Determining Disease Activity Biomarkers in Individuals With Wegener's Granulomatosis and Microscopic Polyangiitis - Full Text View

Sponsors and Collaborators:	Office of Rare Diseases (ORD) Rare Diseases Clinical Research Network
Information provided by:	Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:	NCT00315393

Purpose

Wegener's granulomatosis (WG) 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 WG or MPA.

Condition
Wegener's Granulomatosis Microscopic Polyangiitis

MedlinePlus related topics: Vasculitis Wegener's Granulomatosis

U.S. FDA Resources

Study Type:	Observational
Study Design:	Cohort, Prospective
Official Title:	Longitudinal Protocol for Wegener's Granulomatosis and Microscopic Polyangiitis

Further study details as provided by Office of Rare Diseases (ORD):

Biospecimen Retention: Samples With DNA

Biospecimen Description:

Blood (serum and plasma), urine, and DNA

Estimated Enrollment:	300
Study Start Date:	April 2006
Estimated Study Completion Date:	April 2016
Estimated Primary Completion Date:	April 2011 (Final data collection date for primary outcome measure)

Detailed Description:

WG and MPA are two autoimmune disorders that cause systemic vasculitis. WG 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 WG and MPA patients. These biomarkers may be used to help direct clinical care for WG and MPA patients and assist in future drug development.

This study will last 5 years. 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 Wegener's granulomatosis and microscopic polyangiitis. Enrollment will be sequential and participants will have disease in various stages and of different duration.

Criteria

Inclusion Criteria:

Diagnosis of WG or MPA. Widely accepted diagnostic criteria, as opposed to classification criteria or definitions, have not been developed for WG and MPA.
For diagnosis of WG, 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 WG and MPA
Churg-Strauss syndrome
Takayasu's arteritis
Giant cell arteritis
Polyarteritis nodosa
Cogan's syndrome
Behcet's disease
Sarcoidosis
Kawasaki's 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

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

Locations

United States, Maryland
The Johns Hopkins Vasculitis Center	Recruiting
Baltimore, Maryland, United States, 21224
Contact: Cynthia Bethea 410-550-4390 cbethea3@jhmi.edu
Principal Investigator: Philip Seo, MD, MHS
United States, Massachusetts
Boston University School of Medicine Arthritis Center	Recruiting
Boston, Massachusetts, United States, 02118
Contact: Jessica Martin 617-414-2507 jmartin@bu.edu
Principal Investigator: Peter A. Merkel, MD, MPH
Principal Investigator: Paul A. Monach, MD
United States, Minnesota
Mayo Clinic	Recruiting
Rochester, Minnesota, United States, 55905
Contact: Kathy Mieras 507-284-9187 mieras.kathleen@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: Katherine Tuthill 216-444-5257 TUTHILLK@ccf.org
Principal Investigator: Carol A. Langford, MD, MHS
Canada, Ontario
Mount Sinai Hospital	Recruiting
Toronto, Ontario, Canada, M5T 3L9
Contact: Sara Sutherland 416-586-8616 SSutherland2@mtsinai.on.ca
Principal Investigator: Simon Carette, MD
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

Sponsors and Collaborators

Office of Rare Diseases (ORD)

Rare Diseases Clinical Research Network

Investigators

Study Chair:	Peter A. Merkel, MD, MPH	Boston University
Principal Investigator:	Carol A. Langford, MD, MHS	The Cleveland Clinic
Principal Investigator:	Philip Seo, MD, MHS	Johns Hopkins Vasculitis Center
Principal Investigator:	Ulrich Specks, MD	Mayo Clinic
Principal Investigator:	Steven R. Yetterberg, MD	Mayo Clinic

More Information

Publications:

Borgmann S, Haubitz M. Genetic impact of pathogenesis and prognosis of ANCA-associated vasculitides. Clin Exp Rheumatol. 2004;22(6 Suppl 36):S79-86. Review.

Cooley P, Taylor KH, Czika W, Seifer C, Taylor JF. Analysis of a biomarker for Wegener's granulomatosis. Int J Immunogenet. 2005 Aug;32(4):237-43.

Jagiello P, Gross WL, Epplen JT. Complex genetics of Wegener granulomatosis. Autoimmun Rev. 2005 Jan;4(1):42-7. Review.

Radice A, Sinico RA. Antineutrophil cytoplasmic antibodies (ANCA). Autoimmunity. 2005 Feb;38(1):93-103. Review.

Stone JH, Rajapakse VN, Hoffman GS, Specks U, Merkel PA, Spiera RF, Davis JC, St Clair EW, McCune J, Ross S, Hitt BA, Veenstra TD, Conrads TP, Liotta LA, Petricoin EF 3rd; Wegener's Granulomatosis Etanercept Trial Research Group. A serum proteomic approach to gauging the state of remission in Wegener's granulomatosis. Arthritis Rheum. 2005 Mar;52(3):902-10.

Responsible Party:	Boston University School of Medicine ( Peter A. Merkey, MD, MPH )
Study ID Numbers:	RDCRN 5505, U54RR019497, VCRC 5505
Study First Received:	April 14, 2006
Last Updated:	September 10, 2008
ClinicalTrials.gov Identifier:	NCT00315393
Health Authority:	United States: Federal Government

Keywords provided by Office of Rare Diseases (ORD):

WG
MPA

Study placed in the following topic categories:

Microscopic polyangiitis
Lung Diseases, Interstitial
Vasculitis
Urologic Diseases
Respiratory Tract Diseases

Wegener Granulomatosis
Lung Diseases
Wegener's granulomatosis
Vascular Diseases
Kidney Diseases

Additional relevant MeSH terms:

Cardiovascular Diseases

ClinicalTrials.gov processed this record on January 16, 2009