Rationale and Design of ACO-REAL: A Real-World Non-Interventional Study of Acoramidis in Routine Clinical Practice

Area of Science:

• Cardiology
• Pharmacology
• Real-world evidence studies

Background:

• Acoramidis is an approved oral transthyretin stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM).
• Clinical trial data from ATTRibute-CM (NCT03860935) support acoramidis approval.
• The ACO-REAL study (NCT07235462) was designed to complement trial data with real-world evidence.

Purpose of the Study:

• To assess the demographics, disease characteristics, and treatment patterns of adults with ATTR-CM initiating acoramidis.
• To evaluate the safety profile and clinical course of acoramidis treatment in a real-world setting.
• To gather insights into ATTR-CM management within European healthcare systems.

Main Methods:

• Prospective, non-interventional, multicenter study involving approximately 2000 adults with ATTR-CM across 21 European countries.
• Observational period of around 12 months to assess treatment patterns, adherence, persistence, and modifications.
• Secondary objectives include evaluating safety, cardiac function, functional capacity, health status, and quality of life.

Main Results:

• This section is not applicable as the study is ongoing and results are not yet available.

Conclusions:

• ACO-REAL is the first multinational observational study collecting prospective, real-world data on acoramidis for ATTR-CM.
• The study aims to provide valuable real-world evidence to guide clinical decision-making in ATTR-CM management.
• Findings will offer insights into acoramidis use within diverse European healthcare settings.