Study of Acoramidis in Patients With Transthyretin Amyloid Cardiomyopathy
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorBayer
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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)
Disqualifiers
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 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.
Trial design
Design model
Other
Time perspective
Prospective
Treatments tested in this trial
Acoramidis
Drug356 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.
Treatment groups
500 Participants
are divided into 1 treatment groupGroup A: Single Arm1 intervention
Trial outcomes
Primary outcomes
1
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 acoramidis2
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 acoramidis3
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 acoramidis4
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 acoramidisSecondary outcomes
1
Demographic characteristics: Age
Age of patients initiating treatment with acoramidis
Time frame
Before treatment with acoramidis2
Demographic characteristics: Sex
Sex of patients initiating treatment with acoramidis
Time frame
Before treatment with acoramidis3
Demographic characteristics: Body Mass Index
Body Mass Index of patients initiating treatment with acoramidis
Time frame
Before treatment with acoramidis4
Clinical characteristics: Time between diagnosis and treatment initiation
Time between ATTR-CM diagnosis and treatment initiation
Time frame
Before treatment with acoramidisLocations
1Map coordinates are unavailable for these locations. Locations are shown below instead.
Many locationsRecruiting Many Locations
France
FranceSponsors and contacts
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Source ClinicalTrials.gov