Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Studies of the Natural History, Pathogenesis, and Outcome of Idiopathic Systemic Vasculitis

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02257866
Recruitment Status : Recruiting
First Posted : October 7, 2014
Last Update Posted : June 12, 2019
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) )

Tracking Information
First Submitted Date October 4, 2014
First Posted Date October 7, 2014
Last Update Posted Date June 12, 2019
Study Start Date October 4, 2014
Estimated Primary Completion Date January 1, 2050   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: October 4, 2014)
To study the pathogenesis of patients affected with idiopathic systemic vasculitis, including clinical, radiographic, immunological, and genetic characteristics of the disease [ Time Frame: Ongoing ]
Original Primary Outcome Measures Same as current
Change History Complete list of historical versions of study NCT02257866 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures
 (submitted: October 4, 2014)
  • Screening for other NIAMS clinical trials- To evaluate patients referred for suspected vasculitis and provide an additional medical opinion on diagnosis and make recommendations for treatment [ Time Frame: Ongoing ]
  • To develop long term clinical and laboratory outcome parameters of multiorgan involvement in patients with GPA, MPA, EGPA, PAN, TAK, and GCA- Evaluation of blood, body fluid, tissue, and imaging biomarkers during disease flares and quiescence [ Time Frame: Ongoing ]
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Studies of the Natural History, Pathogenesis, and Outcome of Idiopathic Systemic Vasculitis
Official Title Studies of the Natural History, Pathogenesis, and Outcome of Idiopathic Systemic Vasculitis
Brief Summary

Background:

- Vasculitis is a group of diseases that inflame and damage blood vessels and tissue. It can cause many medical problems. Few tests can diagnose the disease, and none can reliably predict a relapse. Researchers want to study people s genes and follow people over time to see how the disease affects them.

Objective:

- To learn the signs, symptoms, imaging tests, genetic markers, and blood tests that can help identify people with vasculitis and predict what will happen to them over time.

Eligibility:

  • People age 5 and older who have or are thought to have vasculitis, or are related to someone with it.
  • Healthy volunteers.

Design:

  • Participants will be evaluated by a doctor who has expertise caring for patients with vasculitis.
  • Participants will give a blood sample. Some will give a urine sample.
  • Some participants may have brushings or biopsies taken from the inside lining of the nose.
  • Images of participants blood vessels may be taken using scans. For some scans, participants will lie on a table that moves in and out of a cylinder that takes pictures. For some scans, a contrast agent may be injected into an arm vein. Other scans may use a radioactive form of sugar. Healthy minors will not have scans.
  • Some participants will answer questionnaires. - Some participants will have their tests done at NIH. Others will have their doctor take the blood, saliva, or cheek swab samples and send them to NIH.
  • Some participants will have one visit lasting 1 2 (but sometimes up to 4) days. Some participants may have follow-up visits every 3 6 months, indefinitely.
Detailed Description

The purpose of this protocol is to study the natural history of idiopathic systemic vasculitis in children and adults. The idiopathic vasculitides are a group of rare, systemic diseases involving inflammation of arteries and other tissue with resulting organ- and lifethreatening disease courses. The different forms of idiopathic vasculitis are typically classified based upon the predominant size of the arteries affected in each condition, including small vessel vasculitis [granulomatosis with polyangiitis (GPA, Wegener s), microscopic polyangiitis (MPA), and eosinophilic granulomatosis with polyangiitis (EGPA, Churg-Strauss)]; medium vessel vasculitis [polyarteritis nodosa (PAN)]; and large vessel vasculitis [giant cell arteritis (GCA), Takayasu s arteritis (TAK), idiopathic aortitis (IA)], or variable sized vessel vasculitis [relapsing polychondritis (RP), Behcet s disease (BD). Although patients with each type of vasculitis manifest disease-specific aspects of illness, there are substantial disease and treatment burdens common to patients with vasculitis. For each type of idiopathic vasculitis, the disease course is often chronic, relapse is common and unpredictable, organ and tissue damage can accrue over time, new symptoms can occur late into the disease course, and treatment is often associated with toxicity and serious side effects.

The goals of this natural history protocol are to establish a cohort of pediatric and adult patients with vasculitis to prospectively evaluate the signs and symptoms, imaging findings, and blood and tissue biomarkers associated with pathogenesis and disease outcomes. In the small vessel vasculitides, where considerable progress has been made towards identifying pathologic mechanisms of disease, we will focus on elucidating the pathogenic role of neutrophils, selected biomarkers such as SERPINA1, and novel candidate biomarkers in circulating blood and at local tissue sites including the nasal mucosa. In the medium and large vessel vasculitides, we will identify novel candidate biomarkers for disease pathogenesis and outcomes and develop disease activity indices that incorporate existing and novel clinical, laboratory, genomic, and imaging biomarkers. For all types of vasculitis, a goal of the protocol is to identify patients for possible entry into future

treatment studies.

Patients enrolled in this protocol will undergo a history, physical examination, and laboratory evaluation. Since vasculitis is multi-system disease with variable patterns of organ involvement, patients may undergo further comprehensive evaluation of a particular affected organ system when clinically indicated. Peripheral blood samples will be collected from affected patients, unrelated healthy volunteers matched for age, sex, and ethnicity whenever possible, and in some cases unaffected family members to help identify and study the genes involved in vasculitis and their functions. We may ask some patients to undergo nasal biopsy and additional imaging studies for research purposes. For a small number of patients and family members, we may ask permission to perform whole genome or exome sequencing. Successful completion of these studies will improve our understanding of disease pathogenesis.

Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population idiopathic systemic vasculitis in children and adults@@@
Condition
  • Autoimmune Disease
  • Vascular Disease
  • Takayasu's Arteritis
  • Giant Cell Arteritis
  • Polyarteritis Nodosa
  • Relapsing Polychondritis
  • ANCA-Associated Vasculitis
Intervention Not Provided
Study Groups/Cohorts Vasculitis
Patients with known or suspected vasculitis age 5 or older
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: October 4, 2014)
500
Original Estimated Enrollment Same as current
Estimated Study Completion Date January 1, 2050
Estimated Primary Completion Date January 1, 2050   (Final data collection date for primary outcome measure)
Eligibility Criteria
  • INCLUSION CRITERIA:

SUBJECTS WITH VASCULITIS

  • Subjects who fulfill modified versions of the 1990 American College of Rheumatology (ACR) Classification Criteria for GPA31 and PAN
  • Subjects who fulfill the 1990 ACR Classification Criteria for EGPA, GCA, and TAK
  • Subjects who fulfill the 2012 Chapel Hill Nomenclature definition for MPA
  • Subjects who fulfill the International Study Group Classification Criteria for BD
  • Subjects with other suspected systemic or single-organ vasculitides

HEALTHY VOLUNTEERS

-Volunteers able to provide consent, or in the case of minors, assent

EXCLUSION CRITERIA:

SUBJECTS WITH VASCULITIS:

  • Subjects less than 5 years of age
  • Active malignancy, infection, or any medical condition that in the opinion of the investigator would warrant exclusion
  • Inability to provide consent, or in the case of minors, assent
  • Subjects with bleeding diathesis or on anticoagulant medications (eg coumadin, heparin, clopidogrel but not including aspirin or NSAIDs) are excluded from participation in nasal brushing or biopsy studies

HEALTHY VOLUNTEERS

  • Volunteers less than 5 years of age
  • Diagnosis of vasculitis or other autoimmune/autoinflamamtory disease, including systemic lupus erythematosus, rheumatoid arthritis, sarcoidosis, mixed connective tissue disease or any overlap autoimmune syndrome
  • Active malignancy, infection, or any medical condition that in the opinion of the investigator would warrant exclusion
  • Pregnant (by history of last menstrual period) or breast feeding subjects
  • Subjects with bleeding diathesis or taking anticoagulant medications (eg coumadin, heparin, clopidogrel but not including aspirin or NSAIDs) are excluded from participating in nasal brushing studies
Sex/Gender
Sexes Eligible for Study: All
Ages 5 Years to 99 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers Yes
Contacts
Contact: Elaine M Novakovich (301) 451-1450 enovakovich@mail.cc.nih.gov
Contact: Peter C Grayson, M.D. (301) 827-9187 peter.grayson@nih.gov
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02257866
Other Study ID Numbers 140200
14-AR-0200
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party National Institutes of Health Clinical Center (CC) ( National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) )
Study Sponsor National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Collaborators Not Provided
Investigators
Principal Investigator: Peter C Grayson, M.D. National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
PRS Account National Institutes of Health Clinical Center (CC)
Verification Date June 7, 2019