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
  Purpose

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
Granulomatosis With Polyangiitis
Microscopic Polyangiitis
Wegener's

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Determining Disease Activity Biomarkers in Individuals With Granulomatosis With Polyangiitis (Wegener's) and Microscopic Polyangiitis

Resource links provided by NLM:


Further study details as provided by University of Pennsylvania:

Primary Outcome Measures:
  • Discover biomarkers in GPA/MPA capable of measuring disease activity and response to treatment. [ Time Frame: Study completion ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Measure the predictive value of biomarkers for clinical outcome in GPA/MPA [ Time Frame: Study completion. ] [ Designated as safety issue: No ]

Biospecimen Retention:   Samples With DNA

Blood (serum and plasma), urine, and DNA


Estimated Enrollment: 600
Study Start Date: April 2006
Estimated Study Completion Date: April 2016
Estimated Primary Completion Date: April 2016 (Final data collection date for primary outcome measure)
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.

  Eligibility

Genders Eligible for Study:   Both
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.

Criteria

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
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00315393

Locations
United States, Massachusetts
Boston University School of Medicine Recruiting
Boston, Massachusetts, United States, 02118
Contact: Naomi Amudala, NP    617-414-2512    namudala@bu.edu   
Principal Investigator: Paul A. Monach, MD, PhD         
United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Contact: Sara Biorn    507-284-4862    biorn.sara@mayo.edu   
Principal Investigator: Ulrich Specks, MD         
Principal Investigator: Steven R. Ytterberg, MD         
United States, Ohio
Cleveland Clinic Foundation Recruiting
Cleveland, Ohio, United States, 44195
Contact: Katie Gartner    216-445-1397    gartnek@ccf.org   
Principal Investigator: Carol A. Langford, MD, MHS         
United States, Pennsylvania
University of Pennsylvania Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Brian Rice    215-614-4407    Brian.Rice@uphs.upenn.edu   
Principal Investigator: Peter Merkel, MD, MPH         
University of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15260
Contact: Dawn McBride, RN    412-586-3545    dlmc@pitt.edu   
Principal Investigator: Larry Moreland, MD         
United States, Utah
University of Utah Recruiting
Salt Lake City, Utah, United States, 84112
Contact: Julieanne Nielsen    801-585-0798    Julieanne.Nielsen@hsc.utah.edu   
Principal Investigator: Curry Koening, MD, MHS         
Canada, Ontario
St. Joseph's Healthcare Recruiting
Hamilton, Ontario, Canada
Contact: Sandra Messier    905-522-1155 ext 35873    smessier@stjoes.ca   
Principal Investigator: Nader A. Khalidi, MD         
Mount Sinai Hospital Recruiting
Toronto, Ontario, Canada, M5T 3L9
Contact: Julia Farquharson    416-586-8616    JFarquharson@mtsinai.on.ca   
Principal Investigator: Simon Carette, MD         
Sponsors and Collaborators
University of Pennsylvania
Rare Diseases Clinical Research Network
Investigators
Study Chair: Peter A. Merkel, MD, MPH University of Pennsylvania
  More Information

Additional Information:
Publications:
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Peter Merkel, Professor, University of Pennsylvania
ClinicalTrials.gov Identifier: NCT00315393     History of Changes
Other Study ID Numbers: RDCRN 5505, U54AR057319
Study First Received: April 14, 2006
Last Updated: November 22, 2013
Health Authority: United States: Federal Government

Keywords provided by University of Pennsylvania:
GPA
MPA
WG

Additional relevant MeSH terms:
Systemic Vasculitis
Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
Vasculitis
Wegener Granulomatosis
Microscopic Polyangiitis
Lung Diseases, Interstitial
Lung Diseases
Respiratory Tract Diseases
Vascular Diseases
Cardiovascular Diseases
Autoimmune Diseases
Immune System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases

ClinicalTrials.gov processed this record on August 28, 2014