Morpho-functional Cardiac Modifications in Treated Mutated Transthyretin Cardiac Amyloidosis

Cardiac Amyloidosis Clinical Trial

Official title:

Cardiac Modifications in Transthyretin Cardiac Amyloidosis

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT04387344
• Other study ID #: UNIME_CARD_2
• Status: Not yet recruiting
• Start date: May 20, 2020
• Est. completion date: December 30, 2020

Study information

• Verified date: May 2020
• Source: University of Messina
• Contact: Francesco Cappelli, MD
• Phone: +39 055 794 9946
• Email: cappellifrancesco[@]inwind.it
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

Cardiac Amyloidosis (CA) is characterized by a long subclinical phase characterized by deposition of amyloid fibrils in atria, valves and walls of ventricles. Longitudinal dysfunction of the left ventricle (LV) with preserved ejection fraction (EF) is the early phase of CA.

Longitudinal dysfunction mainly involves the LV basal and middle segments with less involvement of the distal segments (apical sparing).

Strain echocardiography (STE) measures myocardial deformation. The technique has been shown to be sensitive for early detection of impaired systolic function and for the study of CA. Additionally, cardiac efficiency (myocardial work) can be derived from myocardial strain data analysis.

In the year 2018, "RNA interferences" (patisiran and inotersen) were included in the list of compassionate therapeutic use programs for exclusive use for the treatment of adult patients with hereditary amyloidosis neuropathy. The aim of our study is to evaluate the morpho-functional modifications with RNA interferences.

Description:

Transthoracic echocardiographic images are recorded in the available ultrasound units (Vivid 7 and Vivid 9, GE Ultrasound, Horten, Norways and MyLab - Esaote, Genoa, Italy). The diameters and the wall thicknesses are measured according to the insurance of the American Society of Echocardiography. The images obtained with apical view and in format 4 bedrooms with frame rates (70-80 frames / s) - stored for three cardiac cycles in cine-loop format - will be used for evaluate off-line to evaluate left ventricle deformation in the longitudinal and radial direction, basalelateral, and top segments (post processing) through software already in use and available (Suitestenza, Esaote, Florence, Italy and GE Ultrasound, Horten, Norway).

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 25
• Est. completion date: December 30, 2020
• Est. primary completion date: June 30, 2020
• Gender: All
• Age group: 18 Years to 90 Years

Eligibility

Inclusion Criteria:

• presence of data obtained from historical records of patients who retrospectively met the prescriptive (neurological) criteria and received the prescription of the drugs in question (RNA interferences) having

• Cardiological evaluation (examination ECG, echocardiography) at the time of prescription

• Cardiological evaluation (examination ECG, echocardiography) evaluated six months after therapy,

• Cardiological evaluation (examination ECG, echocardiography) evaluated 12-18 months after therapy,

Exclusion Criteria:

• it wasn't possible to putatively confirm the reasonable shipment of the innovative therapy prescribed

• it wasn't possible to putatively confirm the reasonable shipment of conventional therapy prescribed (background therapy)

Related Conditions & MeSH terms

• Amyloidosis
• Cardiac Amyloidosis

Locations

Country	Name	City	State
Italy	Careggi Hospital	Firenze
Italy	Fondazione Policlinico Universitario A. Gemelli IRCCS.	Roma

Sponsors (2)

Lead Sponsor	Collaborator
University of Messina	Campisi Mariapaola

Country where clinical trial is conducted

Italy, 

References & Publications (6)

Adams D, Gonzalez-Duarte A, O'Riordan WD, Yang CC, Ueda M, Kristen AV, Tournev I, Schmidt HH, Coelho T, Berk JL, Lin KP, Vita G, Attarian S, Planté-Bordeneuve V, Mezei MM, Campistol JM, Buades J, Brannagan TH 3rd, Kim BJ, Oh J, Parman Y, Sekijima Y, Hawki — View Citation

Benson MD, Waddington-Cruz M, Berk JL, Polydefkis M, Dyck PJ, Wang AK, Planté-Bordeneuve V, Barroso FA, Merlini G, Obici L, Scheinberg M, Brannagan TH 3rd, Litchy WJ, Whelan C, Drachman BM, Adams D, Heitner SB, Conceição I, Schmidt HH, Vita G, Campistol J — View Citation

Di Bella G, Minutoli F, Piaggi P, Casale M, Mazzeo A, Zito C, Oreto G, Baldari S, Vita G, Pingitore A, Khandheria BK, Carerj S. Usefulness of Combining Electrocardiographic and Echocardiographic Findings and Brain Natriuretic Peptide in Early Detection of — View Citation

Di Bella G, Minutoli F, Pingitore A, Zito C, Mazzeo A, Aquaro GD, Di Leo R, Recupero A, Stancanelli C, Baldari S, Vita G, Carerj S. Endocardial and epicardial deformations in cardiac amyloidosis and hypertrophic cardiomyopathy. Circ J. 2011;75(5):1200-8. — View Citation

Di Bella G, Pizzino F, Minutoli F, Zito C, Donato R, Dattilo G, Oreto G, Baldari S, Vita G, Khandheria BK, Carerj S. The mosaic of the cardiac amyloidosis diagnosis: role of imaging in subtypes and stages of the disease. Eur Heart J Cardiovasc Imaging. 20 — View Citation

Emdin M, Aimo A, Rapezzi C, Fontana M, Perfetto F, Seferovic PM, Barison A, Castiglione V, Vergaro G, Giannoni A, Passino C, Merlini G. Treatment of cardiac transthyretin amyloidosis: an update. Eur Heart J. 2019 Dec 1;40(45):3699-3706. doi: 10.1093/eurhe — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change of Atrial and Ventricular Walls Dimensions	Measurements (mm) of Interatrial Septum, Ventricular Walls, Coumadin Ridge, Mitro-aortic Lamina, Valves	Change from Baseline Wall Thickness to 18 months
Primary	Change of Atrial and Ventricular Strain	Mean paired change in myocardial strain (percent) by echocardiographic strain-rate imaging (unitless)	Change from Baseline Atrial and Ventricular Strain to 18 months
Secondary	Change of Myocardial Work index of Left Ventricle	Evaluation of the Global work index (GWI): total work within the area of the LV pressure-strain loops calculated from mitral valve closure to mitral valve opening.	Change from Baseline Myocardial work to 18 months