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Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Bayer Identifier:
23023
ClinicalTrials.gov Identifier:
NCT07235462
EudraCT Number:
Not Available
EU CT Number:
Not Available
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

Trial purpose

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.

Key Participants Requirements

Sex

All

Age

18 - N/A
    - - 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.
    - 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.

Trial summary

Enrollment Goal
2000
Trial Dates
October 2025 - July 2028
Phase
Phase 4
Could I Receive a placebo
No
Products
Acoramidis (BAY3684938)
Accepts Healthy Volunteer
No

Where to participate

StatusInstitutionLocation
Recruiting
Universitaetsklinik HeidelbergHeidelberg, 69120, Germany

Primary Outcome

  • Patient demographic characteristics: age
    Demographic characteristics at the first documented regular visit in the study, referred to as the initial study visit.
    date_rangeTime Frame:
    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.
    date_rangeTime Frame:
    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.
    date_rangeTime Frame:
    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.
    date_rangeTime Frame:
    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.
    date_rangeTime Frame:
    Baseline (Initial study visit)
  • Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Type
    ATTR-CM type: mixed phenotype (yes/no)
    date_rangeTime Frame:
    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)
    date_rangeTime Frame:
    Baseline up to 15 months
  • Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Diagnosis
    ATTR-CM diagnosis (year of diagnosis)
    date_rangeTime Frame:
    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)
    date_rangeTime Frame:
    Baseline up to 15 months
  • Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Manifestations
    ATTR-CM manifestations (type)
    date_rangeTime Frame:
    Baseline up to 15 months
  • Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) -relevant comorbidities
    ATTR-CM-relevant comorbidities (type)
    date_rangeTime Frame:
    Baseline up to 15 months
  • Clinical Characteristics: Transthyretin Amyloid Cardiomyopathy (ATTR-CM) -relevant procedures
    Prior and concomitant ATTR-CM-relevant procedures (type)
    date_rangeTime Frame:
    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)
    date_rangeTime Frame:
    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)
    date_rangeTime Frame:
    Baseline up to 15 months
  • Treatment Patterns with Acoramidis: Initiation
    Acoramidis initiation (date)
    date_rangeTime Frame:
    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)
    date_rangeTime Frame:
    Baseline up to 15 months
  • Treatment Patterns with Acoramidis: discontinuation
    Acoramidis discontinuation (reason)
    date_rangeTime Frame:
    Baseline up to 15 months
  • Treatment Patterns with Acoramidis: interruption
    Acoramidis interruption (reason)
    date_rangeTime Frame:
    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)
    date_rangeTime Frame:
    Baseline up to 15 months

Secondary Outcome

  • Incidence of Adverse Events
    Adverse events (AEs) documentation
    date_rangeTime Frame:
    From acoramidis initiation up to end of observation (approximately 12-15 months).
  • Incidence of Serious Adverse Events
    Serious Adverse events (SAEs) documentation
    date_rangeTime Frame:
    From acoramidis initiation up to end of observation (approximately 12-15 months).

Trial design

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
Trial Type
Observational
Intervention Type
Drug
Trial Purpose
Treatment
Allocation
N/A
Blinding
N/A
Assignment
N/A
Trial Arms
N/A

Additional Information

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