Pediatric Vasculitis Initiative

Microscopic Polyangiitis Clinical Trial

— PedVas  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02006134
• Other study ID #: H12-00894
• Secondary ID: TR2-119188
• Status: Recruiting
• Start date: January 2013
• Est. completion date: March 2025

Study information

• Verified date: November 2023
• Source: University of British Columbia
• Contact: Else S. Bosman, PhD
• Email: pedvas[@]cw.bc.ca
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 1600
• Est. completion date: March 2025
• Est. primary completion date: March 2025
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: N/A to 20 Years

Eligibility

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

Related Conditions & MeSH terms

• Arteritis
• Churg-Strauss Syndrome
• Eosinophilic Granulomatosis With Polyangiitis
• Granulomatosis with Polyangiitis
• Microscopic Polyangiitis
• Polyarteritis Nodosa
• Primary CNS Vasculitis
• Systemic Vasculitis
• Takayasu Arteritis
• Unclassified Vasculitis
• Vasculitis
• Vasculitis, Central Nervous System
• Wegeners Granulomatosis (Granulomatosis With Polyangiitis)

Locations

Country	Name	City	State
Canada	University of Calgary / Alberta Children's Hospital	Calgary	Alberta
Canada	IIWK Health Centre	Halifax	Nova Scotia
Canada	London Health Sciences Centre	London	Ontario
Canada	Children's Hospital of Eastern Ontario	Ottawa	Ontario
Canada	Royal University Hospital	Saskatoon	Saskatchewan
Canada	Janeway Childrens Health and Rehabilitation Centre	St. John's	Newfoundland and Labrador
Canada	Hospital for Sick Children	Toronto	Ontario
Canada	BC Children's Hospital	Vancouver	British Columbia
Denmark	Rigshospitalet	Copenhagen
Germany	University Children's Hospital	Munster
India	Sanjay Gandhi Post Graduate Institute	Lucknow
Thailand	Siriraj Hospital	Bangkok
United Kingdom	Birmingham Children's Hospital NHS Foundation Trust	Birmingham
United Kingdom	Royal Hospital for Children	Glasgow
United Kingdom	Leeds Children's Hospital	Leeds
United Kingdom	Alder Hey Children's Hospital	Liverpool
United Kingdom	Royal Manchester Children's Hospital	Manchester
United Kingdom	Great North Children's Hospital	Newcastle upon Tyne
United Kingdom	Nottingham Children's Hospital	Nottingham
United Kingdom	Nuffield Orthopaedic Centre	Oxford
United Kingdom	Sheffield Children's Foundation Trust	Sheffield
United Kingdom	Southampton General Hospital	Southampton
United States	Akron Children's Hospital	Akron	Ohio
United States	Children's Hospital at Montefiore	Bronx	New York
United States	Comer Children's Hospital	Chicago	Illinois
United States	University of Florida	Gainesville	Florida
United States	The Joseph M. Sanzari Children's Hospital	Hackensack	New Jersey
United States	Texas Children's Hospital	Houston	Texas
United States	Riley Hospital for Children	Indianapolis	Indiana
United States	University of Utah / Primary Children's Hospital	Salt Lake City	Utah
United States	University of San Francisco	San Francisco	California
United States	Seattle Children's Hospital	Seattle	Washington

Sponsors (3)

Lead Sponsor	Collaborator
University of British Columbia	BC Childrens Hospital Research Institute, University of Oxford

Countries where clinical trial is conducted

United States,  Canada,  Denmark,  Germany,  India,  Thailand,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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

External Links

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