Not Yet Recruiting

Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Bayer Identifier:

23181

ClinicalTrials.gov Identifier:

Not Available

EudraCT Number:

Not Available

EU CT Number:

Not Available

Study of Acoramidis in Patients with Transthyretin Amyloid Cardiomyopathy

Trial purpose

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.

Key Participants Requirements

Sex

All

Age

18 - N/A (No Limit)

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.

Trial summary

Enrollment Goal

500

Trial Dates

September 2026 - December 2028

Phase

Phase IV

Could I Receive a placebo

Products

Acoramidis, BAY 3684938

Accepts Healthy Volunteer

Primary Outcome

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

Secondary Outcome

Demographic characteristics: Age
Age of patients initiating treatment with acoramidis
Time Frame:
Before treatment with acoramidis
Demographic characteristics: Sex
Sex of patients initiating treatment with acoramidis
Time Frame:
Before treatment with acoramidis
Demographic characteristics: Body Mass Index
Body Mass Index of patients initiating treatment with acoramidis
Time Frame:
Before treatment with acoramidis
Clinical characteristics: Time between diagnosis and treatment initiation
Time between ATTR-CM diagnosis and treatment initiation
Time Frame:
Before treatment with acoramidis
Clinical characteristics: ATTR-CM diagnosis
ATTR-CM diagnosis (genetic status including mutation type [if applicable], phenotype)
Time Frame:
Before treatment with acoramidis
Clinical characteristics: ATTR-CM manifestations
ATTR-CM manifestations prior to treatment initiation
Time Frame:
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
Time Frame:
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
Time Frame:
Up to 12 months before initiation of acoramidis
Treatment patterns: Treatment duration
Duration of treatment with acoramidis
Time Frame:
During treatment with acoramidis
Treatment patterns: Treatment interruptions
Proportion of patients with temporary treatment interruptions and reason for interruption
Time Frame:
During treatment with acoramidis
Treatment patterns: Treatment discontinuation
Proportion of patients with permanent treatment discontinuation and reason for discontinuation
Time Frame:
During treatment with acoramidis
Treatment patterns: Time to treatment discontinuation
Time to acoramidis treatment discontinuation
Time Frame:
During treatment with acoramidis
Treatment patterns: Concomitant medications and procedures
Concomitant medications taken and procedures performed during treatment with acoramidis
Time Frame:
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
Time Frame:
During treatment with acoramidis and up to 6 days after the last intake of acoramidis in case of premature permanent treatment discontinuation

Trial design

A prospective, longitudinal, Multi-center, Observational Study of Acoramidis In patients with wild-type or variant transthyretin amyloid Cardiomyopathy (ATTR-CM):

Trial Type

Observational

Intervention Type

Drug

Trial Purpose

N/A

Allocation

N/A

Blinding

Open Label

Assignment

N/A

Trial Arms

N/A

Additional Information

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