, 36 months)
- Quality of Life (Part A only)(0, 6 months)
- Change in disease activity as measured by Brief Pain Inventory form (Part B only)(0, 3, 6, 9, 12, 18, 24, 30, 36 months)
- Urine biomarkers (Part A only)(0, 6 months and During Attacks)
- Change in disease activity as measured by Porphyria Screening/Follow Up Questionnaire (Part B only)(0, 3, 6, 9, 12, 18, 24, 30, 36 months)
研究者
研究点 (1)
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