Study of Acoramidis in Patients With Transthyretin Amyloid Cardiomyopathy (MOSAIC-TTR)
A Prospective, Longitudinal, Multi-center, Observational Study of Acoramidis In Patients With Wild-type or Variant Transthyretin Amyloid Cardiomyopathy (ATTR-CM): MOSAIC-TTR Study
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.
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
Type d'étude
Inscription (Estimé)
Contacts et emplacements
Coordonnées de l'étude
- Nom: Bayer Clinical Trials Contact
- Numéro de téléphone: 4930300139003
- E-mail: clinical-trials-contact@bayer.com
Lieux d'étude
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Many Locations, France
- Recrutement
- Many locations
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Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
- Adulte
- Adulte plus âgé
Accepte les volontaires sains
Méthode d'échantillonnage
Population étudiée
La description
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.
Plan d'étude
Comment l'étude est-elle conçue ?
Détails de conception
Cohortes et interventions
Groupe / Cohorte |
Intervention / Traitement |
|---|---|
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Single Arm
Patients will receive acoramidis 712 mg orally BID (twice daily)
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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.
Autres noms:
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Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
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Patient Reported Outcome Measure: Amylo-AFFECT-QOL score
Délai: At 3, 6, 9, and 12 months after initiation of acoramidis
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Change from baseline in Amylo-AFFECT-QOL total score
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At 3, 6, 9, and 12 months after initiation of acoramidis
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Patient Reported Outcome Measure: KCCQ-23 score
Délai: At 3, 6, 9, and 12 months after initiation of acoramidis
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Change from baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ-23) total score
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At 3, 6, 9, and 12 months after initiation of acoramidis
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Patient Reported Outcome Measure: EQ-5D-3L index score
Délai: At 3, 6, 9, and 12 months after initiation of acoramidis
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3L index score of EQ-5D Health-Related Quality of Life Questionnaire
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At 3, 6, 9, and 12 months after initiation of acoramidis
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Patient Reported Outcome Measure: EQ VAS score
Délai: At 3, 6, 9, and 12 months after initiation of acoramidis
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Visual Analog scale score of EQ-5D Health-Related Quality of Life Questionnaire
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At 3, 6, 9, and 12 months after initiation of acoramidis
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Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
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Demographic characteristics: Age
Délai: Before treatment with acoramidis
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Age of patients initiating treatment with acoramidis
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Before treatment with acoramidis
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Demographic characteristics: Sex
Délai: Before treatment with acoramidis
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Sex of patients initiating treatment with acoramidis
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Before treatment with acoramidis
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Demographic characteristics: Body Mass Index
Délai: Before treatment with acoramidis
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Body Mass Index of patients initiating treatment with acoramidis
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Before treatment with acoramidis
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Clinical characteristics: Time between diagnosis and treatment initiation
Délai: Before treatment with acoramidis
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Time between ATTR-CM diagnosis and treatment initiation
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Before treatment with acoramidis
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Clinical characteristics: ATTR-CM diagnosis
Délai: Before treatment with acoramidis
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ATTR-CM diagnosis (genetic status including mutation type [if applicable], phenotype)
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Before treatment with acoramidis
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Clinical characteristics: ATTR-CM manifestations
Délai: Before treatment with acoramidis
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ATTR-CM manifestations prior to treatment initiation
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Before treatment with acoramidis
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Clinical characteristics: Comorbidities
Délai: Up to 12 months before initiation of acoramidis, and during treatment with acoramidis
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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
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Up to 12 months before initiation of acoramidis, and during treatment with acoramidis
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Clinical characteristics: Prior medication and procedures
Délai: Up to 12 months before initiation of acoramidis
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Prior medication taken and procedures performed up to 12 months before initiation of acoramidis
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Up to 12 months before initiation of acoramidis
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Treatment patterns: Treatment duration
Délai: During treatment with acoramidis
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Duration of treatment with acoramidis
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During treatment with acoramidis
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Treatment patterns: Treatment interruptions
Délai: During treatment with acoramidis
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Proportion of patients with temporary treatment interruptions and reason for interruption
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During treatment with acoramidis
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Treatment patterns: Treatment discontinuation
Délai: During treatment with acoramidis
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Proportion of patients with permanent treatment discontinuation and reason for discontinuation
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During treatment with acoramidis
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Treatment patterns: Time to treatment discontinuation
Délai: During treatment with acoramidis
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Time to acoramidis treatment discontinuation
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During treatment with acoramidis
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Treatment patterns: Concomitant medications and procedures
Délai: During treatment with acoramidis
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Concomitant medications taken and procedures performed during treatment with acoramidis
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During treatment with acoramidis
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Safety and tolerability: Adverse events
Délai: During treatment with acoramidis and up to 6 days after the last intake of acoramidis in case of premature permanent treatment discontinuation
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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
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During treatment with acoramidis and up to 6 days after the last intake of acoramidis in case of premature permanent treatment discontinuation
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Collaborateurs et enquêteurs
Parrainer
Publications et liens utiles
Publications générales
- 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.
- 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.
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude (Réel)
Achèvement primaire (Estimé)
Achèvement de l'étude (Estimé)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Réel)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Termes MeSH pertinents supplémentaires
Autres numéros d'identification d'étude
- 23181
Plan pour les données individuelles des participants (IPD)
Prévoyez-vous de partager les données individuelles des participants (DPI) ?
Description du régime IPD
Currently, there is no established plan for the sharing of Individual Patient Data (IPD) from this study. The availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA 'Principles for responsible clinical trial data sharing.' This pertains to the scope, timepoint, and process of data access.
As such, Bayer commits to considering requests from qualified researchers for patient- / study-level clinical trial data, and documents from clinical trials involving medicines and indications approved in the US and EU. However, this commitment does not reflect an active IPD sharing plan. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.
Researchers can use www.vivli.org to request access to IPD and documents from clinical studies to conduct research. Information on Bayer's criteria for listing studies is provided in the member section of the portal.
Informations sur les médicaments et les dispositifs, documents d'étude
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