Determining Disease Activity Biomarkers in Individuals With Polyarteritis Nodosa

NCT ID: NCT00315406

Last Updated: 2022-07-12

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 107 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2006-04-30
• Study Completion Date: 2019-12-31

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.

Conditions

Polyarteritis Nodosa

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

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

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Office of Rare Diseases (ORD)

NIH

Sponsor Role: collaborator

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

NIH

Sponsor Role: collaborator

Rare Diseases Clinical Research Network

NETWORK

Sponsor Role: collaborator

University of Pennsylvania

OTHER

Sponsor Role: lead

Responsible Party

Peter Merkel

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Peter A. Merkel, MD, MPH

Role: STUDY_CHAIR

University of Pennsylvania

Locations

Brigham and Women's Hospital

Boston, Massachusetts, United States

Boston University School of Medicine

Boston, Massachusetts, United States

Mayo Clinic College of Medicine

Rochester, Minnesota, 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

Countries

United States; Canada

References

Bonsib SM. Polyarteritis nodosa. Semin Diagn Pathol. 2001 Feb;18(1):14-23.

Reference Type: BACKGROUND

PMID: 11296989 (View on PubMed)

Said G, Lacroix C. Primary and secondary vasculitic neuropathy. J Neurol. 2005 Jun;252(6):633-41. doi: 10.1007/s00415-005-0833-9. Epub 2005 Apr 5.

Reference Type: BACKGROUND

PMID: 15806339 (View on PubMed)

Related Links

http://rarediseasesnetwork.org/cms/vcrc/

Vasculitis Clinical Research Consortium

http://rarediseasesnetwork.org/

Rare Diseases Clinical Research Network

Other Identifiers

U54AR057319

Identifier Type: NIH

Identifier Source: secondary_id

View Link

VCRC5504

Identifier Type: -

Identifier Source: org_study_id