Multimodality Cardiac Imaging for Disease Progression in ATTR-CM
Ориентир для пациента и семьи
Простыми словами
Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.
- Что изучают
- В протоколе указаны: FAPI tracer.
- Кому может быть актуально
- Состояния в реестре: Amyloidosis Cardiac. Базовые параметры: Без ограничений · Все.
- Что важно проверить
- Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
- Где проводится
- Швейцария
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
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Официальное название
Natural History of Cardiac Transthyretin Amyloidosis - Mechanistic Insights by Multimodality Imaging
Обзор
The goal of this clinical trial is to investigate whether new imaging techniques can help us to better understand the cardiac amyloidosis. The disease can be slowed down with various medications (e.g., tafamidis, acoramidis, or vutrisiran). However, treatment is not effective in all patients-in about one-third of cases, the disease continues to progress. So far, we know little about the exact causes of this and what biological changes occur in the heart muscle. The main question it aims to answer is: Will new imaging techniques help us understand the course of the cardiac amyloidosis? Participants will have additional examinations: * At the beginning of the study: one additional heart ultrasound examination, one cardiac MRI and one cardiac PET, blood examination during the regular examination, questionnaires. * After a year: one additional heart ultrasound examination, one cardiac MRI and one cardiac PET, blood examination during the regular examination. Time required: * Heart ultrasound examination: 5-10 Minutes * Cardiac MRI: 2 hours * Cardiac PET: 2 hours * Questionnaires: 5-10 Minutes.
Подробное описание
Experimental Design The study design is an open label prospective longitudinal study with serial imaging at baseline and after 12-month follow-up. The entire study population will include 50 participants with cardiac ATTR amyloidosis, as recently defined by multi-societal criteria.
Conventional markers of disease progression
These markers are collected during routine clinical follow-up of patients in our Outpatient Clinic. The markers are divided into three domains and include the following outcomes:
* Clinical and functional: any heart failure hospitalization, NYHA class, Kansas City Cardiomyopathy Questionnaire, 6-minute walk test; * Laboratory biomarkers: NT-proBNP, troponin, creatinine; * Imaging and ECG: LV wall thickness, diastolic dysfunction, LVEF, stroke volume, global longitudinal strain, conduction disturbance.
Data Analysis Plan Power calculation for sample size is challenging as no previous study has evaluated the response of our endpoints in myocardial FAPI uptake or serum BMP4 concentration to tafamidis. We therefore focused sample size calculation of echocardiographic myocardial stiffness. Based on our own preliminary data, we estimate median baseline values for myocardial stiffness of 2.6 m/s \[IQR, 1.7-3.8\] in cardiac amyloidosis. Disease progression is expected in about 30% of participants. While myocardial stiffness is expected to remain unchanged in cardiac amyloidosis without disease progression a 20% relative increase (i.e. 0.5 ± 0.5 m/s) is considered in cardiac amyloidosis with disease progression. A sample size of 40 participants (i.e. 30 participants without and 10 participants with disease progression) has a statistical power of 80% to predict a difference in response of myocardial stiffness to tafamidis.
Non-normally distributed, paired data of repeated measures (i.e. longitudinal changes per group) are compared by Wilcoxon signed rank test. Non-normally distributed unpaired data of changes (i.e. longitudinal changes between groups) are compared by Mann-Whitney test. The correlations are compared by a z-test on Fisher z-transformed correlation coefficients.
Вмешательства
- Диагностический тест FAPI tracer
18F-FAPI-74 PET/CT, cardiac MRI, echocardiography and blood sample for BMP5 serum concetration is performed at baseline and 12-month follow-up.
Первичные конечные точки
- Change in FAPI uptake [Срок оценки: Baseline and 1 year]
Вторичные конечные точки (3)
- Change in echocardiographic myocardial stiffness [Срок оценки: Baseline and 1 year]
- Change in extracellular volume (ECV) [Срок оценки: Baseline and 1 year]
- Change in BMP4 expression and serum concentration [Срок оценки: Baseline and 1 year]
Критерии участия
Критерии включения
- Participants with cardiac ATTR amyloidosis, as recently defined by multi-societal criteria, who are about to start tafamidis at the University Hospital Zurich
Критерии исключения
- Any other disease-modifying therapy (e.g. patisiran)
- Ongoing supraventricular arrhythmia
- Ventricular pacing
- Prior septal myocardial infarction
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Распределение
- Не применимо
- Модель
- Одна группа
- Маскирование
- Открытое
- Основная цель
- Диагностика
Центры проведения
Швейцария · 1 центр
- University Hospital Zurich — Zurich
Идентификаторы
NCT: NCT07112066 · 2024-02504