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Determining Disease Activity Biomarkers in Individuals With Polyarteritis Nodosa

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: NCT00315406
Recruitment Status : Active, not recruiting
First Posted : April 18, 2006
Last Update Posted : May 25, 2021
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:
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
Polyarteritis Nodosa

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.

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Study Type : Observational
Estimated Enrollment : 150 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Longitudinal Protocol of Polyarteritis Nodosa
Actual Study Start Date : April 2006
Estimated Primary Completion Date : September 2022
Estimated Study Completion Date : September 2022

Resource links provided by the National Library of Medicine

Primary Outcome Measures :
  1. Discover biomarkers in PAN 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 PAN. [ 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 polyarteritis nodosa. Enrollment will be sequential and participants will have disease in various stages and of different duration.

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:


  1. Arteriographic abnormality
  2. Presence of granulocyte or mixed leukocyte infiltrate in an arterial wall on biopsy
  3. Mononeuropathy or polyneuropathy


  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

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): NCT00315406

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United States, Massachusetts
Brigham and Women's Hospital
Boston, Massachusetts, United States, 02115
Boston University School of Medicine
Boston, Massachusetts, United States, 02118
United States, Minnesota
Mayo Clinic College of Medicine
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:
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Responsible Party: Peter Merkel, Professor, University of Pennsylvania Identifier: NCT00315406    
Other Study ID Numbers: VCRC5504
U54AR057319 ( U.S. NIH Grant/Contract )
First Posted: April 18, 2006    Key Record Dates
Last Update Posted: May 25, 2021
Last Verified: May 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Peter Merkel, University of Pennsylvania:
Periarteritis Nodosa
Additional relevant MeSH terms:
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Polyarteritis Nodosa
Vascular Diseases
Cardiovascular Diseases
Systemic Vasculitis
Skin Diseases, Vascular
Skin Diseases