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Longitudinal Protocol for Granulomatosis With Polyangiitis (Wegener's) and Microscopic Polyangiitis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00315393
Recruitment Status : Active, not recruiting
First Posted : April 18, 2006
Last Update Posted : October 10, 2019
Office of Rare Diseases (ORD)
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Rare Diseases Clinical Research Network
Information provided by (Responsible Party):
Peter Merkel, University of Pennsylvania

Brief 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. 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.

Condition or disease
Granulomatosis With Polyangiitis Microscopic Polyangiitis Wegener's

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.

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Study Type : Observational
Estimated Enrollment : 1200 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Determining Disease Activity Biomarkers in Individuals With Granulomatosis With Polyangiitis (Wegener's) and Microscopic Polyangiitis
Study Start Date : April 2006
Estimated Primary Completion Date : April 2020
Estimated Study Completion Date : April 2020

Primary Outcome Measures :
  1. Discover biomarkers in GPA/MPA capable of measuring disease activity and response to treatment. [ Time Frame: Study completion ]

Secondary Outcome Measures :
  1. Measure the predictive value of biomarkers for clinical outcome in GPA/MPA [ Time Frame: Study completion. ]

Biospecimen Retention:   Samples With DNA
Blood (serum and plasma), urine, and DNA

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
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.

Inclusion Criteria:

  • 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:

    1. Nasal or oral inflammation with oral ulcers or nasal discharge with pus or blood
    2. Abnormal chest radiograph with nodules, fixed infiltrates, or cavities
    3. Urinary sediment with microhematuria or red cell casts
    4. Granulomatous inflammation within the wall of an artery or in the perivascular area on biopsy
    5. 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:

    1. Necrotizing vasculitis, with few or no immune deposits, that affects small vessels (i.e., capillaries, venules, arterioles)
    2. Necrotizing arteritis involving small- and medium-sized arteries may be present
    3. Necrotizing glomerulonephritis is very common
    4. Pulmonary capillaritis often occurs
  • Parent or guardian willing to provide informed consent, if applicable

Exclusion Criteria:

  • Simultaneous diagnoses of both GPA and MPA
  • Granulomatosis with polyangiitis (Churg-Strauss)
  • Takayasu's arteritis
  • Giant cell arteritis
  • Polyarteritis nodosa
  • Cogan's syndrome
  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00315393

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United States, Massachusetts
Boston University School of Medicine
Boston, Massachusetts, United States, 02118
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
United States, Ohio
Cleveland Clinic Foundation
Cleveland, Ohio, United States, 44195
United States, Pennsylvania
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
University of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15260
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84112
Canada, Ontario
St. Joseph's Healthcare
Hamilton, Ontario, Canada
Mount Sinai Hospital
Toronto, Ontario, Canada, M5T 3L9
Sponsors and Collaborators
University of Pennsylvania
Office of Rare Diseases (ORD)
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Rare Diseases Clinical Research Network
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Study Chair: Peter A. Merkel, MD, MPH University of Pennsylvania

Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Peter Merkel, Professor, University of Pennsylvania Identifier: NCT00315393    
Other Study ID Numbers: VCRC5505
U54AR057319 ( U.S. NIH Grant/Contract )
First Posted: April 18, 2006    Key Record Dates
Last Update Posted: October 10, 2019
Last Verified: October 2019
Keywords provided by Peter Merkel, University of Pennsylvania:
Additional relevant MeSH terms:
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Granulomatosis with Polyangiitis
Microscopic Polyangiitis
Systemic Vasculitis
Vascular Diseases
Cardiovascular Diseases
Lung Diseases, Interstitial
Lung Diseases
Respiratory Tract Diseases
Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
Autoimmune Diseases
Immune System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases