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.
Studieoversikt
Status
Intervensjon / Behandling
Studietype
Registrering (Antatt)
Kontakter og plasseringer
Studiekontakt
- Navn: Bayer Clinical Trials Contact
- Telefonnummer: 4930300139003
- E-post: clinical-trials-contact@bayer.com
Studiesteder
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Many Locations, Frankrike
- Rekruttering
- Many locations
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Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
- Voksen
- Eldre voksen
Tar imot friske frivillige
Prøvetakingsmetode
Studiepopulasjon
Beskrivelse
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.
Studieplan
Hvordan er studiet utformet?
Designdetaljer
Kohorter og intervensjoner
Gruppe / Kohort |
Intervensjon / Behandling |
|---|---|
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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.
Andre navn:
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Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
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Patient Reported Outcome Measure: Amylo-AFFECT-QOL score
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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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Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
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Demographic characteristics: Age
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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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Samarbeidspartnere og etterforskere
Sponsor
Publikasjoner og nyttige lenker
Generelle publikasjoner
- 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.
Studierekorddatoer
Studer hoveddatoer
Studiestart (Faktiske)
Primær fullføring (Antatt)
Studiet fullført (Antatt)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Faktiske)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- 23181
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
IPD-planbeskrivelse
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.
Legemiddel- og utstyrsinformasjon, studiedokumenter
Studerer et amerikansk FDA-regulert medikamentprodukt
Studerer et amerikansk FDA-regulert enhetsprodukt
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