Determining Disease Activity Biomarkers in Individuals With Polyarteritis Nodosa

Brief Title

Determining Disease Activity Biomarkers in Individuals With Polyarteritis Nodosa

Official Title

Longitudinal Protocol of Polyarteritis Nodosa

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.
    

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 Type

Observational


Primary Outcome

Discover biomarkers in PAN capable of measuring disease activity and response to treatment.

Secondary Outcome

 Measure the predictive value of biomarkers for clinical outcome in PAN.

Condition

Polyarteritis Nodosa



Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

150

Start Date

April 2006

Completion Date

September 2022

Primary Completion Date

September 2022

Eligibility Criteria

        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
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Peter A. Merkel, MD, MPH, , 

Location Countries

Canada

Location Countries

Canada

Administrative Informations


NCT ID

NCT00315406

Organization ID

VCRC5504

Secondary IDs

U54AR057319

Responsible Party

Principal Investigator

Study Sponsor

University of Pennsylvania

Collaborators

 Office of Rare Diseases (ORD)

Study Sponsor

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


Verification Date

May 2021