Partners Calciphylaxis Biobank and Patient Registry
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 300
- 试验地点
- 2
- 主要终点
- Number of patients with improvement in calciphylaxis skin lesions
研究概览
简要总结
Calciphylaxis, a vascular calcification disorder, is a rare and serious disorder characterized by calcification of dermal arterioles.
There are significant gaps in the understanding of the pathophysiology and risk factors for calciphylaxis. At present, there is no effective treatment. Uncertain pathobiology, rare incidence and lack of collaborative approach have been some of the major limiting factors towards treating calciphylaxis.
The Partners Calciphylaxis Biorepository (PCB) aims to address these gaps within calciphylaxis research by utilizing existing and, when necessary, developing new infrastructure to support the consent of patients and the collection of dedicated samples for a calciphylaxis repository.
详细描述
Calciphylaxis, a vascular calcification disorder, is a rare and serious disorder characterized by calcification of dermal arterioles.
There are significant gaps in the understanding of the pathophysiology and risk factors for calciphylaxis. At present, there is no effective treatment. Uncertain pathobiology, rare incidence and lack of collaborative approach have been some of the major limiting factors towards treating calciphylaxis.
The Partners Calciphylaxis Biorepository (PCB) aims to address these gaps within calciphylaxis research by utilizing existing and, when necessary, developing new infrastructure to support the consent of patients and the collection of dedicated samples for a calciphylaxis repository.
This repository is working in collaboration with the Partners Biobank as they will still be receiving samples from the PCB and storing them and distributing them according to their own procedures.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female ≥18 years old
- •Skin lesions consistent with calciphylaxis diagnosis as determined by the treating clinician
- •Informed of the investigational nature of the study and sign written Informed consent OR are eligible for surrogate consent process based on impaired decision making
排除标准
- •Patients <18 years of age
研究组 & 干预措施
Study participants
No intervention
结局指标
主要结局
Number of patients with improvement in calciphylaxis skin lesions
时间窗: 6 months
Skin lesions will be assessed by investigators at baseline (entry into study) and at 6 months to determine whether there is improvement. The investigators will determine clinical, laboratory and genetic predictors of this outcome in calciphylaxis patients.
次要结局
- Number of patients with >30% improvement in pain severity(4weeks)
- Time to death in patients with calciphylaxis(Up to 5 years)
研究者
Sagar U. Nigwekar, MD, MMSc
Assistant in Medicine
Massachusetts General Hospital