Longitudinal Study for Central Nervous System Vasculitis

University of Pennsylvania

Status: Enrolling

Conditions: CNSV; CNS Vasculitis; Vasculitis; Central Nervous System Vasculitis

Study type: Observational

Funder types: Other

Identifiers

NCT05734404

VCRC5509

Details and patient eligibility

About

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.

Full 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.

Enrollment: 40 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.