Screening of Fabry Disease in Portuguese Patients With Idiopathic Cardiomyopathies

NCT ID: NCT05409846

Last Updated: 2025-10-02

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

COMPLETED

Total Enrollment

409 participants

Study Classification

OBSERVATIONAL

Study Start Date

2022-04-15

Study Completion Date

2025-04-15

Brief Summary

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In Portugal, the prevalence of Fabry disease is largely unknown as recently has been stressed by the Portuguese hypertrophic cardiomyopathy registry investigators.

On the other hand, few data on Fabry screening protocols in patients with compromised ejection fraction including burned-out hypertrophic cardiomyopathy series have been published.

This project intends to perform screening of Fabry disease in patients with distinct cardiomyopathy phenotypes of unknown or dubious etiology and explore the less knew impact of the disease in other cardiac phenotypes.

Detailed Description

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Conditions

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Fabry Disease

Study Design

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Observational Model Type

OTHER

Study Time Perspective

CROSS_SECTIONAL

Study Groups

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Group A

Idiopathic hypertrophic cardiomyopathy

Alfa-galactosidase activity and genetic testing for Fabry diagnosis

Intervention Type DIAGNOSTIC_TEST

Dry blood spot analysis and blood sample (if necessary)

Group B

Idiopathic left ventricle hypertrophy

Alfa-galactosidase activity and genetic testing for Fabry diagnosis

Intervention Type DIAGNOSTIC_TEST

Dry blood spot analysis and blood sample (if necessary)

Group C

Idiopathic burned-out hypertrophic cardiomyopathy

Alfa-galactosidase activity and genetic testing for Fabry diagnosis

Intervention Type DIAGNOSTIC_TEST

Dry blood spot analysis and blood sample (if necessary)

Group D

Idiopathic dilated cardiomyopathy

Alfa-galactosidase activity and genetic testing for Fabry diagnosis

Intervention Type DIAGNOSTIC_TEST

Dry blood spot analysis and blood sample (if necessary)

Interventions

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Alfa-galactosidase activity and genetic testing for Fabry diagnosis

Dry blood spot analysis and blood sample (if necessary)

Intervention Type DIAGNOSTIC_TEST

Eligibility Criteria

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Inclusion Criteria

Patients with heart disease diagnosed after the age of 30:

* unexplained hypertrophic cardiomyopathy (Group A)
* unexplained left ventricle hypertrophy confirmed in two different examinations using the same or different imaging methods (Group B)
* unexplained burned-out hypertrophic cardiomyopathy (Group C)
* unexplained dilated cardiomyopathy with evidence of late gadolinium enhancement involving the basal posterolateral wall segments (Group D)

Exclusion Criteria

* previous exclusion of Fabry disease
* previous identified causal pathogenic/likely pathogenic genetic variant
* evidence of cardiomyopathy under the age of 30
Minimum Eligible Age

30 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Centro Hospitalar De São João, E.P.E.

OTHER

Sponsor Role collaborator

Hospital Pedro Hispano

OTHER

Sponsor Role collaborator

Centro Hospitalar do Tâmega e Sousa, Hospital Padre Américo

UNKNOWN

Sponsor Role collaborator

Hospital da Luz, Lisboa

UNKNOWN

Sponsor Role collaborator

Centro Hospitalar De Trás-Os-Montes E Alto Douro, E.P.E.

UNKNOWN

Sponsor Role collaborator

Centro Hospitalar Universitário Lisboa Norte, EPE., Hospital de Santa Maria

UNKNOWN

Sponsor Role collaborator

Centro Hospitalar de Entre Douro e Vouga, E.P.E., Hospital São Sebastião

UNKNOWN

Sponsor Role collaborator

Centro Hospitalar Universitário de Coimbra

UNKNOWN

Sponsor Role collaborator

Centro Hospitalar de Vila Nova de Gaia e Espinho, E.P.E.

UNKNOWN

Sponsor Role collaborator

Centro Hospitalar Universitário de Santo António

OTHER

Sponsor Role collaborator

Centro de Investigação em Tecnologias e Serviços de Saúde

OTHER

Sponsor Role collaborator

Rede de Investigação em Saúde

OTHER

Sponsor Role collaborator

Universidade do Porto

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Elisabete Martins, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Universidade do Porto

Locations

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Centro Hospitalar Universitário de Coimbra

Coimbra, , Portugal

Site Status

Hospital da Luz, Lisboa

Lisbon, , Portugal

Site Status

Centro Hospitalar Universitário Lisboa Norte, EPE., Hospital de Santa Maria

Lisbon, , Portugal

Site Status

Hospital Pedro Hispano (Unidade Local de Saúde Matosinhos)

Matosinhos Municipality, , Portugal

Site Status

Centro Hospitalar do Tâmega e Sousa, Hospital Padre Américo

Penafiel, , Portugal

Site Status

Centro Hospitalar Universitário de Santo António

Porto, , Portugal

Site Status

Centro Hospitalar Universitário São João, E.P.E.

Porto, , Portugal

Site Status

Faculty of Medicine (FMUP)

Porto, , Portugal

Site Status

Centro Hospitalar de Entre Douro e Vouga, E.P.E., Hospital São Sebastião

Santa Maria da Feira, , Portugal

Site Status

Centro Hospitalar de Vila Nova de Gaia e Espinho, E.P.E.

Vila Nova de Gaia, , Portugal

Site Status

Centro Hospitalar De Trás-Os-Montes E Alto Douro, E.P.E.

Vila Real, , Portugal

Site Status

Countries

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Portugal

Other Identifiers

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F-CHECK

Identifier Type: -

Identifier Source: org_study_id

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