Study of Acoramidis in Patients With Transthyretin Amyloid Cardiomyopathy

Bayer500 enrolled

Overview

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 Type

OBSERVATIONAL

Enrollment

500

Conditions

Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Interventions

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Outcomes

Primary Outcomes

Patient Reported Outcome Measure: Amylo-AFFECT-QOL score

Change from baseline in Amylo-AFFECT-QOL total score

Time frame: 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

Time frame: 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

Time frame: 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

Time frame: At 3, 6, 9, and 12 months after initiation of acoramidis