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- Essai clinique NCT04893889
Substudy (NCT04456582): Noninvasive Assessment of Myocardial Stiffness by 2D-SWE Ultrasound Technique (Two-dimensional Shear Wave Elastography) in Patients With Amyloidosis and Fabry Disease. (FABRY)
Introduction: Heart failure with preserved systolic function encompasses several different diseases, but which have diastolic dysfunction and its components in common: myocardial stiffness and altered relaxation. Myocardial stiffness represents an important parameter for diagnosis and prognosis, but only changes in relaxation are evaluated in clinical practice. Cardiac elastography has been proposed as a diagnostic modality for noninvasive assessment of myocardial stiffness.
Objective: The aim of our study is to investigate the potential of myocardial elastography by shear waves to assess myocardial stiffness by non-invasively quantifying diastolic myocardial elasticity (EMD) in Fabry disease (DF) and cardiac amyloidosis (AC ) in the ATTRh form and correlate with other complementary imaging and laboratory tests (electrocardiogram, 2D echocardiogram, troponin and BNP) and with a 6-minute walk test and quality of life questionnaires.
Material and methods: 60 adults will be prospectively included: 20 patients with Fabry disease, 40 patients with hRTRT (20 with cardiac involvement) and 20 patients as a control group. Echocardiography, electrocardiogram and laboratory evaluations will be performed. The elastocardiographic assessment of myocardial stiffness will be performed in ultrasound equipment (Canon, Aplio i800) using a multifrequency convex transducer, under specific adjustment of the equipment to perform myocardial elastography.
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
Type d'étude
Inscription (Anticipé)
Phase
- N'est pas applicable
Contacts et emplacements
Coordonnées de l'étude
- Nom: Caio R Cafezeiro, MD
- Numéro de téléphone: +551126615057
- E-mail: caiocafezeiro@usp.br
Lieux d'étude
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Sao Paulo
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São Paulo, Sao Paulo, Brésil, 05403-900
- Recrutement
- Instituto do Coração - Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
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Contact:
- Caio R Cafezeiro, MD
- Numéro de téléphone: +551126615057
- E-mail: caiocafezeiro@usp.br
-
-
Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
Accepte les volontaires sains
Sexes éligibles pour l'étude
La description
Inclusion Criteria:
- Documented diagnosis of ATTRh with and without cardiac involvement.
For the definition of cardiac involvement by ATTRh, all the criteria below must be presented:
- Pathogenic mutation of TTR consistent with ATTRh.
- Evidence of cardiac commitment by echocardiography or nuclear magnetic resonance with measurement of the interventricular septum at the end of diastole (SIVd)> 12mm or / and medical history of heart failure, and / or levels of troponin and / or BNP above the reference value without another more likely cause.
- Amyloid deposit in cardiac or extra-cardiac tissue (eg, abdominal fat aspirate, salivary gland, connective sheath of the median nerve) confirmed by congo red staining or presence of grade 2 or 3 myocardial scintigraphy (DPD-CT) of cardiac uptake, in cases where the presence of monoclonal gammopathy of undetermined significance (MGUS) was ruled out.
- In the presence of MGUS, confirmation of the TTR protein in the tissue is necessary through immunohistochemistry (IHC) or mass spectrometry.
Exclusion Criteria:
- Presence of another type of cardiomyopathy such as hypertension, valve or ischemic heart disease (eg, previous myocardial infarction documented with myocardial necrosis markers and electrocardiographic changes).
- Presence of diseases other than cardiac amyloidosis, impairing the assessment of functional capacity, such as chronic obstructive pulmonary disease, severe arthritis or peripheral arterial disease, recent or planned orthopedic procedure during the course of the study (eg, spine surgery or under lower limbs) that impairs walking in the 6-minute test evaluation.
- Acute coronary heart disease or unstable angina in the past 3 months Report of disease of the sinus or atrioventricular node with indication of a pacemaker, but with no intention of implantation.
- Presence of untreated hypothyroidism or hyperthyroidism.
- Previous heart, liver or other organ transplant.
- Presence of neoplasia in the last 3 years, except for basal and squamous cell carcinomas of skin or cervical cancer in situ previously treated.
- Presence of other medical conditions or comorbidities that, in the investigator's opinion, could interfere with the course of the study or the interpretation of the data.
- Pregnancy.
- History of alcohol abuse in the last 2 years or excessive daily alcohol intake (for women, more than 14 units per week; for men, more than 21 units of alcohol per week [unit: one glass of wine (125ml) = one dose of distillate = 332.5ml of beer).
Plan d'étude
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Diagnostique
- Répartition: Non randomisé
- Modèle interventionnel: Affectation parallèle
- Masquage: Seul
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
|---|---|
|
Expérimental: Fabry disease patients
Patients diagnosed with Fabry disease.
Interventions will be as follows: Shear wave myocardial elastography to assess myocardial stiffness by non-invasively quantifying diastolic myocardial elasticity (EMD) in Fabry disease (DF) and cardiac amyloidosis (AC) in the ATTRh form and correlating with other complementary imaging tests and laboratory tests (electrocardiogram, 2D echocardiogram, troponin and BNP) and with a 6-minute walk test and quality of life questionnaires.
|
Myocardial shear wave elastography to assess myocardial stiffness by non-invasively quantifying diastolic myocardial elasticity.
|
|
Comparateur actif: Amyloidosis transthyretin with cardiac commitment
Patients diagnosed with amyloidosis who present cardiac commitment.
Interventions will be as follows: Shear wave myocardial elastography to assess myocardial stiffness by non-invasively quantifying diastolic myocardial elasticity (EMD) in Fabry disease (DF) and cardiac amyloidosis (AC) in the ATTRh form and correlating with other complementary imaging tests and laboratory tests (electrocardiogram, 2D echocardiogram, troponin and BNP) and with a 6-minute walk test and quality of life questionnaires.
|
Myocardial shear wave elastography to assess myocardial stiffness by non-invasively quantifying diastolic myocardial elasticity.
|
|
Comparateur placebo: Healthy subjects
Healthy patients.
Interventions will be as follows: Shear wave myocardial elastography to assess myocardial stiffness by non-invasively quantifying diastolic myocardial elasticity (EMD) in Fabry disease (DF) and cardiac amyloidosis (AC) in the ATTRh form and correlating with other complementary imaging tests and laboratory tests (electrocardiogram, 2D echocardiogram, troponin and BNP) and with a 6-minute walk test.
|
Myocardial shear wave elastography to assess myocardial stiffness by non-invasively quantifying diastolic myocardial elasticity.
|
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
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Myocardial stiffness
Délai: 7 months
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To evaluate the diastolic myocardial elasticity in patients with Fabry's disease and compare it with the ATTR group with cardiac involvement and the control group.
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7 months
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Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Systolic and diastolic function
Délai: 7 months
|
To correlate diastolic myocardial elasticity with systolic and diastolic function indexes assessed by two-dimensional echocardiogram and with strain analysis using the speckle tracking technique;
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7 months
|
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Brain Natriuretic Peptide and troponin assessment
Délai: 7 months
|
To correlate diastolic myocardial elasticity with a 6-minute walk test, Brain Natriuretic Peptide and troponin.
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7 months
|
Collaborateurs et enquêteurs
Parrainer
Collaborateurs
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude (Réel)
Achèvement primaire (Anticipé)
Achèvement de l'étude (Anticipé)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Réel)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Termes MeSH pertinents supplémentaires
- Maladies cardiovasculaires
- Maladies vasculaires
- Maladies métaboliques
- Troubles cérébrovasculaires
- Maladies du cerveau
- Maladies du système nerveux central
- Maladies du système nerveux
- Maladies génétiques, innées
- Maladies génétiques liées à l'X
- Déficits de protéostase
- Métabolisme, erreurs innées
- Maladies de surcharge lysosomale
- Troubles du métabolisme lipidique
- Maladies cérébrales métaboliques
- Maladies cérébrales, métaboliques, innées
- Sphingolipidoses
- Maladies de surcharge lysosomale, système nerveux
- Maladies des petits vaisseaux cérébraux
- Lipidoses
- Métabolisme des lipides, erreurs innées
- Amylose
- Maladie de Fabry
Autres numéros d'identification d'étude
- 4963/19/182 FABRY
Plan pour les données individuelles des participants (IPD)
Prévoyez-vous de partager les données individuelles des participants (DPI) ?
Informations sur les médicaments et les dispositifs, documents d'étude
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