Clinical trial NCT06960538: Enpowering Progression Risk of Cerebral Amyloid Angiopathy
Official title: PRIORITY (Enpowering Progression Risk of Cerebral Amyloid Angiopathy)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Cerebral Amyloid Angiopathy
CAA - Cerebral Amyloid Angiopathy
Keywords: Cerebral Amyloid Angiopathy
CAA
plasma biomarkers
cerebrospinal fluid biomarkers
Lead sponsor: Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Sponsor class: OTHER
First posted: 2025-05-07
Enrollment: 200
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: Patients with possible or probable CAA identified on brain MRI according to modified Boston Neuroradiological Criteria 2.0 regardless of age.

Brief summary: Cerebral amyloid angiopathy (CAA) is a microangiopathy characterized by the progressive deposition of β-amyloid in cerebral vessel walls, contributing to intracerebral hemorrhages, cognitive decline, and other clinical manifestations. Despite recent advances in diagnosis and understanding, many pathogenic, prognostic, and therapeutic aspects remain unclear.

Study Objective:

PRIORITY is a prospective observational study aimed at identifying clinical, neuroradiological, and biochemical biomarkers that could improve early diagnosis, risk stratification, and the identification of personalized therapeutic targets for CAA.

Detailed description: PRIORITY is a prospective, single-center observational study conducted at the Fondazione IRCCS Istituto Neurologico Carlo Besta in Milan. It will consecutively enroll patients over 18 years of age with possible or probable cerebral amyloid angiopathy (CAA), symptomatic or asymptomatic, with or without histological confirmation. Diagnosis will follow the updated Boston criteria 2.0, and a brain MRI is mandatory for inclusion.

The study duration is 36 months, with clinical and neuroimaging assessments at baseline (T0), 12 months (T1), and 24 months (T2). CSF analysis will be performed at T0; plasma biomarkers (via ELISA and SIMOA) will be assessed at all time points. Lipid profiles will be analyzed using mass spectrometry with both untargeted and targeted lipidomic approaches (e.g., sphingolipidomics).

The comprehensive clinical and biological dataset will be used to develop a machine learning-based predictive model to support diagnostic, prognostic, and therapeutic decision-making in CAAThe study duration is 36 months, with clinical and neuroimaging assessments at baseline (T0), 12 months (T1), and 24 months (T2). CSF analysis will be performed at T0; plasma biomarkers (via ELISA and SIMOA) will be assessed at all time points. Lipid profiles will be analyzed using mass spectrometry with both untargeted and targeted lipidomic approaches (e.g., sphingolipidomics).

Eligibility criteria: Inclusion Criteria:

* patients of either sex over 18 years of age;
* patients with possible and probable symptomatic or asymptomatic CAA with or without histological demonstration (modified Boston criteria);
* patients who have had at least one brain MRI.

Exclusion Criteria:

* patients who have contraindications to undergoing brain MRI (e.g., pacemaker, incompatible mechanical valves, claustrophobia);
* patients who have contraindications to or refuse to undergo lumbar puncture;
* patients who are unable to provide informed consent for the study due to aphasic or cognitive impairment;
* patients who are pregnant or breastfeeding.

Primary outcomes:
measure: Clinical Progression of CAA
timeFrame: Baseline (T0), 12 months (T1), 24 months (T2).
measure: Radiological Progression of CAA
timeFrame: Baseline (T0), 12 months (T1), 24 months (T2).
measure: Identification of Protein and Lipid Biomarkers
timeFrame: Baseline (T0), 12 months (T1), 24 months (T2).
Secondary outcomes:
measure: Cognitive Decline Assessment
timeFrame: Baseline (T0), 12 months (T1), 24 months (T2).
measure: Development of a Predictive Model for Disease Progression
timeFrame: 24 months (T2).
measure: Hemorrhagic and Non-Hemorrhagic Event Incidence
timeFrame: 24 months (T2)
measure: Therapeutic Target Identification
timeFrame: 24 months (T2)
measure: Functional assessment
timeFrame: Baseline (T0), 12 months (T1), 24 months (T2)
Other outcomes:
Locations:
city: Milano
country: Italy
facility: Fondazione IRCCS Istituto Neurologico Carlo Besta
Study officials:
References:
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