Clinical trial NCT03032835: Partners Calciphylaxis Biobank
Official title: Partners Calciphylaxis Biobank and Patient Registry
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Calciphylaxis
Calcific Uremic Arteriolopathy
End Stage Renal Disease
Chronic Kidney Diseases
Keywords: Calciphylaxis
Warfarin
Dialysis
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
First posted: 2017-01-26
Enrollment: 300
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with skin lesions consistent with calciphylaxis

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.

Eligibility criteria: 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

Primary outcomes:
measure: Number of patients with improvement in calciphylaxis skin lesions
timeFrame: 6 months
Secondary outcomes:
measure: Number of patients with >30% improvement in pain severity
timeFrame: 4weeks
measure: Time to death in patients with calciphylaxis
timeFrame: Up to 5 years
Other outcomes:
Locations:
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
Study officials:
affiliation: Massachusetts General Hospital
name: Sagar U Nigwekar, MD, MMSc
role: PRINCIPAL_INVESTIGATOR
References:
citation: Nigwekar SU, Bloch DB, Nazarian RM, Vermeer C, Booth SL, Xu D, Thadhani RI, Malhotra R. Vitamin K-Dependent Carboxylation of Matrix Gla Protein Influences the Risk of Calciphylaxis. J Am Soc Nephrol. 2017 Jun;28(6):1717-1722. doi: 10.1681/ASN.2016060651. Epub 2017 Jan 3.
citation: Nigwekar SU, Zhao S, Wenger J, Hymes JL, Maddux FW, Thadhani RI, Chan KE. A Nationally Representative Study of Calcific Uremic Arteriolopathy Risk Factors. J Am Soc Nephrol. 2016 Nov;27(11):3421-3429. doi: 10.1681/ASN.2015091065. Epub 2016 Apr 14.
citation: Nigwekar SU, Kroshinsky D, Nazarian RM, Goverman J, Malhotra R, Jackson VA, Kamdar MM, Steele DJ, Thadhani RI. Calciphylaxis: risk factors, diagnosis, and treatment. Am J Kidney Dis. 2015 Jul;66(1):133-46. doi: 10.1053/j.ajkd.2015.01.034. Epub 2015 May 7.