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

This study is currently recruiting participants. (see Contacts and Locations)
Verified November 2013 by University of Pennsylvania
Sponsor:
Collaborators:
Rare Diseases Clinical Research Network
Information provided by (Responsible Party):
Peter Merkel, University of Pennsylvania
ClinicalTrials.gov Identifier:
NCT00315393
First received: April 14, 2006
Last updated: November 22, 2013
Last verified: November 2013

April 14, 2006
November 22, 2013
April 2006
April 2016   (final data collection date for primary outcome measure)
Discover biomarkers in GPA/MPA capable of measuring disease activity and response to treatment. [ Time Frame: Study completion ] [ Designated as safety issue: No ]
Not Provided
Complete list of historical versions of study NCT00315393 on ClinicalTrials.gov Archive Site
Measure the predictive value of biomarkers for clinical outcome in GPA/MPA [ Time Frame: Study completion. ] [ Designated as safety issue: No ]
Not Provided
Not Provided
Not Provided
 
Longitudinal Protocol for Granulomatosis With Polyangiitis (Wegener's) and Microscopic Polyangiitis
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.

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.

Observational
Observational Model: Cohort
Time Perspective: Prospective
Not Provided
Retention:   Samples With DNA
Description:

Blood (serum and plasma), urine, and DNA

Non-Probability Sample

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.

  • Granulomatosis With Polyangiitis
  • Microscopic Polyangiitis
  • Wegener's
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
600
April 2016
April 2016   (final data collection date for primary outcome measure)

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
Both
Not Provided
No
United States,   Canada
 
NCT00315393
RDCRN 5505, U54AR057319
Yes
Peter Merkel, University of Pennsylvania
University of Pennsylvania
  • Office of Rare Diseases (ORD)
  • National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
  • Rare Diseases Clinical Research Network
Study Chair: Peter A. Merkel, MD, MPH University of Pennsylvania
University of Pennsylvania
November 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP