Wegeners Granulomatosis (Granulomatosis With Polyangiitis)PedVasNCT02006134
Pediatric Vasculitis Initiative
An observational study of Wegeners Granulomatosis (Granulomatosis With Polyangiitis) and Microscopic Polyangiitis, run by University of British Columbia at 32 sites in 7 countries.
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. Read more about this study
- Who is it for?
- Ages Up to 20 yearsHealthy volunteers can take part
- Conditions the study is for
- Wegeners Granulomatosis (Granulomatosis With Polyangiitis), Microscopic Polyangiitis, Eosinophilic Granulomatosis With Polyangiitis
- How many people
- 1,600 participants
- Where
- 32 sitesin 7 countries
- Study dates
- Jan 2013to Mar 2025
- How long will I take part?
- 3 Years
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 20 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Study population: Children or adolescents diagnosed with vasculitis at any of the participating PedVas study centres
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
32 sites in 7 countries.
Else S. Bosman, PhD contact
32 sites in 7 countries
United States10 sites, 10 recruiting
- Recruiting
Akron Children's Hospital
Akron, Ohio
Mary Toth, MD
- Recruiting
Comer Children's Hospital
Chicago, Illinois
Linda Wagner-Weiner
- Recruiting
University of Florida
Gainesville, Florida
Melissa Elder, MD, PhD
- Recruiting
The Joseph M. Sanzari Children's Hospital
Hackensack, New Jersey
Suzanne Li, MD
- Recruiting
Texas Children's Hospital
Houston, Texas
Alvaro Orjuela, MD
Marietta DeGuzman, MD
- Recruiting
Riley Hospital for Children
Indianapolis, Indiana
Stacey Tarvin
- Recruiting
University of Utah / Primary Children's Hospital
Salt Lake City, Utah
Aimee Hersh, MD
- Recruiting
University of San Francisco
San Francisco, California
Susan Kim, MD
- Recruiting
Seattle Children's Hospital
Seattle, Washington
Susan Shenoi
- Recruiting
Children's Hospital at Montefiore
The Bronx, New York
Dawn Wahezi, MD
United Kingdom10 sites, 10 recruiting
- Recruiting
Birmingham Children's Hospital NHS Foundation Trust
Birmingham
ukpedvas@ndorms.ox.ac.uk
Taunton Southwood
- Recruiting
Royal Hospital for Children
Glasgow
ukpedvas@ndorms.ox.ac.uk
Neil Martin
- Recruiting
Leeds Children's Hospital
Leeds
ukpedvas@ndorms.ox.ac.uk
Mark Wood
- Recruiting
Alder Hey Children's Hospital
Liverpool
ukpedvas@ndorms.ox.ac.uk
Eileen Baildam
- Recruiting
Royal Manchester Children's Hospital
Manchester
ukpedvas@ndorms.ox.ac.uk
Janet McDonagh
- Recruiting
Great North Children's Hospital
Newcastle upon Tyne
ukpedvas@ndorms.ox.ac.uk
Mark Friswell
- Recruiting
Nottingham Children's Hospital
Nottingham
Lampros Fotis
- Recruiting
Nuffield Orthopaedic Centre
Oxford
ukpedvas@ndorms.ox.ac.uk
Raashid Luqmani, DM FRCP, FRCP(E)
- Recruiting
Sheffield Children's Foundation Trust
Sheffield
ukpedvas@ndorms.ox.ac.uk
Anne-Marie McMahon
- Recruiting
Southampton General Hospital
Southampton
ukpedvas@ndorms.ox.ac.uk
Alice Leahy
Canada8 sites, 5 recruiting
- Recruiting
University of Calgary / Alberta Children's Hospital
Calgary, Alberta
Susanne Benseler, MD, PhD
- Recruiting
IIWK Health Centre
Halifax, Nova Scotia
Adam Huber, MD
- Recruiting
London Health Sciences Centre
London, Ontario
Roberta Berard
- Recruiting
Children's Hospital of Eastern Ontario
Ottawa, Ontario
Ciaran Duffy, MD
- Completed
Royal University Hospital
Saskatoon, Saskatchewan
- Active, not recruiting
Janeway Childrens Health and Rehabilitation Centre
St. John's, Newfoundland and Labrador
- Completed
Hospital for Sick Children
Toronto, Ontario
- Recruiting
BC Children's Hospital
Vancouver, British Columbia
David Cabral, MBBS
India1 site, 1 recruiting
- Recruiting
Sanjay Gandhi Post Graduate Institute
Lucknow
Amita Aggarwal, MD
Thailand1 site, 1 recruiting
- Recruiting
Siriraj Hospital
Bangkok
Sirirat Charuvanij, MD
About this study
Official title: Chronic Childhood Vasculitis: Characterizing the Individual Rare Diseases to Improve Patient Outcomes
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.
Read the detailed 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.
- Conditions
- Wegeners Granulomatosis (Granulomatosis With Polyangiitis), Microscopic Polyangiitis, Eosinophilic Granulomatosis With Polyangiitis, Polyarteritis Nodosa, Takayasu Arteritis, Primary CNS 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
- Medical subject headings
- Granulomatosis with Polyangiitis, Microscopic Polyangiitis, Churg-Strauss Syndrome, Polyarteritis Nodosa, Takayasu Arteritis, Vasculitis, Central Nervous System, Vasculitis, Systemic Vasculitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: within 3 yrs
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Dec 2013 (estimated)
- Study startJan 2013
- Primary completionMar 2025 (estimated)
- Study completionMar 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Wegeners Granulomatosis (Granulomatosis With Polyangiitis)Studies of the same condition, open and under way.
- More trials run by University of British ColumbiaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1,600 participants (estimated)
- Target duration
- 3 Years
Sponsor and investigators
- Lead sponsor
- University of British Columbia
- Sponsor type
- University, hospital or other
- Collaborators
- BC Children's Hospital Research Institute, University of Oxford
- Responsible party
- Principal investigator, David Cabral, University of British Columbia
- Investigators
- David Cabral, MBBS, Principal investigator, University of British Columbia; BC Children's HospitalRaashid Luqmani, DM FRCP(E), Principal investigator, University of OxfordDirk Foell, MD, Principal investigator, Universität MünsterRobert Hancock, PhD, Principal investigator, University of British ColumbiaColin Ross, PhD, Principal investigator, University of British Columbia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02006134
- Sponsor's protocol ID
- H12-00894
- Other IDs
- TR2-119188
- Acronym
- PedVas
- Status last verified
- Nov 2023
- First submitted
- 27 Nov 2013
- Last updated
- 30 Nov 2023
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Chronic Childhood Vasculitis: Characterizing the Individual Rare Diseases to Improve Patient Outcomes
- ClinicalTrials.gov ID
- NCT02006134
- Acronym / sponsor ID
- PedVas / H12-00894
- Conditions
- Wegeners Granulomatosis (Granulomatosis With Polyangiitis), Microscopic Polyangiitis, Eosinophilic Granulomatosis With Polyangiitis, Polyarteritis Nodosa, Takayasu Arteritis, Primary CNS Vasculitis
- Keywords
- Vasculitis, Primary CNS Vasculitis, Systemic vasculitis, Churg-Strauss Syndrome, Polyarteritis Nodosa, Wegener Granulomatosis, Pediatric vasculitis, Childhood vasculitis
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 1,600 participants (estimated)
- Lead sponsor
- University of British ColumbiaUniversity, hospital or other
- Collaborators
- BC Children's Hospital Research Institute, University of Oxford
- Study start
- Jan 2013
- Primary completion
- Mar 2025 (estimated)
- Study completion
- Mar 2025 (estimated)
- First posted
- 10 Dec 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 3 Years
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- Up to 20 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- Children or adolescents diagnosed with vasculitis at any of the participating PedVas study centres
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Develop new benchmarks for outcome in pediatric patients with systemic vasculitis
Time frame: within 3 yrs
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.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Nov 2023.
- BrainWorks: The International Childhood CNS Vasculitis Outcome StudyLink provided by the sponsor.
- DCVAS: Diagnostic and Classification Criteria in Vasculitis StudyLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Else S. Bosman, PhD contact
David Cabral, MBBS principal investigator
University of British Columbia; BC Children's Hospital
Raashid Luqmani, DM FRCP(E) principal investigator
University of Oxford
Dirk Foell, MD principal investigator
Universität Münster
32 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Wegeners Granulomatosis (Granulomatosis With Polyangiitis) and Microscopic Polyangiitis, run by University of British Columbia at 32 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 30 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites in United States, United Kingdom, Canada, Denmark, Germany and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Else S. Bosman, PhD (pedvas@cw.bc.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Develop new benchmarks for outcome in pediatric patients with systemic vasculitis (time frame: within 3 yrs). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Nov 2023.