Granulomatosis With PolyangiitisNCT00315393
Longitudinal Protocol for Granulomatosis With Polyangiitis (Wegener's) and Microscopic Polyangiitis
An observational study of Granulomatosis With Polyangiitis and Microscopic Polyangiitis, run by University of Pennsylvania at 8 sites in 2 countries.
Trial summary
Granulomatosis with polyangiitis (Wegener's) (GPA) and microscopic polyangiitis (MPA) are two rare immune system disorders that cause the inflammation of blood vessels, or vasculitis. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Granulomatosis With Polyangiitis, Microscopic Polyangiitis, Wegener's
- How many people
- 1,046 participants
- Where
- 8 sitesin 2 countries
- Study dates
- Apr 2006to Dec 2019
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Individuals with granulomatosis with polyangiitis (Wegener's)and microscopic polyangiitis. Enrollment will be sequential and participants will have disease in various stages and of different duration.
Who may be able to join
- Diagnosis of GPA or MPA. Widely accepted diagnostic criteria, as opposed to classification criteria or definitions, have not been developed for GPA and MPA.
- For diagnosis of GPA, meets at least 2 of the following 5 modified American College of Rheumatology (ACR) criteria:
- Nasal or oral inflammation with oral ulcers or nasal discharge with pus or blood
- Abnormal chest radiograph with nodules, fixed infiltrates, or cavities
- Urinary sediment with microhematuria or red cell casts
- Granulomatous inflammation within the wall of an artery or in the perivascular area on biopsy
Show all 13Show fewer
- Antineutrophil cytoplasmic antibody (ANCA) positive by enzyme immunoassay for either PR3- or MPO-ANCA
- For diagnosis of MPA, meets the Chapel Hill Consensus Conference Definition for MPA:
- Necrotizing vasculitis, with few or no immune deposits, that affects small vessels (i.e., capillaries, venules, arterioles)
- Necrotizing arteritis involving small- and medium-sized arteries may be present
- Necrotizing glomerulonephritis is very common
- Pulmonary capillaritis often occurs
- Parent or guardian willing to provide informed consent, if applicable
Who cannot join
- Simultaneous diagnoses of both GPA and MPA
- Granulomatosis with polyangiitis (Churg-Strauss)
- Takayasu's arteritis
- Giant cell arteritis
- Polyarteritis nodosa
- Cogan's syndrome
Show all 16Show fewer
- Behcet's disease
- Sarcoidosis
- Kawasaki disease
- Tuberculosis or any atypical mycobacterial infections
- Deep fungal infections
- Lymphoma, lymphomatoid granulomatosis, or any other type of cancer that mimics anti-neutrophil cytoplasmic antibody-associated vasculitis (AAVs)
- Cryoglobulinemic vasculitis
- Systemic lupus erythematosus
- Rheumatoid arthritis
- Mixed connective tissue disease or any overlapping autoimmune syndrome
Trial locations and contacts
8 sites in 2 countries.
8 sites in 2 countries
United States6 sites
Boston University School of Medicine
Boston, Massachusetts, 02118
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
University of Pittsburgh
Pittsburgh, Pennsylvania, 15260
Mayo Clinic
Rochester, Minnesota, 55905
University of Utah
Salt Lake City, Utah, 84112
Canada2 sites
St. Joseph's Healthcare
Hamilton, Ontario
Mount Sinai Hospital
Toronto, Ontario, M5T 3L9
About this study
Official title: Determining Disease Activity Biomarkers in Individuals With Granulomatosis With Polyangiitis (Wegener's) and Microscopic Polyangiitis
Granulomatosis with polyangiitis (Wegener's) (GPA) and microscopic polyangiitis (MPA) are two rare immune system disorders that cause the inflammation of blood vessels, or vasculitis. In order to properly treat these diseases, it is critical that the level of disease activity can be determined over the course of the disease. The purpose of this study is to determine new biological markers, or biomarkers, that may be used to assess the severity of disease in people with GPA or MPA.
Read the detailed description
GPA and MPA are two autoimmune disorders that cause systemic vasculitis. GPA commonly affects the upper respiratory tract, the lungs, and the kidneys. MPA is marked by kidney inflammation, weight loss, skin lesions, nerve damage, and fever. Many patients with WG or MPA show no visible symptoms of active disease; it is known that underlying subclinical disease activity leads to long-term damage in these patients. Also, because it is difficult to monitor WG and MPA disease activity, it is difficult for clinicians to know when and how to treat these patients. This study will use new scientific methods to identify new biomarkers that can be used to monitor disease activity in GPA and MPA patients. These biomarkers may be used to help direct clinical care for GPA and MPA patients and assist in future drug development.
Study visits will occur monthly for the first year, then every 3 months thereafter for the remainder of the study. Blood and urine collection will occur at every visit. A physical exam and medical and medication history will occur every 3 months; also, participants will be asked to complete several questionnaires to assess disease activity, health status, and tobacco, alcohol, and drug use. Participants may have additional study visits if a disease flare or disease-related complications occur during the study.
- Keywords
- GPA, MPA, WG
- Medical subject headings
- Granulomatosis with Polyangiitis, Microscopic Polyangiitis
What is being measured
The primary outcome is the main question the study is designed to answer.
Discover biomarkers in GPA/MPA capable of measuring disease activity and response to treatment.
Measured: Study completion
Secondary outcomes (1)
Measure the predictive value of biomarkers for clinical outcome in GPA/MPA
Measured: Study completion.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Apr 2006 (estimated)
- Study startApr 2006
- Primary completionDec 2019
- Study completionDec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Granulomatosis With PolyangiitisStudies of the same condition, open and under way.
- More trials run by University of PennsylvaniaThe 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
- 1,046 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Pennsylvania
- Sponsor type
- University, hospital or other
- Collaborators
- Office of Rare Diseases (ORD), National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), Rare Diseases Clinical Research Network
- Responsible party
- Principal investigator, Peter Merkel, University of Pennsylvania
- Investigators
- Peter A. Merkel, MD, MPH, Study chair, University of Pennsylvania
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00315393
- Sponsor's protocol ID
- VCRC5505
- Other IDs
- U54AR057319
- Status last verified
- Jul 2022
- First submitted
- 14 Apr 2006
- Last updated
- 12 Jul 2022
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Borgmann S, Haubitz M. Genetic impact of pathogenesis and prognosis of ANCA-associated vasculitides. Clin Exp Rheumatol. 2004;22(6 Suppl 36):S79-86.
PubMed 15675141Cooley P, Taylor KH, Czika W, Seifer C, Taylor JF. Analysis of a biomarker for Wegener's granulomatosis. Int J Immunogenet. 2005 Aug;32(4):237-43. doi: 10.1111/j.1744-313X.2005.00519.x.
PubMed 16026591Jagiello P, Gross WL, Epplen JT. Complex genetics of Wegener granulomatosis. Autoimmun Rev. 2005 Jan;4(1):42-7. doi: 10.1016/j.autrev.2004.06.003.
PubMed 15652778Radice A, Sinico RA. Antineutrophil cytoplasmic antibodies (ANCA). Autoimmunity. 2005 Feb;38(1):93-103. doi: 10.1080/08916930400022673.
PubMed 15804710Stone JH, Rajapakse VN, Hoffman GS, Specks U, Merkel PA, Spiera RF, Davis JC, St Clair EW, McCune J, Ross S, Hitt BA, Veenstra TD, Conrads TP, Liotta LA, Petricoin EF 3rd; Wegener's Granulomatosis Etanercept Trial Research Group. A serum proteomic approach to gauging the state of remission in Wegener's granulomatosis. Arthritis Rheum. 2005 Mar;52(3):902-10. doi: 10.1002/art.20938.
PubMed 15751091Tomasson G, Boers M, Walsh M, LaValley M, Cuthbertson D, Carette S, Davis JC, Hoffman GS, Khalidi NA, Langford CA, McAlear CA, McCune WJ, Monach PA, Seo P, Specks U, Spiera R, St Clair EW, Stone JH, Ytterberg SR, Merkel PA. Assessment of health-related quality of life as an outcome measure in granulomatosis with polyangiitis (Wegener's). Arthritis Care Res (Hoboken). 2012 Feb;64(2):273-9. doi: 10.1002/acr.20649.
PubMed 21954229
Study at a glance
From the registry record, last updated by the sponsor on 12 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Determining Disease Activity Biomarkers in Individuals With Granulomatosis With Polyangiitis (Wegener's) and Microscopic Polyangiitis
- ClinicalTrials.gov ID
- NCT00315393
- Acronym / sponsor ID
- VCRC5505
- Keywords
- GPA, MPA, WG
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 1,046 participants (actual)
- Lead sponsor
- University of PennsylvaniaUniversity, hospital or other
- Collaborators
- Office of Rare Diseases (ORD), National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), Rare Diseases Clinical Research Network
- Study start
- Apr 2006
- Primary completion
- Dec 2019 (actual)
- Study completion
- Dec 2019 (actual)
- First posted
- 18 Apr 2006 (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.
Granulomatosis with polyangiitis (Wegener's) (GPA) and microscopic polyangiitis (MPA) are two rare immune system disorders that cause the inflammation of blood vessels, or vasculitis. In order to properly treat these diseases, it is critical that the level of disease activity can be determined over the course of the disease. The purpose of this study is to determine new biological markers, or biomarkers, that may be used to assess the severity of disease in people with GPA or MPA.
Detailed description
GPA and MPA are two autoimmune disorders that cause systemic vasculitis. GPA commonly affects the upper respiratory tract, the lungs, and the kidneys. MPA is marked by kidney inflammation, weight loss, skin lesions, nerve damage, and fever. Many patients with WG or MPA show no visible symptoms of active disease; it is known that underlying subclinical disease activity leads to long-term damage in these patients. Also, because it is difficult to monitor WG and MPA disease activity, it is difficult for clinicians to know when and how to treat these patients. This study will use new scientific methods to identify new biomarkers that can be used to monitor disease activity in GPA and MPA patients. These biomarkers may be used to help direct clinical care for GPA and MPA patients and assist in future drug development.
Study visits will occur monthly for the first year, then every 3 months thereafter for the remainder of the study. Blood and urine collection will occur at every visit. A physical exam and medical and medication history will occur every 3 months; also, participants will be asked to complete several questionnaires to assess disease activity, health status, and tobacco, alcohol, and drug use. Participants may have additional study visits if a disease flare or disease-related complications occur during the study.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Individuals with granulomatosis with polyangiitis (Wegener's)and microscopic polyangiitis. Enrollment will be sequential and participants will have disease in various stages and of different duration.
Key inclusion criteria (13 listed)
- Diagnosis of GPA or MPA. Widely accepted diagnostic criteria, as opposed to classification criteria or definitions, have not been developed for GPA and MPA.
- For diagnosis of GPA, meets at least 2 of the following 5 modified American College of Rheumatology (ACR) criteria:
- For diagnosis of MPA, meets the Chapel Hill Consensus Conference Definition for MPA:
- Parent or guardian willing to provide informed consent, if applicable
Key exclusion criteria (16 listed)
- Simultaneous diagnoses of both GPA and MPA
- Granulomatosis with polyangiitis (Churg-Strauss)
- Takayasu's arteritis
- Giant cell arteritis
- Polyarteritis nodosa
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
Discover biomarkers in GPA/MPA capable of measuring disease activity and response to treatment.
Time frame: Study completion
1 secondary outcome
Measure the predictive value of biomarkers for clinical outcome in GPA/MPA
Time frame: Study completion.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jul 2022.
- Background: PubMed 15675141Borgmann S, Haubitz M. Genetic impact of pathogenesis and prognosis of ANCA-associated vasculitides. Clin Exp Rheumatol. 2004;22(6 Suppl 36):S79-86.
- Background: PubMed 16026591Cooley P, Taylor KH, Czika W, Seifer C, Taylor JF. Analysis of a biomarker for Wegener's granulomatosis. Int J Immunogenet. 2005 Aug;32(4):237-43. doi: 10.1111/j.1744-313X.2005.00519.x.
- Background: PubMed 15652778Jagiello P, Gross WL, Epplen JT. Complex genetics of Wegener granulomatosis. Autoimmun Rev. 2005 Jan;4(1):42-7. doi: 10.1016/j.autrev.2004.06.003.
- Background: PubMed 15804710Radice A, Sinico RA. Antineutrophil cytoplasmic antibodies (ANCA). Autoimmunity. 2005 Feb;38(1):93-103. doi: 10.1080/08916930400022673.
- Background: PubMed 15751091Stone JH, Rajapakse VN, Hoffman GS, Specks U, Merkel PA, Spiera RF, Davis JC, St Clair EW, McCune J, Ross S, Hitt BA, Veenstra TD, Conrads TP, Liotta LA, Petricoin EF 3rd; Wegener's Granulomatosis Etanercept Trial Research Group. A serum proteomic approach to gauging the state of remission in Wegener's granulomatosis. Arthritis Rheum. 2005 Mar;52(3):902-10. doi: 10.1002/art.20938.
- Derived from this study: PubMed 21954229Tomasson G, Boers M, Walsh M, LaValley M, Cuthbertson D, Carette S, Davis JC, Hoffman GS, Khalidi NA, Langford CA, McAlear CA, McCune WJ, Monach PA, Seo P, Specks U, Spiera R, St Clair EW, Stone JH, Ytterberg SR, Merkel PA. Assessment of health-related quality of life as an outcome measure in granulomatosis with polyangiitis (Wegener's). Arthritis Care Res (Hoboken). 2012 Feb;64(2):273-9. doi: 10.1002/acr.20649.
- Vasculitis Clinical Research ConsortiumLink provided by the sponsor.
- Rare Diseases Clinical Research ConsortiumLink 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.
Peter A. Merkel, MD, MPH study chair
University of Pennsylvania
8 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Granulomatosis With Polyangiitis and Microscopic Polyangiitis, run by University of Pennsylvania at 8 sites in 2 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 13 inclusion and 16 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: finished, as of the sponsor's last update on 12 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States, Canada. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Discover biomarkers in GPA/MPA capable of measuring disease activity and response to treatment. (time frame: Study completion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00315393, which also lists 6 linked publications.
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 12 Jul 2022.