Longitudinal Study for Central Nervous System Vasculitis

Primary central nervous system vasculitis (CNSV) is a potentially fatal, single-organ vasculitis that often involves a spectrum of neurologic complications, including strokes, cognitive and speech impairment, visual loss, dementia, and encephalopathy. The purpose of this study is to establish a research cohort to investigate the disease process, treatments, and patient outcomes in CNSV.

Study Overview

• Status: Recruiting
• Conditions: Vasculitis; CNS Vasculitis; Central Nervous System Vasculitis; CNSV

Detailed Description

Primary central nervous system vasculitis (CNSV) is a potentially fatal, single-organ vasculitis that often involves a spectrum of neurologic complications, including strokes, cognitive and speech impairment, visual loss, dementia, and encephalopathy. Originally described in the early 1950s, research in the disease has been slow to progress, largely limited by the rarity of the disease and the lack of biologic materials for basic and translational investigations. To date, the bulk of research in the field has been clinical, largely consisting of retrospective case series, predominately from single sites, focusing on correlations among clinical, imaging, and histopathology data. Collectively these series suggest the existence of clinically-relevant subsets. Such studies, while enhancing our knowledge of this rare disease, are limited by small numbers of patients (especially those confirmed by histopathology), and the near absence of detailed investigations into the biomolecular and immunopathogenic basis of the disease.

Development of this longitudinal cohort and multicenter approach offers the potential to generate new insights, generate new questions, and further our understanding of its natural history, pathophysiology, genetic basis, and discovery of biomarkers to assess disease activity and prognosis.

Study visits will occur every 3 months for this first year, and then annually for two more years. Blood collection will occur at every visit. A physical exam and medical and medication history will at every visit; also, participants will be asked to complete several questionnaires to assess disease activity, health status, and tobacco, alcohol, and drug use.

• Study Type: Observational
• Enrollment (Estimated): 40

Contacts and Locations

• Study Contact: Name: Carol McAlear; Phone Number: 7813214567; Email: cmcalear@upenn.edu

Study Locations

• Canada
  • Hamilton, Canada
    • Recruiting
    • St. Joseph's Healthcare
    • Contact: Sandra Messier; Email: smessier@stjosham.on.ca
  • Toronto, Canada
    • Recruiting
    • University of Toronto/Sinai Health
    • Contact: Nazrana Haq; Email: Nazrana.Haq@sinaihealth.ca
• United States
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Recruiting
      • Mayo Clinic
      • Contact: Jenni Sletten; Email: Sletten.Jennifer@mayo.edu
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Recruiting
      • Cleveland Clinic
      • Contact: Brittany McKnight; Email: mcknigb@ccf.org
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • University of Pennsylvania
      • Contact: Jessica Nguyen; Email: Jessica.Nguyen2@PennMedicine.upenn.edu
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Recruiting
      • Vanderbilt University
      • Contact: Pamela Schlairet; Email: pamela.schlairet@vumc.org

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Individuals with central nervous system vasculitis (CNSV). Enrollment will be sequential and participants will have disease in various stages and of different duration.

Description

Inclusion Criteria:

i. Diagnostic criteria for CNSV

1. The presence of an acquired and otherwise unexplained neurologic deficit
2. The presence of either classic angiographic or histopathologic features of angiitis in the CNS
3. No evidence of systemic vasculitis or any condition that could cause the angiographic or pathologic features

ii. ≥18 years of age

Exclusion Criteria:

i. Unwilling to sign informed consent.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evaluate the pathogenesis of central nervous system vasculitis using disease history.	To study the manner of development of disease in patients affected with central nervous system vasculitis.	Study completion; from baseline through month 36.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
PROMIS Questionnaires	Questions will ask about Cognition function on the PROMIS short form.	Baseline, month 3, month 6, month 9, month 12, month 18, month 24, month 30, month 36
PROMIS Questionnaires	Questions will ask about depression on the PROMIS short form.	Baseline, month 3, month 6, month 9, month 12, month 18, month 24, month 30, month 36
PROMIS Questionnaires	Questions will ask about fatigue on the PROMIS short form.	Baseline, month 3, month 6, month 9, month 12, month 18, month 24, month 30, month 36
PROMIS Questionnaires	Questions will ask about Ability to participate in social roles and activities on the PROMIS short form.	Baseline, month 3, month 6, month 9, month 12, month 18, month 24, month 30, month 36
PROMIS Questionnaires	Questions will ask about Upper extremity function on the PROMIS short form.	Baseline, month 3, month 6, month 9, month 12, month 18, month 24, month 30, month 36
PROMIS Questionnaires	Questions will ask about lower extremity function on the PROMIS short form.	Baseline, month 3, month 6, month 9, month 12, month 18, month 24, month 30, month 36
PROMIS Questionnaires	Questions will ask about Global Health (physical) on the PROMIS short form.	Baseline, month 3, month 6, month 9, month 12, month 18, month 24, month 30, month 36
PROMIS Questionnaires	Questions will ask about Global Health (mental) on the PROMIS short form.	Baseline, month 3, month 6, month 9, month 12, month 18, month 24, month 30, month 36

Collaborators and Investigators

• Sponsor: University of Pennsylvania
• Collaborators: The Cleveland Clinic
• Investigators: Principal Investigator: Rula Hajj-Ali, MD, The Cleveland Clinic; Principal Investigator: Leonard Calabrese, MD, The Cleveland Clinic; Principal Investigator: Peter Merkel, MD, University of Pennsylvania

Study record dates

Study Major Dates

• Study Start (Actual): March 1, 2023
• Primary Completion (Estimated): December 31, 2030
• Study Completion (Estimated): December 31, 2030

Study Registration Dates

• First Submitted: February 8, 2023
• First Submitted That Met QC Criteria: February 8, 2023
• First Posted (Actual): February 21, 2023

Study Record Updates

• Last Update Posted (Actual): January 23, 2026
• Last Update Submitted That Met QC Criteria: January 21, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Vascular Diseases; Cardiovascular Diseases; Autoimmune Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Vasculitis; Vasculitis, Central Nervous System
• Other Study ID Numbers: VCRC5509

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No