Pediatric Vasculitis Initiative (PedVas)

November 27, 2023 updated by: David Cabral, University of British Columbia

Chronic Childhood Vasculitis: Characterizing the Individual Rare Diseases to Improve Patient Outcomes

Childhood chronic vasculitis describes a group of rare life-threatening diseases that have in common inflammation of blood vessels in vital organs such as kidneys, lungs and brain. Most knowledge about them comes from adult patients. Severe disease requires aggressive life-saving treatments with steroids and some cancer drugs which can themselves cause damage, and increase risks of cancer and severe infections. Conversely, milder disease can be treated with less toxic drugs. Different classification and "scoring tools" are used to define the types and severity of vasculitis and to measure damage caused by disease or drugs. These in turn help direct how aggressively to treat a patient and to measure outcome. None of these tools however have been assessed in children and the best balance of disease and treatment risks against outcome for children is not known. Although causes of these diseases in children and adults are probably the same, the effects of the disease and the response (good and bad) to drugs will differ in growing children. Because specialists may see only one new child with vasculitis each year, obtaining enough information to learn about childhood vasculitis requires cooperation. We will use an international web-based registry to which doctors from 50 or more centers can contribute patient data. We will determine the features which help better classify and diagnose children compared to adults. Through the web we will collect and analyze information on patients similarly classified and "scored" so that most successful treatments can be identified. Children with vasculitis are less likely to have diseases associated with aging, alcohol and smoking etc., and therefore may be a better group in whom to study the underlying biology of vasculitis. We will use this opportunity and collect spit, blood and tissue from registry patients for laboratory study with an aim to find biomarkers to better classify, define and direct optimal treatment and outcomes.

Study Overview

Status

Recruiting

Conditions

Detailed Description

We anticipate enrollment and collection of clinical data from as many as 600 children with various forms of childhood vasculitis, with approximately one third (200) of those children also contributing biological samples for study.

For children with vasculitis who are enrolled in the study, clinical information will be obtained from the medical chart from the time of diagnosis, post-induction (3-6 months post diagnosis) visit, 12-month clinic visit, and their most recent clinic visit or last clinic visit before discharge to adult care (ie. final outcome visit). Information that will be collected includes laboratory test results, biopsy and imaging results, disease activity, clinical history, and medications. Blood, urine, and saliva samples will also be collected at each clinic visit. If the subject experiences a disease flare, clinical data and biological samples will be collected at the time of the flare and at a later date when the disease remits.

The PedVas study is linked to an adult vasculitis initiative called DCVAS: Diagnosis and Classification Criteria in Vasculitis. Our DCVAS co-investigators and collaborators will recruit up to 250 adults at or near the time of diagnosis of the following forms of vasculitis: GPA, MPA, EGPA, TA, and UCV. Clinical data will be collected as part of the DCVAS study; this includes information such as laboratory test results, disease activity, and clinical history. Blood will also be collected and analyzed in parallel with samples collected from children with vasculitis. Finally, a DNA-biobank will be created and will house samples from approximately 700 adults and representing all forms of vasculitis. Recruitment will proceed according to DCVAS approved protocols and it will be conducted at participating DCVAS centres after the patient has formally consented to participation in the DCVAS study.

All biological samples will be processed and analyzed in Vancouver at the BC Children's Hospital Research Institute and at the University of British Columbia. Detailed data will be collected in electronic format and include demographic variables, socioeconomic status, detailed clinical history & physical findings, anthropometric measures, and measures of disease activity. All data for systemic vasculitis patients will be directly entered at each site into a secure, online, web-based data entry system called REDCap which is managed through the data management centre at the University of British Columbia in Vancouver.

Study Type

Observational

Enrollment (Estimated)

1600

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Canada
    • Alberta
      • Calgary, Alberta, Canada
        • Recruiting
        • University of Calgary / Alberta Children's Hospital
        • Principal Investigator:
          • Susanne Benseler, MD, PhD
    • British Columbia
      • Vancouver, British Columbia, Canada
        • Recruiting
        • BC Children's Hospital
        • Principal Investigator:
          • David Cabral, MBBS
    • Newfoundland and Labrador
      • St. John's, Newfoundland and Labrador, Canada
        • Active, not recruiting
        • Janeway Childrens Health and Rehabilitation Centre
    • Nova Scotia
      • Halifax, Nova Scotia, Canada
        • Recruiting
        • IIWK Health Centre
        • Principal Investigator:
          • Adam Huber, MD
    • Ontario
      • London, Ontario, Canada
        • Recruiting
        • London Health Sciences Centre
        • Principal Investigator:
          • Roberta Berard
      • Ottawa, Ontario, Canada
        • Recruiting
        • Children's Hospital of Eastern Ontario
        • Principal Investigator:
          • Ciaran Duffy, MD
      • Toronto, Ontario, Canada
        • Completed
        • Hospital for Sick Children
    • Saskatchewan
      • Saskatoon, Saskatchewan, Canada
        • Completed
        • Royal University Hospital
  • Denmark
      • Copenhagen, Denmark
        • Completed
        • Rigshospitalet
  • Germany
      • Munster, Germany
        • Active, not recruiting
        • University Children's Hospital
  • India
      • Lucknow, India
        • Recruiting
        • Sanjay Gandhi Post Graduate Institute
        • Principal Investigator:
          • Amita Aggarwal, MD
  • Thailand
      • Bangkok, Thailand
        • Recruiting
        • Siriraj Hospital
        • Principal Investigator:
          • Sirirat Charuvanij, MD
  • United Kingdom
      • Birmingham, United Kingdom
        • Recruiting
        • Birmingham Children's Hospital NHS Foundation Trust
        • Contact:
        • Principal Investigator:
          • Taunton Southwood
      • Glasgow, United Kingdom
      • Leeds, United Kingdom
      • Liverpool, United Kingdom
        • Recruiting
        • Alder Hey Children's Hospital
        • Contact:
        • Principal Investigator:
          • Eileen Baildam
      • Manchester, United Kingdom
        • Recruiting
        • Royal Manchester Children's Hospital
        • Contact:
        • Principal Investigator:
          • Janet McDonagh
      • Newcastle upon Tyne, United Kingdom
        • Recruiting
        • Great North Children's Hospital
        • Contact:
        • Principal Investigator:
          • Mark Friswell
      • Nottingham, United Kingdom
        • Recruiting
        • Nottingham Children's Hospital
        • Principal Investigator:
          • Lampros Fotis
      • Oxford, United Kingdom
        • Recruiting
        • Nuffield Orthopaedic Centre
        • Contact:
        • Principal Investigator:
          • Raashid Luqmani, DM FRCP, FRCP(E)
      • Sheffield, United Kingdom
        • Recruiting
        • Sheffield Children's Foundation Trust
        • Contact:
        • Principal Investigator:
          • Anne-Marie McMahon
      • Southampton, United Kingdom
  • United States
    • California
      • San Francisco, California, United States
        • Recruiting
        • University of San Francisco
        • Principal Investigator:
          • Susan Kim, MD
    • Florida
      • Gainesville, Florida, United States
        • Recruiting
        • University of Florida
        • Principal Investigator:
          • Melissa Elder, MD, PhD
    • Illinois
      • Chicago, Illinois, United States
        • Recruiting
        • Comer Children's Hospital
        • Principal Investigator:
          • Linda Wagner-Weiner
    • Indiana
      • Indianapolis, Indiana, United States
        • Recruiting
        • Riley Hospital for Children
        • Principal Investigator:
          • Stacey Tarvin
    • New Jersey
      • Hackensack, New Jersey, United States
        • Recruiting
        • The Joseph M. Sanzari Children's Hospital
        • Principal Investigator:
          • Suzanne Li, MD
    • New York
      • Bronx, New York, United States
        • Recruiting
        • Children's Hospital at Montefiore
        • Principal Investigator:
          • Dawn Wahezi, MD
    • Ohio
      • Akron, Ohio, United States
        • Recruiting
        • Akron Children's Hospital
        • Principal Investigator:
          • Mary Toth, MD
    • Texas
      • Houston, Texas, United States
        • Recruiting
        • Texas Children's Hospital
        • Principal Investigator:
          • Marietta DeGuzman, MD
        • Principal Investigator:
          • Alvaro Orjuela, MD
    • Utah
      • Salt Lake City, Utah, United States
        • Recruiting
        • University of Utah / Primary Children's Hospital
        • Principal Investigator:
          • Aimee Hersh, MD
    • Washington
      • Seattle, Washington, United States
        • Recruiting
        • Seattle Children's Hospital
        • Principal Investigator:
          • Susan Shenoi

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 20 years (Child, Adult)

Accepts Healthy Volunteers

Yes

Sampling Method

Non-Probability Sample

Study Population

Children or adolescents diagnosed with vasculitis at any of the participating PedVas study centres

Description

Inclusion criteria for vasculitis subjects:

  • Diagnosed with ANCA-associated vasculitis (AAV: such as Granulomatosis with Polyangiitis (GPA), Eosinophilic Granulomatosis with Polyangiitis (EGPA) and Microscopic Polyangiitis (MPA)), Primary Angiitis of the Central Nervous System (PACNS), Unclassified vasculitis, Takayasu's Arteritis (TA) or Polyarteritis Nodosa (PAN) before age 18

Inclusion criteria for healthy controls:

  • Healthy adult or child

Exclusion Criteria for vasculitis subjects:

  • Diagnosed with other vasculitis subtypes not listed above
  • More than 20 years of age

Exclusion criteria for healthy controls:

  • Donated greater than 20 ml of blood in the previous three weeks
  • Has an immune disorder or blood borne infectious diseases (such as HIV or Hepatitis)
  • Has vasculitis, multiple sclerosis, diabetes, an autoimmune disease, a thyroid condition, or other chronic conditions involving the heart, lungs, gut or kidney
  • Has a previous history of anaemia or abnormal blood clotting
  • Has a current or previous drug abuse problem

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
PEDIATRIC VASCULITIS/PROSPECTIVE
Pediatric patients in this cohort are those diagnosed with vasculitis within 12 months from study entry. Clinical data, blood (RNA, plasma, serum), urine, and saliva (DNA) will be collected at 3 to 5 timepoints: time-of-diagnosis, post-induction, 12-month post diagnosis, disease flare, and remission/post-flare.
PEDIATRIC VASCULITIS/RETROSPECTIVE
Patients in this cohort are those diagnosed with vasculitis more than 12 months from study entry and/or were previously enrolled in the ARChiVe or Brainworks registries. Clinical outcome data will be collected retrospectively. Blood (RNA & serum), urine, and saliva (DNA) will be collected at 2 timepoints: disease flare, and remission/post-flare.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Develop new benchmarks for outcome in pediatric patients with systemic vasculitis
Time Frame: within 3 yrs
Specific and generic disease assessment tools will be used to analyze our registry cohorts to enable the first-ever benchmarks of outcome in children with GPA who have had a minimum of 12 months follow up.
within 3 yrs

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of British Columbia

Collaborators

University of Oxford
BC Childrens Hospital Research Institute

Investigators

  • Principal Investigator: David Cabral, MBBS, University of British Columbia; BC Children's Hospital
  • Principal Investigator: Raashid Luqmani, DM FRCP(E), University of Oxford
  • Principal Investigator: Dirk Foell, MD, University of Muenster
  • Principal Investigator: Robert Hancock, PhD, University of British Columbia
  • Principal Investigator: Colin Ross, PhD, University of British Columbia

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2013

Primary Completion (Estimated)

March 1, 2025

Study Completion (Estimated)

March 1, 2025

Study Registration Dates

First Submitted

November 27, 2013

First Submitted That Met QC Criteria

December 4, 2013

First Posted (Estimated)

December 10, 2013

Study Record Updates

Last Update Posted (Actual)

November 30, 2023

Last Update Submitted That Met QC Criteria

November 27, 2023

Last Verified

November 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • H12-00894
  • TR2-119188 (Other Grant/Funding Number: Canadian Institutes of Health Research)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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