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

Completed

• Conditions: Granulomatosis With Polyangiitis; Microscopic Polyangiitis; Wegener's

• Registration Number: NCT00315393
• Lead Sponsor: 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.

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 Timeline

• Study Start: 2006-04
• Study First Submitted: 2006-04-14
• Study First Submitted QC: 2006-04-14
• Study First Posted: 2006-04-18
• Primary Completion: 2019-12
• Study Completion: 2019-12
• Status Verified: 2022-07
• Last Update Submitted: 2022-07-11
• Last Update Posted: 2022-07-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 1046

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Discover biomarkers in GPA/MPA capable of measuring disease activity and response to treatment.	Study completion

Secondary Outcome Measures

Name	Time	Method
Measure the predictive value of biomarkers for clinical outcome in GPA/MPA	Study completion.

Trial Locations

Locations (8)

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States

Boston University School of Medicine; 🇺🇸 Boston, Massachusetts, United States

Cleveland Clinic Foundation; 🇺🇸 Cleveland, Ohio, United States

University of Pennsylvania; 🇺🇸 Philadelphia, Pennsylvania, United States

University of Pittsburgh; 🇺🇸 Pittsburgh, Pennsylvania, United States

University of Utah; 🇺🇸 Salt Lake City, Utah, United States

St. Joseph's Healthcare; 🇨🇦 Hamilton, Ontario, Canada

Mount Sinai Hospital; 🇨🇦 Toronto, Ontario, Canada