Pediatric Vasculitis Initiative (PedVas)

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2014 by University of British Columbia
Sponsor:
Collaborators:
Canadian Institutes of Health Research (CIHR)
Simon Fraser University
Child and Family Research Institute
Alberta Children's Hospital
University Hospital Muenster
The Hospital for Sick Children
University of Oxford
Information provided by (Responsible Party):
University of British Columbia
ClinicalTrials.gov Identifier:
NCT02006134
First received: November 27, 2013
Last updated: February 5, 2014
Last verified: February 2014
  Purpose

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.


Condition
Wegeners Granulomatosis
Microscopic Polyangiitis
Churg Strauss Syndrome
Polyarteritis Nodosa
Takayasu Arteritis
Primary CNS Vasculitis
Unclassified Vasculitis

Study Type: Observational [Patient Registry]
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 2 Years
Official Title: Chronic Childhood Vasculitis: Characterizing the Individual Rare Diseases to Improve Patient Outcomes

Resource links provided by NLM:


Further study details as provided by University of British Columbia:

Primary Outcome Measures:
  • Develop new benchmarks for outcome in pediatric patients with systemic or CNS vasculitis [ Time Frame: within 3 yrs ] [ Designated as safety issue: No ]
    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 or PACNS who have had a minimum of 12 months follow up.


Biospecimen Retention:   Samples With DNA

Serum, plasma, biopsy tissue, urine, DNA


Estimated Enrollment: 1600
Study Start Date: January 2013
Estimated Study Completion Date: January 2017
Estimated Primary Completion Date: January 2017 (Final data collection date for primary outcome measure)
Groups/Cohorts
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.
ADULT VASCULITIS/ COHORT 1
Adult patients in this cohort are those at or near the time of diagnosis of GPA, MPA, EGPA or unclassified vasculitis that are participants in DCVAS. Clinical data and blood (RNA, DNA) will be collected at the time-of-diagnosis only.
ADULT VASCULITIS / COHORT 2
Adult patients in this cohort are those individuals that are participants in DCVAS and have any form of vasculitis. Clinical data and blood (DNA) collected at the time-of-diagnosis will be used for study.
HEALTHY CHILDREN / PEDIATRIC CONTROL
Participants in this cohort are otherwise healthy children with no history of inflammatory disease. Children will provide a one time donation of blood (RNA, serum) and urine.
HEALTHY ADULTS / ADULT CONTROL
Participants in this cohort are otherwise healthy adults with no history of inflammatory disease. Adults will provide a one time donation urine and will provide blood (RNA, serum) as many as 4 times.

Detailed Description:

Over a 3-year period, 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.

Healthy volunteers from the community will be recruited to participate in this study by word of mouth and recruitment posters. Participation for children involves a one-time donation of blood and a urine sample, while adults may donate blood and urine up to 4 times over the course of 18 months.

All biological samples will be processed and analyzed in Vancouver at the Child and Family 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. All CNS vasculitis data will be entered into the Brainworks database which is managed by the data management team at the Hospital for Sick Kids in Toronto.

  Eligibility

Ages Eligible for Study:   up to 20 Years
Genders Eligible for Study:   Both
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

Criteria

Inclusion criteria for vasculitis subjects:

  • Diagnosed with ANCA-associated vasculitis (AAV: such as Granulomatosis with Polyangiitis (GPA) 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02006134

Contacts
Contact: Angelyne Sarmiento pedvas@cw.bc.ca

  Show 22 Study Locations
Sponsors and Collaborators
University of British Columbia
Canadian Institutes of Health Research (CIHR)
Simon Fraser University
Child and Family Research Institute
Alberta Children's Hospital
University Hospital Muenster
The Hospital for Sick Children
University of Oxford
Investigators
Principal Investigator: David Cabral, MBBS University of British Columbia; BC Children's Hospital
Principal Investigator: Susanne Benseler, MD, PhD Alberta 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
Principal Investigator: Jinko Graham, PhD Simon Fraser University
  More Information

Additional Information:
No publications provided

Responsible Party: University of British Columbia
ClinicalTrials.gov Identifier: NCT02006134     History of Changes
Other Study ID Numbers: H12-00894, TR2-119188
Study First Received: November 27, 2013
Last Updated: February 5, 2014
Health Authority: Canada: Health Canada

Keywords provided by University of British Columbia:
Wegener Granulomatosis
Granulomatosis with polyangiitis
Vasculitis
Primary CNS Vasculitis
Systemic vasculitis
Churg-Strauss Syndrome
Polyarteritis Nodosa
Pediatric vasculitis
Childhood vasculitis
Microscopic Polyangiitis
Takayasu Arteritis
Autoimmune

Additional relevant MeSH terms:
Wegener Granulomatosis
Arteritis
Churg-Strauss Syndrome
Polyarteritis Nodosa
Takayasu Arteritis
Aortic Arch Syndromes
Vasculitis
Systemic Vasculitis
Microscopic Polyangiitis
Vasculitis, Central Nervous System
Vascular Diseases
Cardiovascular Diseases
Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
Granuloma
Lymphoproliferative Disorders
Lymphatic Diseases
Autoimmune Diseases
Immune System Diseases
Skin Diseases, Vascular
Skin Diseases
Aortic Diseases
Lung Diseases, Interstitial
Lung Diseases
Respiratory Tract Diseases
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Cerebrovascular Disorders
Brain Diseases
Central Nervous System Diseases

ClinicalTrials.gov processed this record on July 24, 2014