NCT05409846

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
409

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Apr 2022

Typical duration for all trials

Geographic Reach
1 country

11 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 15, 2022

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

June 3, 2022

Completed
5 days until next milestone

First Posted

Study publicly available on registry

June 8, 2022

Completed
2.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2025

Completed
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

April 15, 2025

Completed
Last Updated

October 2, 2025

Status Verified

September 1, 2025

Enrollment Period

2.7 years

First QC Date

June 3, 2022

Last Update Submit

September 26, 2025

Conditions

Keywords

Idiopathic cardiomyopathies

Outcome Measures

Primary Outcomes (1)

  • Frequency of Fabry Disease in patients with idiopathic cardiomyopathies

    Ratio of number of patients with Fabry Disease and total number of idiopathic cardiomyopathies patients

    12 months

Secondary Outcomes (1)

  • Familiar screening of Fabry Disease

    12 months

Study Arms (4)

Group A

Idiopathic hypertrophic cardiomyopathy

Diagnostic Test: Alfa-galactosidase activity and genetic testing for Fabry diagnosis

Group B

Idiopathic left ventricle hypertrophy

Diagnostic Test: Alfa-galactosidase activity and genetic testing for Fabry diagnosis

Group C

Idiopathic burned-out hypertrophic cardiomyopathy

Diagnostic Test: Alfa-galactosidase activity and genetic testing for Fabry diagnosis

Group D

Idiopathic dilated cardiomyopathy

Diagnostic Test: Alfa-galactosidase activity and genetic testing for Fabry diagnosis

Interventions

Dry blood spot analysis and blood sample (if necessary)

Group AGroup BGroup CGroup D

Eligibility Criteria

Age30 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Idiopathic cardiomyopathy patients

You may qualify if:

  • 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)

You may not qualify if:

  • previous identified causal pathogenic/likely pathogenic genetic variant
  • evidence of cardiomyopathy under the age of 30

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (11)

Centro Hospitalar Universitário de Coimbra

Coimbra, 3000-602, Portugal

Location

Hospital da Luz, Lisboa

Lisbon, 1500-650, Portugal

Location

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

Lisbon, 1600-190, Portugal

Location

Hospital Pedro Hispano (Unidade Local de Saúde Matosinhos)

Matosinhos Municipality, 4464-513, Portugal

Location

Centro Hospitalar do Tâmega e Sousa, Hospital Padre Américo

Penafiel, 4564-007, Portugal

Location

Centro Hospitalar Universitário de Santo António

Porto, 4099-001, Portugal

Location

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

Porto, 4200-319, Portugal

Location

Faculty of Medicine (FMUP)

Porto, 4200-319, Portugal

Location

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

Santa Maria da Feira, 4520-220, Portugal

Location

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

Vila Nova de Gaia, 4434-502, Portugal

Location

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

Vila Real, 5000-508, Portugal

Location

Biospecimen

Retention: SAMPLES WITH DNA

Dry blood spot

MeSH Terms

Conditions

Fabry Disease

Interventions

Genetic Testing

Condition Hierarchy (Ancestors)

SphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesCerebral Small Vessel DiseasesCerebrovascular DisordersVascular DiseasesCardiovascular DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolism, Inborn ErrorsLipidosesLipid Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism Disorders

Intervention Hierarchy (Ancestors)

Clinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisInvestigative TechniquesGenetic TechniquesGenetic ServicesHealth ServicesHealth Care Facilities Workforce and ServicesDiagnostic ServicesPreventive Health Services

Study Officials

  • Elisabete Martins, MD, PhD

    Universidade do Porto

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
OTHER
Time Perspective
CROSS SECTIONAL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 3, 2022

First Posted

June 8, 2022

Study Start

April 15, 2022

Primary Completion

January 1, 2025

Study Completion

April 15, 2025

Last Updated

October 2, 2025

Record last verified: 2025-09

Locations