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
- Conditions
- Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
- Interventions
- Drug: Acoramidis (356 mg film-coated tablets)
- Registration Number
- NCT07235462
- Lead Sponsor
- Bayer
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 2000
- - Adults (≥18 years at the date of signing the informed consent form (ICF)).
- Diagnosis of either wild-type or variant ATTR-CM.
- Signed ICF.
- Decision to initiate treatment with acoramidis was made as per treating investigator's routine treatment practice before signature of ICF.
- Treatment start with acoramidis within 90 days after signing the ICF, with the possibility of starting acoramidis on the same day as signing the ICF.
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Participation in an investigational trial with interventions outside of routine clinical practice, except for participation in potential sub-studies related to this observational study. Please note: In addition to this observational study, separate sub-studies may be conducted to collect additional data. Participation in these sub-studies is voluntary and will be governed by separate protocols and informed consent processes. The main observational study does not include interventional procedures beyond routine clinical practice.
- Contra-indications according to the local SmPC of acoramidis.
- Patients who are unable to provide consent, including those whose consent would need to be given by a legal representative.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Acoramidis Arm Acoramidis (356 mg film-coated tablets) Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM) initiating treatment with acoramidis in routine clinical practice.
- Primary Outcome Measures
Name Time Method Patient demographic characteristics: age Baseline (Initial study visit) Demographic characteristics at the first documented regular visit in the study, referred to as the initial study visit.
Patient demographic characteristics: sex Baseline (Initial study visit) Demographic characteristics at the first documented regular visit in the study, referred to as the initial study visit.
Patient demographic characteristics: race Baseline (Initial study visit) Demographic characteristics at the first documented regular visit in the study, referred to as the initial study visit.
Patient demographic characteristics: height Baseline (Initial study visit) Demographic characteristics at the first documented regular visit in the study, referred to as the initial study visit.
Patient demographic characteristics: weight Baseline (Initial study visit) Demographic characteristics at the first documented regular visit in the study, referred to as the initial study visit.
Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Type Baseline up to 15 months ATTR-CM type: mixed phenotype (yes/no)
Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Genetic status Baseline up to 15 months ATTR-CM type: genetic status (mutation / wild type; if mutation: genotype and zygosity)
Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Diagnosis Baseline up to 15 months ATTR-CM diagnosis (year of diagnosis)
Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Setting of Diagnosis Baseline up to 15 months ATTR-CM setting of diagnosis: (endomyocardial biopsy
/ non-invasive / both, NYHA classification at diagnosis)Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Manifestations Baseline up to 15 months ATTR-CM manifestations (type)
Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) -relevant comorbidities Baseline up to 15 months ATTR-CM-relevant comorbidities (type)
Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) -relevant procedures Baseline up to 15 months Prior and concomitant ATTR-CM-relevant procedures (type)
Treatment Patterns with Acoramidis: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) -related medications Baseline (assessment within the past 12 months prior to initiation) Previously administered ATTR-CM-related medications within the past 12 months before initial visit (name)
Treatment Patterns with Acoramidis: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) concomitant medications Baseline up to 15 months Concomitant medications administered alongside acoramidis (name)
Treatment Patterns with Acoramidis: Initiation Baseline up to 15 months Acoramidis initiation (date)
Treatment Patterns with Acoramidis: Initiation after a different therapy Baseline up to 15 months Acoramidis initiation (if patient is switching from a different therapy: reason for switch)
Treatment Patterns with Acoramidis: discontinuation Baseline up to 15 months Acoramidis discontinuation (reason)
Treatment Patterns with Acoramidis: interruption Baseline up to 15 months Acoramidis interruption (reason)
Treatment Patterns with Acoramidis: prescription / refills Baseline up to 15 months Acoramidis prescriptions/refills since the last visit or telephone contact (duration of time from initiation to discontinuation of therapy)
- Secondary Outcome Measures
Name Time Method Incidence of Adverse Events From acoramidis initiation up to end of observation (approximately 12-15 months). Adverse events (AEs) documentation
Incidence of Serious Adverse Events From acoramidis initiation up to end of observation (approximately 12-15 months). Serious Adverse events (SAEs) documentation
Trial Locations
- Locations (1)
Universitaetsklinik Heidelberg
🇩🇪Heidelberg, Germany
Universitaetsklinik Heidelberg🇩🇪Heidelberg, Germany