Partners Calciphylaxis Biobank

Recruiting

• Conditions: Calciphylaxis; Calcific Uremic Arteriolopathy; End Stage Renal Disease; Chronic Kidney Diseases

• Registration Number: NCT03032835
• Lead Sponsor: Massachusetts General Hospital

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 Description

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.

Study Timeline

• Study Start: 2017-01
• Study First Submitted: 2017-01-23
• Study First Submitted QC: 2017-01-25
• Study First Posted: 2017-01-26
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-18
• Last Update Posted: 2024-11-19
• Primary Completion: 2030-12
• Study Completion: 2030-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 300

Inclusion Criteria

1. Male or female ≥18 years old
2. Skin lesions consistent with calciphylaxis diagnosis as determined by the treating clinician
3. 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

Exclusion Criteria

1. Patients <18 years of age
2. Prisoners

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Number of patients with improvement in calciphylaxis skin lesions	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.

Secondary Outcome Measures

Name	Time	Method
Number of patients with >30% improvement in pain severity	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	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.

Trial Locations

Locations (1)

Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States