Clinical Research Directory

A Study to Evaluate the Efficacy and Safety of Concomitant Use of Eplontersen and ALXN2220 Compared With Eplontersen and Placebo for Adults Participants With ATTR-CM

The purpose of this randomised, double-blind, placebo-controlled, multicenter study is to evaluate the efficacy and safety of concomitant use of eplontersen and ALXN2220 compared with eplontersen and placebo in adult participants with Transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).

A Study to Learn About the Use of Acoramidis in Patients With a Heart Condition Called Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in a Real-world Setting

Transthyretin Amyloid Cardiomyopathy (ATTR-CM) is a serious and life-threatening condition where a protein called transthyretin (TTR) misfolds and builds up as amyloid fibrils in the heart muscle. This buildup causes the heart to become stiff, leading to restrictive cardiomyopathy and progressive heart failure. There are two forms of ATTR-CM: a hereditary or 'variant' form (vATTR-CM) caused by a gene mutation, and a 'wild-type' form (wtATTR-CM) which is associated with aging. Because its symptoms can be similar to other heart conditions, ATTR-CM is often diagnosed late. However, recent advances in medical imaging are helping doctors to identify the disease earlier. Acoramidis is a new medication designed to treat ATTR-CM. It works by stabilizing the TTR protein, preventing it from misfolding and forming the harmful amyloid deposits. Acoramidis has been shown to be effective and safe in a major clinical trial (the ATTRibute-CM study), which led to its approval for use in both the United States and Europe. While clinical trials provide valuable information, data on how a new medicine performs in everyday clinical practice is also very important. This type of information is called real-world evidence. Currently, there is limited real-world information about the use of acoramidis. This study, called ACO-REAL, is an observational study, which means researchers will observe patients who are receiving acoramidis as part of their normal clinical care, without introducing any experimental interventions. The study will take place in approximately 20 European countries and aims to enroll up to 2,000 adults who have been diagnosed with either wild-type or variant ATTR-CM and are starting treatment with acoramidis. This includes patients who have not been treated for ATTR-CM before, as well as those who have been treated with other therapies. The main goals of the study are to understand the characteristics of patients being treated with acoramidis and to document how the treatment is used in routine medical practice. The study will also collect information on the safety of acoramidis. Furthermore, researchers will assess how the treatment affects patients' heart function, their functional capacity (such as their ability to walk), their overall health status, and their quality of life. The study will also track how often patients need to use healthcare resources like hospitals or emergency rooms. This information will help to improve the understanding and management of ATTR-CM in a real-world setting, ultimately aiming to optimize care for patients with this progressive disease.

Single-arm, Multi-center Study Evaluating the Acceptance and Feasibility of a Digital Remote Monitoring Tool Configured for Patients With ATTR-CM: ACO-Monitor

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a heart condition caused by a protein called transthyretin (TTR) building up as amyloid in the heart muscle. This build-up makes the heart stiff and can lead to symptoms of heart failure, such as difficulty breathing, tiredness, swelling in the legs, and reduced ability to be physically active. ATTR-CM occurs in two forms: a hereditary (variant) type caused by changes in genes, and a wild-type form that usually develops with aging. People living with ATTR-CM are usually cared for at specialist centers, which may require them to travel long distances for appointments. Because of this, visits may be infrequent, and sometimes people only see their doctor once a year. This can make it harder to notice if the disease is getting worse or if there are problems with following their treatment plan. As a result, important changes in health may go unnoticed for several months, which can be risky. Remote monitoring offers a way for people living with ATTR-CM and their healthcare professionals to stay connected between clinic visits. Recent studies have shown that remote monitoring can help by making it easier to share health information, spot problems earlier, and support people to follow their treatment plans. This can lead to fewer hospital visits and better health outcomes. Luscii is a digital remote monitoring tool designed to help people living with ATTR-CM and their healthcare professionals stay connected between clinic visits. Luscii has two main parts: Luscii contact, a smartphone app that allows secure messaging and video calls, and Luscii vitals, a clinical software system that organizes health data and can alert the healthcare team if there are changes that need attention. Luscii vitals is a CE-marked medical device used within its approved purpose and does not replace a doctor's judgement. By making it easier to communicate and share information, Luscii aims to support earlier detection of problems, better management of ATTR-CM, and a more positive experience for people living with this condition. The main purpose of this observational study, called ACO-MONITOR, is to learn how acceptable and practical Luscii is for people living with ATTR-CM and their healthcare professionals. The study will take place in Austria, Germany, and Italy at three specialist centers. About 60 adults with a diagnosis of hereditary or wild-type ATTR-CM will be invited to take part. There will be no treatments given as part of this study. Instead, participants will use the Luscii app and devices at home to track their health and share information with their healthcare professionals. The study team will provide training on how to use Luscii and will help participants get started. The study team will look at: * How quickly and how often participants and healthcare professionals use Luscii * How long Luscii is used by each person * How many times participants use Luscii after being reminded * How easy Luscii is to use, based on questionnaires for both participants and healthcare professionals The study will last about 18 months in total, including a 6-month period to invite participants and a 12-month follow-up period. During the study, the study team will: * Support participants in using Luscii to share health information from home * Monitor how often and how easily Luscii is used * Ask participants and healthcare professionals about their experiences using Luscii, including what worked well and what could be improved * Record any problems or technical issues with using Luscii By collecting this information, the researchers hope to learn if Luscii can help improve communication, support earlier detection of health problems, and make it easier for people living with ATTR-CM to manage their condition between clinic visits.

Thailand ATTR-CM Registry

The investigators of this registry aim to study the natural history, clinical presentation, characteristics, and imaging findings of patients diagnosed with ATTR amyloidosis in Thailand longitudinally. This will enable the investigators to study this disease in depth. With a better understanding, the investigators can aim to develop early screening programs for at-risk patients, raising awareness among non-cardiologists. As amyloid-specific therapies have now become available in Thailand, the findings of this registry can be helpful for epidemiological studies in Thailand.