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

Plain English Summary

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 is a Not Applicable clinical trial sponsored by Bayer studying Transthyretin Amyloid Cardiomyopathy (ATTR-CM). This study observes how a new heart medication, acoramidis, is used in everyday medical care for patients with ATTR-CM. It is for adults diagnosed with ATTR-CM, a condition where protein buildup stiffens the heart. Participation involves receiving acoramidis as prescribed by your doctor and having your health monitored. There are no experimental treatments; this study follows standard medical practice. The trial aims to enroll 2000 participants.

Official 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 treatm

Who Can Participate

Here is what you need to know about eligibility for this trial. Adults aged 18 and older diagnosed with ATTR-CM (either hereditary or age-related forms). Patients who are starting treatment with acoramidis as part of their regular care. Individuals must be able to give informed consent. Patients with contraindications to acoramidis or those participating in other experimental trials may not be eligible. This trial is studying Transthyretin Amyloid Cardiomyopathy (ATTR-CM), so participants generally need a confirmed diagnosis. The trial is currently accepting new participants.

What They're Measuring

The primary outcomes measure basic patient information like age, sex, height, and weight at the start of the study, helping to understand who is receiving the treatment. The specific primary outcome measures are: Patient demographic characteristics: age (Baseline (Initial study visit)); Patient demographic characteristics: sex (Baseline (Initial study visit)); Patient demographic characteristics: race (Baseline (Initial study visit)); Patient demographic characteristics: height (Baseline (Initial study visit)); Patient demographic characteristics: weight (Baseline (Initial study visit)). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This study does not have a traditional clinical phase designation. It may be an observational study that follows patients without intervening in their care, an expanded access or compassionate use program, or other non-interventional research. These studies contribute valuable data about disease progression, treatment patterns, and patient outcomes.

Why This Trial Matters

This trial matters because it will provide crucial information on how acoramidis works in real-world settings, helping to fill the gap between clinical trial results and everyday patient care for ATTR This research targets Transthyretin Amyloid Cardiomyopathy (ATTR-CM), where improved treatment options are needed.

Investor Insight

This observational study, sponsored by Bayer, aims to gather real-world evidence on acoramidis, a recently approved drug for ATTR-CM, indicating a focus on post-market validation and understanding its The large enrollment target of 2000 participants suggests significant investment in this program.

Is This Trial Right for Me?

Ask your doctor if acoramidis is the right treatment for you and what to expect. Your day-to-day involvement will be receiving acoramidis as prescribed and attending regular medical appointments for monitoring. Be prepared to share your health information and experiences with your doctor throughout the study. This trial is currently recruiting participants. The trial is being conducted at 1 site. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

• Study Type: OBSERVATIONAL
• Enrollment: 2,000 participants

Interventions

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

Primary Outcomes

• Patient demographic characteristics: age (Baseline (Initial study visit))
• Patient demographic characteristics: sex (Baseline (Initial study visit))
• Patient demographic characteristics: race (Baseline (Initial study visit))
• Patient demographic characteristics: height (Baseline (Initial study visit))
• Patient demographic characteristics: weight (Baseline (Initial study visit))

Secondary Outcomes

• 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).)

Full 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.

Trial Locations

• Universitaetsklinik Heidelberg, Heidelberg, Germany

Frequently Asked Questions

Related Conditions

• Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
• Cardiomyopathy
• Heart Failure
• Restrictive Cardiomyopathy
• Amyloidosis
• Hereditary Conditions
• Cardiovascular Diseases
• Protein Misfolding Diseases
• Geriatric Conditions
• Rare Diseases

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