Study of Acoramidis in Patients With Transthyretin Amyloid Cardiomyopathy (MOSAIC-TTR)

A Prospective, Longitudinal, Multi-center, Observational Study of Acoramidis In Patients With Wild-type or Variant Transthyretin Amyloid Cardiomyopathy (ATTR-CM): MOSAIC-TTR Study

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive heart disease caused by the buildup of an abnormal protein, called transthyretin (TTR), in the heart. This buildup can make it harder for the heart to pump blood and may lead to worsening symptoms over time. Acoramidis is a medicine approved for the treatment of adults with wild-type or hereditary (variant) ATTR-CM. While its benefits have been demonstrated in clinical trials, more information is needed about how it is used and how patients do in everyday medical practice.

The MOSAIC-TTR study is an observational study in France. Participants will receive acoramidis as part of their usual medical care. No experimental treatments or additional medical procedures will be required. The study will collect information directly from participating hospitals and from the Healthcare European Amyloidosis Registry (HEAR; NCT05101304). The main goal of the study is to understand how patients' quality of life, daily functioning, and overall well-being change during the first 12 months of treatment with acoramidis, using questionnaires completed by the patients themselves. The study will also collect information about the characteristics of patients receiving acoramidis, how the medicine is used in routine clinical practice, and its safety and tolerability. The information collected will help improve the understanding of ATTR-CM and may help improve the care of people living with this condition.

Study Overview

• Status: Not yet recruiting
• Conditions: Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
• Intervention / Treatment: Drug: Acoramidis

• Study Type: Observational
• Enrollment (Estimated): 500

Contacts and Locations

• Study Contact: Name: Bayer Clinical Trials Contact; Phone Number: 4930300139003; Email: clinical-trials-contact@bayer.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Participants will be recruited from centers that are part of the French Amyloidosis Network or centers that are knowledgeable in the diagnosis and management of transthyretin amyloid cardiomyopathy (ATTR-CM). There are two complementary enrollment pathways: (i) direct recruitment within this study, and (ii) integration of data from the Healthcare Amyloidosis European Registry (HEAR), a multicenter registry maintained by reference centers (see NCT05101304), to enrich the study database.

Description

Inclusion Criteria:

• Greater than or equal to 18 years of age
• Established diagnosis of ATTR-CM with either wild-type TTR or a variant TTR genotype
• Initiating treatment with acoramidis with the decision to treat having been made by the treating physician prior to study enrolment in accordance with the current French Summary of Product Characteristics (SmPC)

Exclusion Criteria:

• Any contraindications as listed in the local approved product information
• Prior treatment with disease-modifying treatment (e.g., tafamidis, TTR silencers and/or depleters)
• Participation in an investigational trial evaluating new Investigational Medicinal Products or new Investigational Medical Device outside of routine clinical practice
• Under legal protection (guardianship, curatorship, or other legal protection measures) or deprived of liberty by administrative or judicial decision at the time of inclusion.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Single Arm; Patients will receive acoramidis 712 mg orally BID (twice daily)	Drug: Acoramidis; 356 mg film-coated tablets. The recommended dose is 712 mg (two tables, 356 mg) orally twice daily, corresponding to a total daily dose of 1424 mg.; Other Names: BEYONTTRA; BAY3684938

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Patient Reported Outcome Measure: Amylo-AFFECT-QOL score	Change from baseline in Amylo-AFFECT-QOL total score	At 3, 6, 9, and 12 months after initiation of acoramidis
Patient Reported Outcome Measure: KCCQ-23 score	Change from baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ-23) total score	At 3, 6, 9, and 12 months after initiation of acoramidis
Patient Reported Outcome Measure: EQ-5D-3L index score	3L index score of EQ-5D Health-Related Quality of Life Questionnaire	At 3, 6, 9, and 12 months after initiation of acoramidis
Patient Reported Outcome Measure: EQ VAS score	Visual Analog scale score of EQ-5D Health-Related Quality of Life Questionnaire	At 3, 6, 9, and 12 months after initiation of acoramidis

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Demographic characteristics: Age	Age of patients initiating treatment with acoramidis	Before treatment with acoramidis
Demographic characteristics: Sex	Sex of patients initiating treatment with acoramidis	Before treatment with acoramidis
Demographic characteristics: Body Mass Index	Body Mass Index of patients initiating treatment with acoramidis	Before treatment with acoramidis
Clinical characteristics: Time between diagnosis and treatment initiation	Time between ATTR-CM diagnosis and treatment initiation	Before treatment with acoramidis
Clinical characteristics: ATTR-CM diagnosis	ATTR-CM diagnosis (genetic status including mutation type [if applicable], phenotype)	Before treatment with acoramidis
Clinical characteristics: ATTR-CM manifestations	ATTR-CM manifestations prior to treatment initiation	Before treatment with acoramidis
Clinical characteristics: Comorbidities	ATTR-CM-relevant comorbidities that were either ongoing at treatment initiation, or were previously managed or resolved in the 12 months prior to initiating acoramidis, as well as those that began after initiating acoramidis	Up to 12 months before initiation of acoramidis, and during treatment with acoramidis
Clinical characteristics: Prior medication and procedures	Prior medication taken and procedures performed up to 12 months before initiation of acoramidis	Up to 12 months before initiation of acoramidis
Treatment patterns: Treatment duration	Duration of treatment with acoramidis	During treatment with acoramidis
Treatment patterns: Treatment interruptions	Proportion of patients with temporary treatment interruptions and reason for interruption	During treatment with acoramidis
Treatment patterns: Treatment discontinuation	Proportion of patients with permanent treatment discontinuation and reason for discontinuation	During treatment with acoramidis
Treatment patterns: Time to treatment discontinuation	Time to acoramidis treatment discontinuation	During treatment with acoramidis
Treatment patterns: Concomitant medications and procedures	Concomitant medications taken and procedures performed during treatment with acoramidis	During treatment with acoramidis
Safety and tolerability: Adverse events	Adverse events (AEs) occurring during treatment with acoramidis and AEs occurring up to 6 days after the last intake of acoramidis if premature permanent treatment discontinuation	During treatment with acoramidis and up to 6 days after the last intake of acoramidis in case of premature permanent treatment discontinuation

Collaborators and Investigators

• Sponsor: Bayer
• Collaborators: Saving Lives Matters; Heart's Foundation

Publications and helpful links

General Publications

• Gillmore JD, Judge DP, Cappelli F, Fontana M, Garcia-Pavia P, Gibbs S, Grogan M, Hanna M, Hoffman J, Masri A, Maurer MS, Nativi-Nicolau J, Obici L, Poulsen SH, Rockhold F, Shah KB, Soman P, Garg J, Chiswell K, Xu H, Cao X, Lystig T, Sinha U, Fox JC; ATTRibute-CM Investigators. Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy. N Engl J Med. 2024 Jan 11;390(2):132-142. doi: 10.1056/NEJMoa2305434.
• Reant P, Kharoubi M, Donal E, Bauer F, Bezard M, Bisson A, Bodez D, Bouchot O, Cariou E, Charron P, Costa J, Courand PY, Dagrenat C, Delelis F, Duval AJ, Eicher JC, Fraix A, Gellen B, Gueffet JP, Guijarro D, Habib G, Hagege A, Huttin O, Jaccard A, Jeanneteau J, Legallois D, Logeart D, Legrand L, Inamo J, Marguerit L, Mirailles R, Pezel T, Piriou N, Roubille F, Mouhat B, Tresorier R, Von Hunolstein JJ, Taieb C, Salvat M, Zaroui A, Lairez O, Damy T. The Healthcare Amyloidosis European Registry (HEAR): design of a national registry with a European extension strategy, and foundation of the F-CRIN GRACE network. Orphanet J Rare Dis. 2025 Oct 27;20(1):534. doi: 10.1186/s13023-025-04062-y.

Helpful Links

• HEART's Foundation
• Réseau Amylose [French]
• Healthcare European Amyloidosis Registry on ClinicalTrials.gov

Study record dates

Study Major Dates

• Study Start (Estimated): September 1, 2026
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): December 31, 2028

Study Registration Dates

• First Submitted: August 25, 2026
• First Submitted That Met QC Criteria: August 25, 2026
• First Posted (Actual): August 27, 2026

Study Record Updates

• Last Update Posted (Actual): August 27, 2026
• Last Update Submitted That Met QC Criteria: August 25, 2026
• Last Verified: April 23, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: attruby
• Other Study ID Numbers: 23181

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

IPD Plan Description

Currently, there is no established plan for the sharing of Individual Patient Data (IPD) from this study. The availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA 'Principles for responsible clinical trial data sharing.' This pertains to the scope, timepoint, and process of data access.

As such, Bayer commits to considering requests from qualified researchers for patient- / study-level clinical trial data, and documents from clinical trials involving medicines and indications approved in the US and EU. However, this commitment does not reflect an active IPD sharing plan. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.

Researchers can use www.vivli.org to request access to IPD and documents from clinical studies to conduct research. Information on Bayer's criteria for listing studies is provided in the member section of the portal.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No