Pediatric Vasculitis Initiative

NCT02006134 | Unknown

• 2 other identifiers: H12-00894TR2-119188
• Study Type: observational
• Target: 1,600
• Locations: 7 countries
• Sites: 32

Brief Summary

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.

Conditions

Wegeners Granulomatosis (Granulomatosis With Polyangiitis); Microscopic Polyangiitis; Eosinophilic Granulomatosis With Polyangiitis; Polyarteritis Nodosa; Takayasu Arteritis; Primary CNS Vasculitis; Unclassified Vasculitis

Keywords

Vasculitis; Primary CNS Vasculitis; Systemic vasculitis; Churg-Strauss Syndrome; Polyarteritis Nodosa; Wegener Granulomatosis; Pediatric vasculitis; Childhood vasculitis; Granulomatosis with polyangiitis; Microscopic Polyangiitis; Takayasu Arteritis; Autoimmune; ANCA-associated vasculitis

Outcome Measures

Primary Outcomes (1)

• Develop new benchmarks for outcome in pediatric patients with systemic vasculitis

  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

Study Arms (2)

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.

Eligibility Criteria

• Age: Up to 20 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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
• Healthy adult or child

You may not qualify if:

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

Sponsors & Collaborators

• University of British Columbia: lead
• BC Childrens Hospital Research Institute: collaborator
• University of Oxford: collaborator

Study Sites (32)

University of San Francisco

San Francisco, California, United States

RECRUITING

University of Florida

Gainesville, Florida, United States

RECRUITING

Comer Children's Hospital

Chicago, Illinois, United States

RECRUITING

Riley Hospital for Children

Indianapolis, Indiana, United States

RECRUITING

The Joseph M. Sanzari Children's Hospital

Hackensack, New Jersey, United States

RECRUITING

Children's Hospital at Montefiore

The Bronx, New York, United States

RECRUITING

Akron Children's Hospital

Akron, Ohio, United States

RECRUITING

Texas Children's Hospital

Houston, Texas, United States

RECRUITING

University of Utah / Primary Children's Hospital

Salt Lake City, Utah, United States

RECRUITING

Seattle Children's Hospital

Seattle, Washington, United States

RECRUITING

University of Calgary / Alberta Children's Hospital

Calgary, Alberta, Canada

RECRUITING

BC Children's Hospital

Vancouver, British Columbia, Canada

RECRUITING

Janeway Childrens Health and Rehabilitation Centre

St. John's, Newfoundland and Labrador, Canada

ACTIVE NOT RECRUITING

IIWK Health Centre

Halifax, Nova Scotia, Canada

RECRUITING

London Health Sciences Centre

London, Ontario, Canada

RECRUITING

Children's Hospital of Eastern Ontario

Ottawa, Ontario, Canada

RECRUITING

Hospital for Sick Children

Toronto, Ontario, Canada

COMPLETED

Royal University Hospital

Saskatoon, Saskatchewan, Canada

COMPLETED

Rigshospitalet

Copenhagen, Denmark

COMPLETED

University Children's Hospital

Münster, Germany

ACTIVE NOT RECRUITING

Sanjay Gandhi Post Graduate Institute

Lucknow, India

RECRUITING

Siriraj Hospital

Bangkok, Thailand

RECRUITING

Birmingham Children's Hospital NHS Foundation Trust

Birmingham, United Kingdom

RECRUITING

Royal Hospital for Children

Glasgow, United Kingdom

RECRUITING

Leeds Children's Hospital

Leeds, United Kingdom

RECRUITING

Alder Hey Children's Hospital

Liverpool, United Kingdom

RECRUITING

Royal Manchester Children's Hospital

Manchester, United Kingdom

RECRUITING

Great North Children's Hospital

Newcastle upon Tyne, United Kingdom

RECRUITING

Nottingham Children's Hospital

Nottingham, United Kingdom

RECRUITING

Nuffield Orthopaedic Centre

Oxford, United Kingdom

RECRUITING

Sheffield Children's Foundation Trust

Sheffield, United Kingdom

RECRUITING

Southampton General Hospital

Southampton, United Kingdom

RECRUITING

Related Links

• BrainWorks: The International Childhood CNS Vasculitis Outcome Study
• DCVAS: Diagnostic and Classification Criteria in Vasculitis Study

Biospecimen

Retention: SAMPLES WITH DNA

Serum, plasma, biopsy tissue, urine, DNA

MeSH Terms

Conditions

Granulomatosis with Polyangiitis; Microscopic Polyangiitis; Churg-Strauss Syndrome; Polyarteritis Nodosa; Takayasu Arteritis; Vasculitis, Central Nervous System; Vasculitis; Systemic Vasculitis; Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

Condition Hierarchy (Ancestors)

Lung Diseases, Interstitial; Lung Diseases; Respiratory Tract Diseases; Vascular Diseases; Cardiovascular Diseases; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Granuloma; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Arteritis; Aortic Arch Syndromes; Aortic Diseases; Autoimmune Diseases of the Nervous System

Study Officials

• 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

  Universität Münster

  PRINCIPAL INVESTIGATOR

• Robert Hancock, PhD

  University of British Columbia

  PRINCIPAL INVESTIGATOR

• Colin Ross, PhD

  University of British Columbia

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Else S. Bosman, PhD

CONTACT

pedvas@cw.bc.ca

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 3 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principle Investigator

Study Record Dates

• First Submitted: November 27, 2013
• First Posted: December 10, 2013
• Study Start: January 1, 2013
• Primary Completion: March 1, 2025
• Study Completion: March 1, 2025
• Last Updated: November 30, 2023

Record last verified: 2023-11

Locations

IN (1); TH (1); GB (10); US (10); DE (1); DK (1); CA (8)