Clinical trial • Phase IV • Cardiology

ACORAMIDIS for Transthyretin amyloid cardiomyopathy (ATTR-CM)

Phase IV trial of ACORAMIDIS for Transthyretin amyloid cardiomyopathy (ATTR-CM). open-label. 62 participants.

Overview

Trial Therapeutic Area
Cardiology
Trial Disease
Transthyretin amyloid cardiomyopathy (ATTR-CM)
Trial Stage
Phase IV
Drug Modality
Small molecule

Key dates

Initial CTIS Submission Date
13-10-2025
First CTIS Authorization Date
07-02-2026

Trial design

open-label Phase IV trial across 17 sites in Austria, Germany, Italy.

Open Label
Yes
Target Sample Size
62
Trial Duration For Participant
178

Eligibility

Recruits 62 Vulnerable population selected (populationOfTrialSubjects.isVulnerablePopulationSelected = true). No further details on consent/assent procedures for vulnerable participants are available in the provided data..

Vulnerable Population
Vulnerable population selected (populationOfTrialSubjects.isVulnerablePopulationSelected = true). No further details on consent/assent procedures for vulnerable participants are available in the provided data.

Inclusion criteria

  • {"criterion_text":"- Participant must be 18 to 90 years of age inclusive, at the time of signing the informed consent.\n- Diagnosis of ATTR-CM; disease defining examination, i.e., SPECT or SPECT/CT or biopsy, within 24 months prior to V1\n- Participants must currently be treated with tafamidis and have used tafamidis for at least the previous 3 months prior to V1 and have adhered to tafamidis therapy\n- NYHA class ≤ II at V1\n- eGFR ≥ 30 mL/min/1.73m2 at V1\n- NT-proBNP > 300 and ≤ 7000 pg/mL at V1"}

Exclusion criteria

  • {"criterion_text":"- Prior liver or heart transplantation or planned within the next 12 months\n- Initiation of treatment with a diuretic or increase in diuretic dose within 3 months before V1\n- Treatment with calcium channel blockers (e.g., verapamil, diltiazem) or digitalis required\n- Recent CV hospitalization within 3 months before V1\n- Known hypersensitivity to acoramidis or to any of the excipients\n- A condition that, as judged by the investigator, would preclude compliance with the study protocol, such as a history of substance abuse, alcoholism, or a psychiatric condition\n- Known or suspected liver disorder and bile secretion/flow (cholestasis, also history of it)\n- Abnormal liver function tests at V1, defined as ALT (GPT) or AST (GOT) ≥ 3 x ULN or total bilirubin ≥ 3 x ULN at V1\n- Current or planned use of ventricular assist device\n- Active cancer or other disease that decreases the life expectancy to less than one year\n- Heart failure due to ischemic heart disease\n- Myocardial infarction, CV surgery, or unstable angina within the last 90 days prior to V1\n- Confirmed diagnosis of light-chain amyloidosis\n- Dialysis or severe renal impairment as reflected by UACR > 300 mg/g at V1\n- Major surgery 90 days prior to V1\n- Recent initiation of SGLT2i within 3 months before V1"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Change in serum TTR level from baseline to month 6 or premature discontinuation of treatment","definition_or_measurement_approach":""}

Secondary endpoints

  • {"endpoint_text":"- Serum TTR level at baseline, week 1, 2, 3, 4 and at month 3.","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Occurrence of TEAEs and type of TEAEs and serious TEAEs.","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Change from baseline to month 6 of NT-proBNP, hs-TnT, hs-CRP, RBP4.","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Signs of remodeling from baseline to month 6 in ECHO.","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Change from baseline to month 6 in 6MWD","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Change from baseline to month 6 in kidney and thyroid biomarkers: eGFR, Creatinine, Cystatin C, UACR, and TSH.","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Change from baseline to month 6 in KCCQ-OS score and in EQ-5D-5L index score.","definition_or_measurement_approach":""}

Recruitment

Planned Sample Size
62
Recruitment Window Months
12
Consent Approach
Informed consent required from the participant (adults aged 18–90). Subject information and informed consent form documents are listed for Germany, Austria and Italy (L1_SIS_and_ICF_Core_Public_DE_DE; L1_SIS_and_ICF_Core_Public_AT_DE; L1_SIS_and_ICF_Core_Public_IT_IT) and expecting-parents female ICFs are present. No assent procedures are listed (participants must be ≥18).

Geography

Total Number Of Sites
17
Total Number Of Participants
62

Austria

Earliest CTIS Part Ii Submission Date
09-01-2026
Latest Decision Or Authorization Date
11-05-2026
Processing Time Days
122
Number Of Sites
5
Number Of Participants
32

Sites

Site Name
Medical University Of Graz
Department Name
Klinische Abteilung für Kardiologie
Principal Investigator Name
Nicolas Verheyen
Principal Investigator Email
nicolas.verheyen@medunigraz.at
Contact Person Name
Nicolas Verheyen
Contact Person Email
nicolas.verheyen@medunigraz.at
Site Name
Medical University Of Vienna
Department Name
Klinische Abteilung für Kardiologie
Principal Investigator Name
Andreas Kammerlander
Principal Investigator Email
andreas.kammerlander@meduniwien.ac.at
Contact Person Name
Andreas Kammerlander
Site Name
Klinik Favoriten
Department Name
Klinische Abteilung für Kardiologie
Principal Investigator Name
Diana Bonderman
Principal Investigator Email
diana.bonderman@gesundheitsverbund.at
Contact Person Name
Diana Bonderman
Site Name
Stadt Wien Wiener Gesundheitsverbund
Department Name
Medizinische Abteilung mit Kardiologie, internistischer Intensivmedizin und Ambulanz
Principal Investigator Name
Stephan Dobner
Principal Investigator Email
stephan.dobner@gesundheitsverbund.at
Contact Person Name
Stephan Dobner
Site Name
A.O. Krankenhaus St. Josef Braunau GmbH
Department Name
Innere Medizin I
Principal Investigator Name
Johann Auer
Principal Investigator Email
johann.auer@khbr.at
Contact Person Name
Johann Auer
Contact Person Email
johann.auer@khbr.at

Germany

Earliest CTIS Part Ii Submission Date
11-03-2026
Latest Decision Or Authorization Date
07-05-2026
Processing Time Days
57
Number Of Sites
2
Number Of Participants
15

Sites

Site Name
Universitaetsmedizin Greifswald KöR
Department Name
Klinik und Poliklinik für Innere Medizin B
Principal Investigator Name
Bishwas Chamling
Principal Investigator Email
bishwas.chamling@uni-greifswald.de
Contact Person Name
Bishwas Chamling
Site Name
Universitaetsklinikum Ulm AöR
Department Name
Klinik fuer Innere Medizin II
Principal Investigator Name
Dominik Buckert
Principal Investigator Email
dominik.buckert@uniklinik-ulm.de
Contact Person Name
Dominik Buckert

Italy

Earliest CTIS Part Ii Submission Date
17-03-2026
Latest Decision Or Authorization Date
30-04-2026
Processing Time Days
44
Number Of Sites
10
Number Of Participants
15

Sites

Site Name
Azienda Socio Sanitaria Territoriale Papa Giovanni Xxiii
Department Name
Cardiologia 1
Principal Investigator Name
Emilia D'Elia
Principal Investigator Email
cardiologia.segreteria@asst-pg23.it
Contact Person Name
Emilia D'Elia
Site Name
Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico
Department Name
Cardiologia
Principal Investigator Name
Simone Longhi
Principal Investigator Email
PEIdirezione.generale@pec.aosp.bo.it
Contact Person Name
Simone Longhi
Site Name
Fondazione Toscana Gabriele Monasterio
Department Name
Cardiologia e Medicina Cardiovascolare
Principal Investigator Name
Michele Emdin
Principal Investigator Email
segreteriadirezione@ftgm.it
Contact Person Name
Michele Emdin
Contact Person Email
segreteriadirezione@ftgm.it
Site Name
Azienda Ospedaliero Universitaria Careggi
Department Name
cardio intensive care unit
Principal Investigator Name
Francesco Cappelli
Principal Investigator Email
aoucareggi@pec.it
Contact Person Name
Francesco Cappelli
Contact Person Email
aoucareggi@pec.it
Site Name
Ospedale Vito Fazzi Lecce
Department Name
Cardiologia e UTIC
Principal Investigator Name
Stefania Marazia
Principal Investigator Email
cardiologiaemodinamica@asl.lecce.it
Contact Person Name
Stefania Marazia
Site Name
Azienda Ospedaliera di Padova
Department Name
UOC Cardiologia
Principal Investigator Name
Alberto Cipriani
Principal Investigator Email
cardiologia@aopd.veneto.it
Contact Person Name
Alberto Cipriani
Contact Person Email
cardiologia@aopd.veneto.it
Site Name
Fondazione IRCCS Policlinico San Matteo
Department Name
Centro Amiloidosi Sistemiche e Malattie ad Alta Complessità
Principal Investigator Name
Giovanni Palladini
Principal Investigator Email
segreteria.amiloidosi@smatteo.pv.it
Contact Person Name
Giovanni Palladini
Site Name
Azienda Ospedaliero-Universitaria Senese
Department Name
Cardiologia
Principal Investigator Name
Elisa Giacomin
Principal Investigator Email
ao-siena@postacert.toscana.it
Contact Person Name
Elisa Giacomin
Contact Person Email
ao-siena@postacert.toscana.it
Site Name
Azienda Ospedaliera Dei Colli
Department Name
Malattie Rare e Genetiche Cardiovascolari
Principal Investigator Name
Giuseppe Limongelli
Contact Person Name
Giuseppe Limongelli
Site Name
Centro Cardiologico Monzino S.p.A.
Department Name
Cardiologia Peri-Operatoria e Imaging Cardiovascolare
Principal Investigator Name
Gianluca Pontone
Principal Investigator Email
segreteria.imaging@cardiologicomonzino.it
Contact Person Name
Gianluca Pontone

Sponsor

Primary sponsor

Full Name
Bayer Consumer Care AG
Organisation Type
Pharmaceutical company
Country Of Registered Address
Switzerland

Third parties

  • {"country":"Germany","full_name":"Alcedis GmbH","duties_or_roles":"Home Nursing; 4; 7","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
BEYONTTRA 356 mg film-coated tablets
Active Substance
ACORAMIDIS
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Authorisation Status
Marketing authorisation: EU/1/24/1906/001
Maximum Dose
1424 mg

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