<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT03032835</url>
  </required_header>
  <id_info>
    <org_study_id>2016P002690</org_study_id>
    <nct_id>NCT03032835</nct_id>
  </id_info>
  <brief_title>Partners Calciphylaxis Biobank</brief_title>
  <acronym>PCB</acronym>
  <official_title>Partners Calciphylaxis Biobank and Patient Registry</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Massachusetts General Hospital</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Massachusetts General Hospital</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      Calciphylaxis, a vascular calcification disorder, is a rare and serious disorder&#xD;
      characterized by calcification of dermal arterioles.&#xD;
&#xD;
      There are significant gaps in the understanding of the pathophysiology and risk factors for&#xD;
      calciphylaxis. At present, there is no effective treatment. Uncertain pathobiology, rare&#xD;
      incidence and lack of collaborative approach have been some of the major limiting factors&#xD;
      towards treating calciphylaxis.&#xD;
&#xD;
      The Partners Calciphylaxis Biorepository (PCB) aims to address these gaps within&#xD;
      calciphylaxis research by utilizing existing and, when necessary, developing new&#xD;
      infrastructure to support the consent of patients and the collection of dedicated samples for&#xD;
      a calciphylaxis repository.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Calciphylaxis, a vascular calcification disorder, is a rare and serious disorder&#xD;
      characterized by calcification of dermal arterioles.&#xD;
&#xD;
      There are significant gaps in the understanding of the pathophysiology and risk factors for&#xD;
      calciphylaxis. At present, there is no effective treatment. Uncertain pathobiology, rare&#xD;
      incidence and lack of collaborative approach have been some of the major limiting factors&#xD;
      towards treating calciphylaxis.&#xD;
&#xD;
      The Partners Calciphylaxis Biorepository (PCB) aims to address these gaps within&#xD;
      calciphylaxis research by utilizing existing and, when necessary, developing new&#xD;
      infrastructure to support the consent of patients and the collection of dedicated samples for&#xD;
      a calciphylaxis repository.&#xD;
&#xD;
      This repository is working in collaboration with the Partners Biobank as they will still be&#xD;
      receiving samples from the PCB and storing them and distributing them according to their own&#xD;
      procedures.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date>January 2017</start_date>
  <completion_date type="Anticipated">December 2030</completion_date>
  <primary_completion_date type="Anticipated">December 2030</primary_completion_date>
  <study_type>Observational [Patient Registry]</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <target_duration>20 Years</target_duration>
  <primary_outcome>
    <measure>Number of patients with improvement in calciphylaxis skin lesions</measure>
    <time_frame>6 months</time_frame>
    <description>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.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Number of patients with &gt;30% improvement in pain severity</measure>
    <time_frame>4weeks</time_frame>
    <description>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.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Time to death in patients with calciphylaxis</measure>
    <time_frame>Up to 5 years</time_frame>
    <description>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.</description>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Anticipated">300</enrollment>
  <condition>Calciphylaxis</condition>
  <condition>Calcific Uremic Arteriolopathy</condition>
  <condition>End Stage Renal Disease</condition>
  <condition>Chronic Kidney Diseases</condition>
  <arm_group>
    <arm_group_label>Study participants</arm_group_label>
    <description>No intervention</description>
  </arm_group>
  <biospec_retention>Samples With DNA</biospec_retention>
  <biospec_descr>
    <textblock>
      Plasma, serum, DNA and tissue samples (e.g. skin)&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        Patients with skin lesions consistent with calciphylaxis&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Male or female ≥18 years old&#xD;
&#xD;
          2. Skin lesions consistent with calciphylaxis diagnosis as determined by the treating&#xD;
             clinician&#xD;
&#xD;
          3. Informed of the investigational nature of the study and sign written Informed consent&#xD;
             OR are eligible for surrogate consent process based on impaired decision making&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Patients &lt;18 years of age&#xD;
&#xD;
          2. Prisoners&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Sagar U Nigwekar, MD, MMSc</last_name>
    <role>Principal Investigator</role>
    <affiliation>Massachusetts General Hospital</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Sagar U Nigwekar, MD, MMSc</last_name>
    <phone>617-726-7872</phone>
    <email>snigwekar@mgh.harvard.edu</email>
  </overall_contact>
  <location>
    <facility>
      <name>Massachusetts General Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02114</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Sagar U Nigwekar, MD, MMSc</last_name>
      <phone>617-726-7872</phone>
      <email>snigwekar@mgh.harvard.edu</email>
    </contact>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>https://www.ncbi.nlm.nih.gov/pubmed?term=((Nigwekar)%20AND%20((Calciphylaxis)%20OR%20(Calcific%20uremic%20arteriolopathy)))</url>
    <description>Link to publications</description>
  </link>
  <reference>
    <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>
    <PMID>28049648</PMID>
  </reference>
  <reference>
    <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>
    <PMID>27080977</PMID>
  </reference>
  <reference>
    <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.</citation>
    <PMID>25960299</PMID>
  </reference>
  <verification_date>August 2022</verification_date>
  <study_first_submitted>January 23, 2017</study_first_submitted>
  <study_first_submitted_qc>January 25, 2017</study_first_submitted_qc>
  <study_first_posted type="Estimate">January 26, 2017</study_first_posted>
  <last_update_submitted>August 22, 2022</last_update_submitted>
  <last_update_submitted_qc>August 22, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">August 24, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Massachusetts General Hospital</investigator_affiliation>
    <investigator_full_name>Sagar U. Nigwekar, MD, MMSc</investigator_full_name>
    <investigator_title>Assistant in Medicine</investigator_title>
  </responsible_party>
  <keyword>Calciphylaxis</keyword>
  <keyword>Warfarin</keyword>
  <keyword>Dialysis</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Kidney Diseases</mesh_term>
    <mesh_term>Renal Insufficiency, Chronic</mesh_term>
    <mesh_term>Kidney Failure, Chronic</mesh_term>
    <mesh_term>Calciphylaxis</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT03032835
org study id: 2016P002690
nct id: NCT03032835
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
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.
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.
observational model: Cohort
time perspective: Prospective
measure: Number of patients with improvement in calciphylaxis skin lesions
time frame: 6 months
description: 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.
measure: Number of patients with >30% improvement in pain severity
time frame: 4weeks
description: 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.
measure: Time to death in patients with calciphylaxis
time frame: Up to 5 years
description: 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.
arm group label: Study participants
description: No intervention
Plasma, serum, DNA and tissue samples (e.g. skin)
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Sagar U Nigwekar, MD, MMSc
role: Principal Investigator
affiliation: Massachusetts General Hospital
last name: Sagar U Nigwekar, MD, MMSc
phone: 617-726-7872
email: snigwekar@mgh.harvard.edu
facility: 
status: Recruiting
contact: 
country: United States
url: https://www.ncbi.nlm.nih.gov/pubmed?term=((Nigwekar)%20AND%20((Calciphylaxis)%20OR%20(Calcific%20uremic%20arteriolopathy)))
description: Link to publications
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.
PMID: 28049648
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.
PMID: 27080977
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.
PMID: 25960299
responsible party type: Principal Investigator
investigator affiliation: Massachusetts General Hospital
investigator full name: Sagar U. Nigwekar, MD, MMSc
investigator title: Assistant in Medicine
mesh term: Kidney Diseases
mesh term: Renal Insufficiency, Chronic
mesh term: Kidney Failure, Chronic
mesh term: Calciphylaxis
sharing ipd: No