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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT04893889
Other study ID # 4963/19/182 FABRY
Secondary ID
Status Recruiting
Phase N/A
First received
Last updated
Start date September 9, 2020
Est. completion date March 30, 2022

Study information

Verified date April 2021
Source University of Sao Paulo General Hospital
Contact Fabio Fernandes, MD, PhD
Phone +551126615057
Email fabio.fernandes@incor.usp.br
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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.


Recruitment information / eligibility

Status Recruiting
Enrollment 60
Est. completion date March 30, 2022
Est. primary completion date August 31, 2021
Accepts healthy volunteers Accepts Healthy Volunteers
Gender All
Age group 18 Years to 85 Years
Eligibility 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).

Study Design


Related Conditions & MeSH terms


Intervention

Diagnostic Test:
Shear-wave elastography
Myocardial shear wave elastography to assess myocardial stiffness by non-invasively quantifying diastolic myocardial elasticity.

Locations

Country Name City State
Brazil Instituto do Coração - Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo São Paulo Sao Paulo

Sponsors (2)

Lead Sponsor Collaborator
University of Sao Paulo General Hospital Pfizer

Country where clinical trial is conducted

Brazil, 

Outcome

Type Measure Description Time frame Safety issue
Primary Myocardial stiffness 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. 7 months
Secondary Systolic and diastolic function 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; 7 months
Secondary Brain Natriuretic Peptide and troponin assessment To correlate diastolic myocardial elasticity with a 6-minute walk test, Brain Natriuretic Peptide and troponin. 7 months
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