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Determining Disease Activity Biomarkers in Individuals With Polyarteritis Nodosa

Sponsor
University of Pennsylvania (Other)
Overall Status
Completed
CT.gov ID
NCT00315406
Collaborator
Office of Rare Diseases (ORD) (NIH), National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) (NIH), Rare Diseases Clinical Research Network (Other)
107
Enrollment
9
Locations
164
Actual Duration (Months)
11.9
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Polyarteritis nodosa (PAN) is a rare immune system disorder that causes swelling and damage to small- and medium-sized blood vessels in the body. In order to properly treat this disease, 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 PAN.

Condition or Disease Intervention/Treatment Phase

    Detailed Description

    PAN, also known as systemic necrotic vasculitis, was the first recognized form of primary systemic vasculitis. PAN causes the inflammation of small- to medium-sized blood vessels, especially those supplying the nerves, skin, kidneys, gastrointestinal tract, heart, eye, and genitals. Unlike another form of vasculitis called microscopic polyangiitis, PAN does not usually cause glomerulonephritis, a type of kidney disease, or vasculitis in the very smallest blood vessels (arterioles, capillaries, and venules). There are no radiographic or serologic tests that can reliably measure disease activity in PAN. Currently, clinicians must rely on patients' symptoms, signs, laboratory tests, and imaging to guide treatment decisions, but such data are rarely consistently reliable in determining PAN disease activity. This study will use new scientific methods to discover new biomarkers that can be used to monitor disease activity in PAN patients. These biomarkers may be used to help direct clinical care for PAN 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

    Study Type:
    Observational
    Actual Enrollment :
    107 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    Longitudinal Protocol of Polyarteritis Nodosa
    Actual Study Start Date :
    Apr 1, 2006
    Actual Primary Completion Date :
    Dec 1, 2019
    Actual Study Completion Date :
    Dec 1, 2019

    Outcome Measures

    Primary Outcome Measures

    1. Discover biomarkers in PAN capable of measuring disease activity and response to treatment. [Study completion]

    Secondary Outcome Measures

    1. Measure the predictive value of biomarkers for clinical outcome in PAN. [Study completion.]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Parent or guardian willing to provide informed consent, if applicable

    • Diagnosis of vasculitis

    • Diagnosis of PAN, meeting at least 1 major criterion and 1 minor criterion OR 2 major criteria of the following adapted American College of Rheumatology (ACR) criteria that fall under the diagnosis of PAN and that are not explained by other causes:

    MAJOR CRITERIA

    1. Arteriographic abnormality

    2. Presence of granulocyte or mixed leukocyte infiltrate in an arterial wall on biopsy

    3. Mononeuropathy or polyneuropathy

    MINOR CRITERIA

    1. Weight loss of more than 4 kg (8.8 lbs)

    2. Livedo reticularis, cutaneous ulcerations, or skin nodules

    3. Testicular pain or tenderness

    4. Myalgias

    5. Diastolic blood pressure greater than 90 mm Hg

    6. Elevated blood urea nitrogen (BUN) or serum creatinine levels

    7. Ischemic abdominal pain

    Exclusion Criteria:

    • Microscopic polyangiitis

    • Granulomatosis with polyangiitis(Wegener's)

    • Eosinophilic granulomatosis with polyangiitis (Churg-Strauss)

    • Takayasu's arteritis

    • Giant cell arteritis

    • Cogan's syndrome

    • Behcet's disease

    • Sarcoidosis

    • Kawasaki disease

    • Cryoglobulinemic vasculitis

    • Systemic lupus erythematosus

    • Rheumatoid arthritis

    • Mixed connective tissue disease or any overlap autoimmune syndrome

    • Presence of antiproteinase 3 or antimyeloperoxidase antineutrophil cytoplasmic antibodies (ANCA)

    • Glomeronephritis

    • Alveolar hemorrhage

    • Hepatitis B, hepatitis C, or HIV infection

    • Any other infectious form of medium vessel vasculitis

    • Isolated cutaneous PAN

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Brigham and Women's Hospital Boston Massachusetts United States 02115
    2 Boston University School of Medicine Boston Massachusetts United States 02118
    3 Mayo Clinic College of Medicine Rochester Minnesota United States 55905
    4 Cleveland Clinic Foundation Cleveland Ohio United States 44195
    5 University of Pennsylvania Philadelphia Pennsylvania United States 19104
    6 University of Pittsburgh Pittsburgh Pennsylvania United States 15260
    7 University of Utah Salt Lake City Utah United States 84112
    8 St. Joseph's Healthcare Hamilton Ontario Canada
    9 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

    Investigators

    • Study Chair: Peter A. Merkel, MD, MPH, University of Pennsylvania

    Study Documents (Full-Text)

    None provided.

    More Information

    Additional Information:

    Publications

    Responsible Party:
    Peter Merkel, Professor, University of Pennsylvania
    ClinicalTrials.gov Identifier:
    NCT00315406
    Other Study ID Numbers:
    • VCRC5504
    • U54AR057319
    First Posted:
    Apr 18, 2006
    Last Update Posted:
    Jul 12, 2022
    Last Verified:
    Jul 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Keywords provided by Peter Merkel, Professor, University of Pennsylvania
    PAN
    Periarteritis Nodosa
    Additional relevant MeSH terms:
    Polyarteritis Nodosa

    Study Results

    No Results Posted as of Jul 12, 2022