NEUROGENETIC AND HEMODYNAMIC OF MIGRAINE AURA AND PFO

NCT ID: NCT07349004

Last Updated: 2026-01-16

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

240 participants

Study Classification

OBSERVATIONAL

Study Start Date

2025-11-24

Study Completion Date

2028-11-30

Brief Summary

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The PFOCUS study aims to clarify the relationship between patent foramen ovale (PFO) and migraine with aura (MA). Patients diagnosed with MA according to ICHD-3 criteria will be classified as cases (PFO+) or controls (PFO-) based on the presence or absence of a right-to-left shunt on transcranial Doppler or prior documented PFO. The study will enroll 240 adult participants over 36 months.

The primary objective is to determine whether genetic variants differ significantly between MA patients with and without PFO. Secondary objectives include assessing cerebral blood flow regulation and platelet function in migraine with aura patients with and without PFO.

Participants will undergo a single study visit including collection of demographic and clinical data and a comprehensive neurosonological assessment. This includes microemboli count, cerebrovascular reactivity tests, and a contrast-enhanced Doppler study for shunt detection. Blood samples will be collected for whole-exome sequencing (focusing on NOTCH3 and an extended gene panel) and for platelet aggregometry.

The study is expected to provide new insights into biological mechanisms linking PFO and MA-potentially involving endothelial dysfunction, altered vascular smooth muscle responses, or platelet activation-thus informing prevention strategies and future research.

Detailed Description

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The PFOCUS study is a prospective, case-control observational investigation designed to explore the biological, vascular, and genetic factors associated with the coexistence of migraine with aura (MA) and patent foramen ovale (PFO). Epidemiological evidence indicates that individuals with MA have an increased risk of ischemic stroke even in the absence of traditional vascular risk factors. In parallel, a higher prevalence of PFO has been consistently reported in patients with MA, suggesting a potential role of PFO as a modifier of cerebrovascular risk. The mechanisms underlying this association are not fully understood and may involve factors beyond paradoxical embolism, including vascular dysfunction, altered cerebral hemodynamics, platelet activation, and genetic susceptibility.

The primary objective of the study is to compare the distribution of NOTCH3 gene variants and single nucleotide polymorphisms (SNPs) between MA patients with PFO (PFO+) and MA patients without PFO (PFO-). NOTCH3 encodes a receptor involved in vascular smooth muscle cell differentiation and small-vessel integrity, and pathogenic variants are known to cause cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy (CADASIL). Variants in this gene have also been associated with migraine phenotypes and alterations in cerebral small-vessel function. Given the role of Notch signaling pathways in cardiac septation and closure of the fetal foramen ovale, genetic variation in this pathway may contribute to both MA susceptibility and persistence of PFO.

Secondary objectives include: (1) the assessment of variants and SNPs in additional genes involved in Notch signaling, cardiac development, vascular regulation, and PFO heritability; (2) evaluation of the relationship between genetic variants and the degree of right-to-left shunt; (3) comparison of cerebral microembolic signals between PFO+ and PFO- participants using automated transcranial Doppler detection; (4) assessment of cerebrovascular reactivity (CVR) and dynamic cerebral autoregulation (dCA) using standardized neurosonological protocols; and (5) comparison of platelet function profiles between groups.

Each participant undergoes a single study visit. Data collection includes a structured clinical and demographic assessment, detailed characterization of migraine phenotype (including aura characteristics, attack frequency, and coexistence of migraine without aura), and review of available brain magnetic resonance imaging (MRI) findings when present. The neurosonological assessment includes continuous transcranial Doppler monitoring for detection of microembolic signals, evaluation of CVR and cerebral autoregulation using breath-hold and hyperventilation maneuvers with transfer function analysis, and contrast-enhanced right-to-left shunt detection using agitated saline. Peripheral blood samples are collected for genetic analysis and platelet function testing. Whole-exome sequencing is performed, with subsequent analysis focused on a predefined panel of genes, including but not limited to NOTCH3, NOTCH1, JAG1, and HTRA1. Platelet aggregation studies are conducted using standardized laboratory methods.

The planned sample size is 240 participants, equally distributed between the PFO+ and PFO- groups. This sample size is based on the expected prevalence of NOTCH3 SNPs and anticipated differences between groups. Statistical analyses include comparison of allele and genotype frequencies, assessment of Hardy-Weinberg equilibrium, linkage disequilibrium analyses, and multivariable comparisons of neurosonological and platelet function parameters.

By integrating genetic, neurosonological, and hematological data, the PFOCUS study aims to characterize potential biological differences between MA patients with and without PFO and to contribute to a better understanding of migraine-related vascular vulnerability.

Conditions

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Migraine With Aura

Study Design

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Observational Model Type

CASE_CONTROL

Study Time Perspective

PROSPECTIVE

Study Groups

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Arm 1

Patients with migraine with aura who have a patent foramen ovale (MAPFO+)

Intervention

Intervention Type OTHER

Neurosonological protocol with continuous Doppler monitoring for microemboli detection, cerebrovascular reactivity and dynamic cerebral autoregulation autoregulation testing, and a contrast-enhanced shunt study using agitated saline; blood samples for whole-exome sequencing and for platelet aggregation study

Arm 2

Patients with migraine with aura without patent foramen ovale (MAPFO-)

Intervention

Intervention Type OTHER

Neurosonological protocol with continuous Doppler monitoring for microemboli detection, cerebrovascular reactivity and dynamic cerebral autoregulation autoregulation testing, and a contrast-enhanced shunt study using agitated saline; blood samples for whole-exome sequencing and for platelet aggregation study

Interventions

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Intervention

Neurosonological protocol with continuous Doppler monitoring for microemboli detection, cerebrovascular reactivity and dynamic cerebral autoregulation autoregulation testing, and a contrast-enhanced shunt study using agitated saline; blood samples for whole-exome sequencing and for platelet aggregation study

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of migraine with aura (according to Chapter 1.2 of the International Classification of Headache Disorders, 3rd edition \[ICHD-3\])
* Age ≥ 18 years
* Both sexes
* Obtained written informed consent to participate in the study

Exclusion Criteria

* History of congenital heart disease
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Azienda Usl di Bologna

OTHER_GOV

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Matteo Paolucci

Role: PRINCIPAL_INVESTIGATOR

IRCCS Istituto delle Scienze Neurologiche di Bologna

Locations

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IRCCS Istituto delle Scienze Neurologiche di Bologna - AUSL of Bologna

Bologna, BO, Italy

Site Status RECRUITING

Countries

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Italy

Central Contacts

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Matteo Paolucci

Role: CONTACT

+390513172087

Andrea Zini

Role: CONTACT

+390516478810

Facility Contacts

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Matteo Paolucci

Role: primary

+390513172087

Andrea Zini

Role: backup

+390516478810

References

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Schmidt H, Zeginigg M, Wiltgen M, Freudenberger P, Petrovic K, Cavalieri M, Gider P, Enzinger C, Fornage M, Debette S, Rotter JI, Ikram MA, Launer LJ, Schmidt R; CHARGE consortium Neurology working group. Genetic variants of the NOTCH3 gene in the elderly and magnetic resonance imaging correlates of age-related cerebral small vessel disease. Brain. 2011 Nov;134(Pt 11):3384-97. doi: 10.1093/brain/awr252. Epub 2011 Oct 17.

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Rutten JW, Dauwerse HG, Gravesteijn G, van Belzen MJ, van der Grond J, Polke JM, Bernal-Quiros M, Lesnik Oberstein SA. Archetypal NOTCH3 mutations frequent in public exome: implications for CADASIL. Ann Clin Transl Neurol. 2016 Sep 28;3(11):844-853. doi: 10.1002/acn3.344. eCollection 2016 Nov.

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Reference Type BACKGROUND
PMID: 34946902 (View on PubMed)

Paolucci M, Altamura C, Vernieri F. The Role of Endothelial Dysfunction in the Pathophysiology and Cerebrovascular Effects of Migraine: A Narrative Review. J Clin Neurol. 2021 Apr;17(2):164-175. doi: 10.3988/jcn.2021.17.2.164.

Reference Type BACKGROUND
PMID: 33835736 (View on PubMed)

van Os HJA, Mulder IA, Broersen A, Algra A, van der Schaaf IC, Kappelle LJ, Velthuis BK, Terwindt GM, Schonewille WJ, Visser MC, Ferrari MD, van Walderveen MAA, Wermer MJH; DUST Investigators. Migraine and Cerebrovascular Atherosclerosis in Patients With Ischemic Stroke. Stroke. 2017 Jul;48(7):1973-1975. doi: 10.1161/STROKEAHA.116.016133. Epub 2017 May 19.

Reference Type BACKGROUND
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Reference Type BACKGROUND
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Headache Classification Committee of the International Headache Society (IHS) The International Classification of Headache Disorders, 3rd edition. Cephalalgia. 2018 Jan;38(1):1-211. doi: 10.1177/0333102417738202. No abstract available.

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Hosseini-Alghaderi S, Baron M. Notch3 in Development, Health and Disease. Biomolecules. 2020 Mar 23;10(3):485. doi: 10.3390/biom10030485.

Reference Type BACKGROUND
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Guo ZN, Xing Y, Liu J, Wang S, Yan S, Jin H, Yang Y. Compromised dynamic cerebral autoregulation in patients with a right-to-left shunt: a potential mechanism of migraine and cryptogenic stroke. PLoS One. 2014 Aug 14;9(8):e104849. doi: 10.1371/journal.pone.0104849. eCollection 2014.

Reference Type BACKGROUND
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Dong B, Li Y, Ai F, Geng J, Tang T, Peng W, Tang Y, Wang H, Tian Z, Bu F, Chen L. Genetic variation in patent foramen ovale: a case-control genome-wide association study. Front Genet. 2025 Jan 13;15:1523304. doi: 10.3389/fgene.2024.1523304. eCollection 2024.

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Elliott GC, Gurtu R, McCollum C, Newman WG, Wang T. Foramen ovale closure is a process of endothelial-to-mesenchymal transition leading to fibrosis. PLoS One. 2014 Sep 12;9(9):e107175. doi: 10.1371/journal.pone.0107175. eCollection 2014.

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Chabriat H, Joutel A, Dichgans M, Tournier-Lasserve E, Bousser MG. Cadasil. Lancet Neurol. 2009 Jul;8(7):643-53. doi: 10.1016/S1474-4422(09)70127-9.

Reference Type BACKGROUND
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Other Identifiers

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PFOCUS

Identifier Type: -

Identifier Source: org_study_id

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