Pediatric Vasculitis Initiative

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

Sponsors

Lead Sponsor: University of British Columbia

Collaborator: 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

Source University of British Columbia
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.

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.

Overall Status Unknown status
Start Date January 2013
Completion Date December 2019
Primary Completion Date March 2019
Study Type Observational [Patient Registry]
Primary Outcome
Measure Time Frame
Develop new benchmarks for outcome in pediatric patients with systemic or CNS vasculitis within 3 yrs
Enrollment 1600
Condition
Eligibility

Sampling Method: Non-Probability Sample

Criteria:

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

Gender: All

Minimum Age: N/A

Maximum Age: 20 Years

Healthy Volunteers: Accepts Healthy Volunteers

Overall Official
Overall Contact

Last Name: Angelyne Sarmiento

Email: [email protected]

Location
Facility: Status: Investigator:
University of San Francisco | San Francisco, California, United States Recruiting Erica Lawson, MD Principal Investigator
University of Florida | Gainesville, Florida, United States Recruiting Melissa Elder, MD, PhD Principal Investigator
Medical College of Georgia | Augusta, Georgia, United States Recruiting Rita Jerath, MB ChB Principal Investigator
Comer Children's Hospital | Chicago, Illinois, United States Not yet recruiting Linda Wagner-Weiner Principal Investigator
Riley Hospital for Children | Indianapolis, Indiana, United States Recruiting Kathleen O'Neil Principal Investigator
University of Louisville | Louisville, Kentucky, United States Recruiting Kenneth Schikler Principal Investigator
The Joseph M. Sanzari Children's Hospital | Hackensack, New Jersey, United States Recruiting Suzanne Li, MD Principal Investigator
Children's Hospital at Montefiore | Bronx, New York, United States Recruiting Dawn Wahezi, MD Principal Investigator
Akron Children's Hospital | Akron, Ohio, United States Recruiting Mary Toth, MD Principal Investigator
Texas Children's Hospital | Houston, Texas, United States Not yet recruiting Eyal Muscal, MD Principal Investigator
University of Utah / Primary Children's Hospital | Salt Lake City, Utah, United States Recruiting Aimee Hersh, MD Principal Investigator
Seattle Children's Hospital | Seattle, Washington, United States Recruiting Susan Shenoi Principal Investigator
Hospital de Pediatría Garrahan | Buenos Aires, Argentina Recruiting Ricardo Russo, MD Principal Investigator
University of Calgary / Alberta Children's Hospital | Calgary, Alberta, Canada Recruiting Susanne Benseler, MD, PhD Principal Investigator
BC Children's Hospital | Vancouver, British Columbia, Canada Recruiting David Cabral, MBBS Principal Investigator
Janeway Childrens Health and Rehabilitation Centre | St. John's, Newfoundland and Labrador, Canada Recruiting Paul Dancey Principal Investigator
IIWK Health Centre | Halifax, Nova Scotia, Canada Recruiting Adam Huber, MD Principal Investigator
London Health Sciences Centre | London, Ontario, Canada Recruiting Roberta Berard Principal Investigator
Children's Hospital of Eastern Ontario | Ottawa, Ontario, Canada Recruiting Ciaran Duffy, MD Principal Investigator
Hospital for Sick Children | Toronto, Ontario, Canada Recruiting Susanne Benseler, MD, PhD Principal Investigator Rae Yeung, MD, PhD, FRCPC Sub-Investigator
Royal University Hospital | Saskatoon, Saskatchewan, Canada Recruiting Alan Rosenberg Principal Investigator
Rigshospitalet | Copenhagen, Denmark Recruiting Susan Nielsen Principal Investigator
University Children's Hospital | Munster, Germany Recruiting Dirk Foell, MD Principal Investigator
Sanjay Gandhi Post Graduate Institute | Lucknow, India Recruiting Amita Aggarwal, MD Principal Investigator
Meyer Children's Hospital of Florence | Florence, Italy Recruiting Rolando Cimaz Principal Investigator
Instituto Nacional de Pediatria | Coyoacán, Mexico Not yet recruiting Ana Luisa Rodriguez Lozano, MD Principal Investigator
Saint-Petersburg State Pediatric Medical University | Saint Petersburg, Russian Federation Recruiting Mikhail Kostik, MD Principal Investigator
Mother and Child Health Care Institute of Serbia | Belgrade, Serbia Recruiting Goran Ristic, MD Principal Investigator
Hospital Sant Joan de Deu | Barcelona, Spain Not yet recruiting Jordi Anton, MD Principal Investigator
Siriraj Hospital | Bangkok, Thailand Recruiting Sirirat Charuvanij, MD Principal Investigator
Birmingham Children's Hospital NHS Foundation Trust | Birmingham, United Kingdom Recruiting [email protected] Taunton Southwood Principal Investigator
Royal Hospital for Children | Glasgow, United Kingdom Recruiting [email protected] Neil Martin Principal Investigator
Leeds Children's Hospital | Leeds, United Kingdom Recruiting [email protected] Mark Wood Principal Investigator
Alder Hey Children's Hospital | Liverpool, United Kingdom Recruiting [email protected] Eileen Baildam Principal Investigator
Royal Manchester Children's Hospital | Manchester, United Kingdom Recruiting [email protected] Janet McDonagh Principal Investigator
Great North Children's Hospital | Newcastle upon Tyne, United Kingdom Recruiting [email protected] Mark Friswell Principal Investigator
Nottingham Children's Hospital | Nottingham, United Kingdom Recruiting Lampros Fotis Principal Investigator
Nuffield Orthopaedic Centre | Oxford, United Kingdom Recruiting [email protected] Raashid Luqmani, DM FRCP, FRCP(E) Principal Investigator
Sheffield Children's Foundation Trust | Sheffield, United Kingdom Recruiting [email protected] Anne-Marie McMahon Principal Investigator
Southampton General Hospital | Southampton, United Kingdom Recruiting [email protected] Alice Leahy Principal Investigator
Location Countries

Argentina

Canada

Denmark

Germany

India

Italy

Mexico

Russian Federation

Serbia

Spain

Thailand

United Kingdom

United States

Verification Date

May 2018

Responsible Party

Type: Principal Investigator

Investigator Affiliation: University of British Columbia

Investigator Full Name: David Cabral

Investigator Title: Principle Investigator

Keywords
Has Expanded Access No
Condition Browse
Arm Group

Label: PEDIATRIC VASCULITIS/PROSPECTIVE

Description: 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.

Label: PEDIATRIC VASCULITIS/RETROSPECTIVE

Description: 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.

Label: ADULT VASCULITIS/ COHORT 1

Description: 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.

Label: ADULT VASCULITIS / COHORT 2

Description: 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.

Label: HEALTHY CHILDREN / PEDIATRIC CONTROL

Description: 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.

Label: HEALTHY ADULTS / ADULT CONTROL

Description: 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.

Acronym PedVas
Study Design Info

Observational Model: Cohort

Time Perspective: Prospective

Source: ClinicalTrials.gov