Clinical Trial: Partners Calciphylaxis Biobank

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational [Patient Registry]

Official Title: Partners Calciphylaxis Biobank and Patient Registry

Brief Summary:

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.


Detailed Summary:

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.


Sponsor: Massachusetts General Hospital

Current Primary Outcome: Number of patients with improvement in calciphylaxis skin lesions [ Time Frame: 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.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Number of patients with >30% improvement in pain severity [ Time Frame: 4weeks ]
    Pain severity will be assessed by investigators at baseline (entry into study) and at 4 weeks to determine whether there is improvement. Pain is measured using Wong-Baker Faces pain rating scale. The investigators will examine clinical, laboratory and genetic predictors of this outcome in calciphylaxis patients.
  • Time to death in patients with calciphylaxis [ Time Frame: Up to 5 years ]
    Time to death is defined as time from the diagnosis of calciphylaxis to death (or to the end of follow up). The investigators will examine clinical, laboratory and genetic predictors of this outcome in calciphylaxis patients.


Original Secondary Outcome: Same as current

Information By: Massachusetts General Hospital

Dates:
Date Received: January 23, 2017
Date Started: January 2017
Date Completion: December 2030
Last Updated: January 25, 2017
Last Verified: January 2017