Pediatric Vasculitis Initiative

Study 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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

Yes
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Observational [Patient Registry]
Eligible Ages N/A - 20 Years
Gender All
More Inclusion & Exclusion 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

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT02006134
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

University of British Columbia
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

David Cabral, MBBSRaashid Luqmani, DM FRCP(E)Dirk Foell, MDRobert Hancock, PhDColin Ross, PhD
Principal Investigator Affiliation University of British Columbia; BC Children's HospitalUniversity of OxfordUniversität MünsterUniversity of British ColumbiaUniversity of British Columbia
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Other
Overall Status Recruiting
Countries Canada, Denmark, Germany, India, Thailand, United Kingdom, United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Wegeners Granulomatosis (Granulomatosis With Polyangiitis), Microscopic Polyangiitis, Eosinophilic Granulomatosis With Polyangiitis, Polyarteritis Nodosa, Takayasu Arteritis, Primary CNS Vasculitis, Unclassified Vasculitis
Study Website: View Trial Website
Additional Details

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.

Arms & Interventions

Arms

: 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.

Interventions

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

University of San Francisco, San Francisco, California

Status

Recruiting

Address

University of San Francisco

San Francisco, California,

Site Contact

[email protected]

University of Florida, Gainesville, Florida

Status

Recruiting

Address

University of Florida

Gainesville, Florida,

Site Contact

[email protected]

Comer Children's Hospital, Chicago, Illinois

Status

Recruiting

Address

Comer Children's Hospital

Chicago, Illinois,

Site Contact

[email protected]

Riley Hospital for Children, Indianapolis, Indiana

Status

Recruiting

Address

Riley Hospital for Children

Indianapolis, Indiana,

Site Contact

[email protected]

Hackensack, New Jersey

Status

Recruiting

Address

The Joseph M. Sanzari Children's Hospital

Hackensack, New Jersey,

Site Contact

[email protected]

Children's Hospital at Montefiore, Bronx, New York

Status

Recruiting

Address

Children's Hospital at Montefiore

Bronx, New York,

Site Contact

[email protected]

Akron Children's Hospital, Akron, Ohio

Status

Recruiting

Address

Akron Children's Hospital

Akron, Ohio,

Site Contact

[email protected]

Texas Children's Hospital, Houston, Texas

Status

Recruiting

Address

Texas Children's Hospital

Houston, Texas,

Site Contact

[email protected]

Salt Lake City, Utah

Status

Recruiting

Address

University of Utah / Primary Children's Hospital

Salt Lake City, Utah,

Site Contact

[email protected]

Seattle Children's Hospital, Seattle, Washington

Status

Recruiting

Address

Seattle Children's Hospital

Seattle, Washington,

Site Contact

[email protected]

International Sites

Calgary, Alberta, Canada

Status

Recruiting

Address

University of Calgary / Alberta Children's Hospital

Calgary, Alberta,

Site Contact

[email protected]

BC Children's Hospital, Vancouver, British Columbia, Canada

Status

Recruiting

Address

BC Children's Hospital

Vancouver, British Columbia,

Site Contact

[email protected]

St. John's, Newfoundland and Labrador, Canada

Status

Active, not recruiting

Address

Janeway Childrens Health and Rehabilitation Centre

St. John's, Newfoundland and Labrador,

Site Contact

[email protected]

IIWK Health Centre, Halifax, Nova Scotia, Canada

Status

Recruiting

Address

IIWK Health Centre

Halifax, Nova Scotia,

Site Contact

[email protected]

London Health Sciences Centre, London, Ontario, Canada

Status

Recruiting

Address

London Health Sciences Centre

London, Ontario,

Site Contact

[email protected]

Children's Hospital of Eastern Ontario, Ottawa, Ontario, Canada

Status

Recruiting

Address

Children's Hospital of Eastern Ontario

Ottawa, Ontario,

Site Contact

[email protected]

Hospital for Sick Children, Toronto, Ontario, Canada

Status

Completed

Address

Hospital for Sick Children

Toronto, Ontario,

Site Contact

[email protected]

Royal University Hospital, Saskatoon, Saskatchewan, Canada

Status

Completed

Address

Royal University Hospital

Saskatoon, Saskatchewan,

Site Contact

[email protected]

Rigshospitalet, Copenhagen, Denmark

Status

Completed

Address

Rigshospitalet

Copenhagen, ,

Site Contact

[email protected]

University Children's Hospital, Munster, Germany

Status

Active, not recruiting

Address

University Children's Hospital

Munster, ,

Site Contact

[email protected]

Sanjay Gandhi Post Graduate Institute, Lucknow, India

Status

Recruiting

Address

Sanjay Gandhi Post Graduate Institute

Lucknow, ,

Site Contact

[email protected]

Siriraj Hospital, Bangkok, Thailand

Status

Recruiting

Address

Siriraj Hospital

Bangkok, ,

Site Contact

[email protected]

Birmingham, United Kingdom

Status

Recruiting

Address

Birmingham Children's Hospital NHS Foundation Trust

Birmingham, ,

Site Contact

[email protected]

Royal Hospital for Children, Glasgow, United Kingdom

Status

Recruiting

Address

Royal Hospital for Children

Glasgow, ,

Site Contact

[email protected]

Leeds Children's Hospital, Leeds, United Kingdom

Status

Recruiting

Address

Leeds Children's Hospital

Leeds, ,

Site Contact

[email protected]

Alder Hey Children's Hospital, Liverpool, United Kingdom

Status

Recruiting

Address

Alder Hey Children's Hospital

Liverpool, ,

Site Contact

[email protected]

Royal Manchester Children's Hospital, Manchester, United Kingdom

Status

Recruiting

Address

Royal Manchester Children's Hospital

Manchester, ,

Site Contact

[email protected]

Great North Children's Hospital, Newcastle upon Tyne, United Kingdom

Status

Recruiting

Address

Great North Children's Hospital

Newcastle upon Tyne, ,

Site Contact

[email protected]

Nottingham Children's Hospital, Nottingham, United Kingdom

Status

Recruiting

Address

Nottingham Children's Hospital

Nottingham, ,

Site Contact

[email protected]

Nuffield Orthopaedic Centre, Oxford, United Kingdom

Status

Recruiting

Address

Nuffield Orthopaedic Centre

Oxford, ,

Site Contact

[email protected]

Sheffield Children's Foundation Trust, Sheffield, United Kingdom

Status

Recruiting

Address

Sheffield Children's Foundation Trust

Sheffield, ,

Site Contact

[email protected]

Southampton General Hospital, Southampton, United Kingdom

Status

Recruiting

Address

Southampton General Hospital

Southampton, ,

Site Contact

[email protected]