Urine Sample Collection From Patients With Fibrodysplasia Ossificans Progressiva (FOP) for Biomarker Analysis
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 25
- 试验地点
- 1
- 主要终点
- Analysis of pre-defined biomarker FGF2
研究概览
简要总结
The purpose of this study is to collect urine samples from patients with fibrodysplasia ossificans progressiva (FOP) for the assessment of biomarkers related to disease, disease progression and for prediction of flare-ups of the disease. Disease related biomarkers in these patients are currently unknown. This study aims to support the development of novel therapy/ies for this disease.
研究设计
- 研究类型
- Observational
入排标准
- 年龄范围
- 5 Years 至 35 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female patients ≥ 5 and ≤ 35 years of age with a diagnosis of FOP.
- •Physically able to provide first-morning urine sample of at least 30 mL
- •Able to communicate well with the investigator, to understand and comply with the requirements of the study
- •About half of the patients enrolled should have a flare-up, which is defined as an acute exacerbation of disease activity characterized by two or more of the following symptoms: pain, swelling, decreased range of motion, impaired function
排除标准
- •Diagnosis of diabetes
- •Diagnosis of other systemic inflammatory disorder (juvenile idiopathic arthritis, systemic lupus erythematosus, etc.)
- •Diagnosis of cancer other than nonmelanomatous skin cancer.
结局指标
主要结局
Analysis of pre-defined biomarker FGF2
时间窗: 3 months
The objective of this study is to collect urine samples from patients with FOP over a time period covering ideally quiescent disease status and progression through a flare-up for biomarker analysis
Analysis of pre-defined biomarker WEGF
时间窗: 3 months
The objective of this study is to collect urine samples from patients with FOP over a time period covering ideally quiescent disease status and progression through a flare-up for biomarker analysis
Analysis of microRNAs
时间窗: 3 months
The objective of this study is to collect urine samples from patients with FOP over a time period covering ideally quiescent disease status and progression through a flare-up for biomarker analysis
次要结局
未报告次要终点