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

NCT07235462 | Recruiting

• 1 other identifier: 23023
• Study Type: observational
• Target: 2,000
• Locations: 1 country
• Sites: 1

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.

Conditions

Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Keywords

Transthyretin Amyloidosis; Cardiomyopathy; Heart Failure; Wild-type Amyloidosis

Outcome Measures

Primary Outcomes (19)

• Patient demographic characteristics: age

  Demographic characteristics at the first documented regular visit in the study, referred to as the initial study visit.

  Baseline (Initial study visit)

• Patient demographic characteristics: sex

  Demographic characteristics at the first documented regular visit in the study, referred to as the initial study visit.

  Baseline (Initial study visit)

• Patient demographic characteristics: race

  Demographic characteristics at the first documented regular visit in the study, referred to as the initial study visit.

  Baseline (Initial study visit)

• Patient demographic characteristics: height

  Demographic characteristics at the first documented regular visit in the study, referred to as the initial study visit.

  Baseline (Initial study visit)

• Patient demographic characteristics: weight

  Demographic characteristics at the first documented regular visit in the study, referred to as the initial study visit.

  Baseline (Initial study visit)

• Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Type

  ATTR-CM type: mixed phenotype (yes/no)

  Baseline up to 15 months

• Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Genetic status

  ATTR-CM type: genetic status (mutation / wild type; if mutation: genotype and zygosity)

  Baseline up to 15 months

• Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Diagnosis

  ATTR-CM diagnosis (year of diagnosis)

  Baseline up to 15 months

• Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Setting of Diagnosis

  ATTR-CM setting of diagnosis: (endomyocardial biopsy / non-invasive / both, NYHA classification at diagnosis)

  Baseline up to 15 months

• Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Manifestations

  ATTR-CM manifestations (type)

  Baseline up to 15 months

• Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) -relevant comorbidities

  ATTR-CM-relevant comorbidities (type)

  Baseline up to 15 months

• Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) -relevant procedures

  Prior and concomitant ATTR-CM-relevant procedures (type)

  Baseline up to 15 months

• Treatment Patterns with Acoramidis: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) -related medications

  Previously administered ATTR-CM-related medications within the past 12 months before initial visit (name)

  Baseline (assessment within the past 12 months prior to initiation)

• Treatment Patterns with Acoramidis: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) concomitant medications

  Concomitant medications administered alongside acoramidis (name)

  Baseline up to 15 months

• Treatment Patterns with Acoramidis: Initiation

  Acoramidis initiation (date)

  Baseline up to 15 months

• Treatment Patterns with Acoramidis: Initiation after a different therapy

  Acoramidis initiation (if patient is switching from a different therapy: reason for switch)

  Baseline up to 15 months

• Treatment Patterns with Acoramidis: discontinuation

  Acoramidis discontinuation (reason)

  Baseline up to 15 months

• Treatment Patterns with Acoramidis: interruption

  Acoramidis interruption (reason)

  Baseline up to 15 months

• Treatment Patterns with Acoramidis: prescription / refills

  Acoramidis prescriptions/refills since the last visit or telephone contact (duration of time from initiation to discontinuation of therapy)

  Baseline up to 15 months

Secondary Outcomes (2)

• Incidence of Adverse Events

  From acoramidis initiation up to end of observation (approximately 12-15 months).

• Incidence of Serious Adverse Events

  From acoramidis initiation up to end of observation (approximately 12-15 months).

Study Arms (1)

Acoramidis Arm

Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM) initiating treatment with acoramidis in routine clinical practice.

Drug: Acoramidis (356 mg film-coated tablets)

Interventions

Acoramidis (356 mg film-coated tablets) DRUG

Follow clinical practice/administration.

Also known as: BEYONTTRA

Acoramidis Arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with an established diagnosis of either wild-type or variant Transthyretin Amyloid Cardiomyopathy (ATTR-CM) who are eligible for treatment with acoramidis based on the clinical decision of the investigator, made independently of the study.

You may qualify if:

• \- 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Bayer: lead

Study Sites (1)

Universitaetsklinik Heidelberg

Heidelberg, 69120, Germany

RECRUITING

MeSH Terms

Conditions

Amyloidosis, Hereditary, Transthyretin-Related; Cardiomyopathies; Heart Failure

Interventions

attruby

Condition Hierarchy (Ancestors)

Heart Diseases; Cardiovascular Diseases

Central Study Contacts

Bayer Clinical Trials Contact

CONTACT

(+)1-888-84 22937; clinical-trials-contact@bayer.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 6, 2025
• First Posted: November 19, 2025
• Study Start: October 29, 2025
• Primary Completion (Estimated): April 30, 2028
• Study Completion (Estimated): July 3, 2028
• Last Updated: March 31, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1)