Clinical trial NCT05734404: Longitudinal Study for Central Nervous System Vasculitis
Official title: Longitudinal Study for Central Nervous System Vasculitis
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Central Nervous System Vasculitis
CNS Vasculitis
CNSV
Vasculitis
Lead sponsor: University of Pennsylvania
Sponsor class: OTHER
Collaborators: name: The Cleveland Clinic
First posted: 2023-02-21
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

Brief summary: 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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Evaluate the pathogenesis of central nervous system vasculitis using disease history.
timeFrame: Study completion; from baseline through month 36.
Secondary outcomes:
measure: PROMIS Questionnaires
timeFrame: Baseline, month 3, month 6, month 9, month 12, month 18, month 24, month 30, month 36
measure: PROMIS Questionnaires
timeFrame: Baseline, month 3, month 6, month 9, month 12, month 18, month 24, month 30, month 36
measure: PROMIS Questionnaires
timeFrame: Baseline, month 3, month 6, month 9, month 12, month 18, month 24, month 30, month 36
measure: PROMIS Questionnaires
timeFrame: Baseline, month 3, month 6, month 9, month 12, month 18, month 24, month 30, month 36
measure: PROMIS Questionnaires
timeFrame: Baseline, month 3, month 6, month 9, month 12, month 18, month 24, month 30, month 36
measure: PROMIS Questionnaires
timeFrame: Baseline, month 3, month 6, month 9, month 12, month 18, month 24, month 30, month 36
measure: PROMIS Questionnaires
timeFrame: Baseline, month 3, month 6, month 9, month 12, month 18, month 24, month 30, month 36
measure: PROMIS Questionnaires
timeFrame: Baseline, month 3, month 6, month 9, month 12, month 18, month 24, month 30, month 36
Other outcomes:
Locations:
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University
state: Tennessee
city: Hamilton
country: Canada
facility: St. Joseph's Healthcare
city: Toronto
country: Canada
facility: University of Toronto/Sinai Health
Study officials:
affiliation: The Cleveland Clinic
name: Rula Hajj-Ali, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: The Cleveland Clinic
name: Leonard Calabrese, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Pennsylvania
name: Peter Merkel, MD
role: PRINCIPAL_INVESTIGATOR
References: