Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Conditions
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.
Interventions
DRUGAcoramidis
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.
Sponsors
Bayer
Saving Lives Matters
Heart's Foundation
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Patient Reported Outcome Measure: Amylo-AFFECT-QOL score | At 3, 6, 9, and 12 months after initiation of acoramidis | Change from baseline in Amylo-AFFECT-QOL total score |
| Patient Reported Outcome Measure: KCCQ-23 score | At 3, 6, 9, and 12 months after initiation of acoramidis | Change from baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ-23) total score |
| Patient Reported Outcome Measure: EQ-5D-3L index score | At 3, 6, 9, and 12 months after initiation of acoramidis | 3L index score of EQ-5D Health-Related Quality of Life Questionnaire |
| Patient Reported Outcome Measure: EQ VAS score | At 3, 6, 9, and 12 months after initiation of acoramidis | Visual Analog scale score of EQ-5D Health-Related Quality of Life Questionnaire |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Demographic characteristics: Age | Before treatment with acoramidis | Age of patients initiating treatment with acoramidis |
| Demographic characteristics: Sex | Before treatment with acoramidis | Sex of patients initiating treatment with acoramidis |
| Demographic characteristics: Body Mass Index | Before treatment with acoramidis | Body Mass Index of patients initiating treatment with acoramidis |
| Clinical characteristics: Time between diagnosis and treatment initiation | Before treatment with acoramidis | Time between ATTR-CM diagnosis and treatment initiation |
| Clinical characteristics: ATTR-CM diagnosis | Before treatment with acoramidis | ATTR-CM diagnosis (genetic status including mutation type \[if applicable\], phenotype) |
| Clinical characteristics: ATTR-CM manifestations | Before treatment with acoramidis | ATTR-CM manifestations prior to treatment initiation |
| Clinical characteristics: Comorbidities | Up to 12 months before initiation of acoramidis, and during treatment with acoramidis | 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 |
| Clinical characteristics: Prior medication and procedures | Up to 12 months before initiation of acoramidis | Prior medication taken and procedures performed up to 12 months before initiation of acoramidis |
| Treatment patterns: Treatment duration | During treatment with acoramidis | Duration of treatment with acoramidis |
| Treatment patterns: Treatment interruptions | During treatment with acoramidis | Proportion of patients with temporary treatment interruptions and reason for interruption |
| Treatment patterns: Treatment discontinuation | During treatment with acoramidis | Proportion of patients with permanent treatment discontinuation and reason for discontinuation |
| Treatment patterns: Time to treatment discontinuation | During treatment with acoramidis | Time to acoramidis treatment discontinuation |
| Treatment patterns: Concomitant medications and procedures | During treatment with acoramidis | Concomitant medications taken and procedures performed during treatment with acoramidis |
| Safety and tolerability: Adverse events | During treatment with acoramidis and up to 6 days after the last intake of acoramidis in case of premature permanent treatment discontinuation | 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 |
Countries
France
Contacts
CONTACTBayer Clinical Trials Contact
Outcome results
None listed