Study of Acoramidis in Patients With Transthyretin Amyloid Cardiomyopathy

NCT07791160 | Not Yet Recruiting

• 1 other identifier: 23181
• Study Type: observational
• Target: 500
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (14)

• Demographic characteristics: Age

  Before treatment with acoramidis

• Demographic characteristics: Sex

  Before treatment with acoramidis

• Demographic characteristics: Body Mass Index

  Before treatment with acoramidis

• Clinical characteristics: Time between diagnosis and treatment initiation

  Before treatment with acoramidis

• Clinical characteristics: ATTR-CM diagnosis

  Before treatment with acoramidis

Study Arms (1)

Single Arm

Patients will receive acoramidis 712 mg orally BID (twice daily)

Drug: Acoramidis

Interventions

Acoramidis DRUG

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.

Also known as: BAY3684938, BEYONTTRA

Single 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

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.

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Bayer: lead
• Saving Lives Matters: collaborator
• Heart's Foundation: collaborator

Related Publications (2)

• 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.

  PMID: 38197816 BACKGROUND

• 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.

  PMID: 41139783 BACKGROUND

Related Links

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

MeSH Terms

Interventions

attruby

Central Study Contacts

Bayer Clinical Trials Contact

CONTACT

4930300139003; clinical-trials-contact@bayer.com

Study Design

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

Study Record Dates

• First Submitted: August 25, 2026
• First Posted: August 27, 2026
• Study Start (Estimated): September 1, 2026
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): December 31, 2028
• Last Updated: August 27, 2026

Record last verified: 2026-04-23

Data Sharing

• IPD Sharing: Will not share