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

Official title

ACO-REAL - A Non-interventional Study Providing Insights Into the Use of Acoramidis in Patients With ATTR Amyloidosis With Cardiomyopathy (ATTR-CM) in Routine Clinical Practice

Overview

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.

Interventions

• Drug Acoramidis (356 mg film-coated tablets)
  Follow clinical practice/administration.

Primary outcome measures

• Patient demographic characteristics: age [Time frame: Baseline (Initial study visit)]
• Patient demographic characteristics: sex [Time frame: Baseline (Initial study visit)]
• Patient demographic characteristics: race [Time frame: Baseline (Initial study visit)]
• Patient demographic characteristics: height [Time frame: Baseline (Initial study visit)]
• Patient demographic characteristics: weight [Time frame: Baseline (Initial study visit)]
• Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Type [Time frame: Baseline up to 15 months]
• Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Genetic status [Time frame: Baseline up to 15 months]
• Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Diagnosis [Time frame: Baseline up to 15 months]
• Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Setting of Diagnosis [Time frame: Baseline up to 15 months]
• Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Manifestations [Time frame: Baseline up to 15 months]

Secondary outcome measures (2)

• Incidence of Adverse Events [Time frame: From acoramidis initiation up to end of observation (approximately 12-15 months).]
• Incidence of Serious Adverse Events [Time frame: From acoramidis initiation up to end of observation (approximately 12-15 months).]

Eligibility criteria

Inclusion criteria

• \- 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.

Exclusion criteria

• 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Cohort

Study locations

Germany · 1 center

• Universitaetsklinik Heidelberg — Heidelberg

Publications

• Fontana M, Cappelli F, Gillmore JD, Obici L, Siepen FAD, Debonnaire P, Gonzalez-Lopez E, Schulze M, Vogtlander K, Ciaccia A, Merz M, Garcia-Pavia P. Rationale and Design of ACO-REAL: A Real-World Non-Interventional Study of Acoramidis in Routine Clinical Practice. Cardiol Ther. 2026 Jul 31. doi: 10.1007/s40119-026-00461-9. Online ahead of print. PMID 42533192

Identifiers

NCT: NCT07235462 · 23023

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗