Switching from tafamidis to acoramidis to see how transthyretin blood levels change

A Prospective, Single-arm, Phase 4 Study to Evaluate the Course of Serum Transthyretin (TTR) Level With Acoramidis in Adult Patients With Variant or Wild-type Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM) Previously Treated With Tafamidis

This test will see if switching people with transthyretin amyloid cardiomyopathy who are taking tafamidis to acoramidis raises transthyretin levels in the blood.

Quick facts

• Phase: Phase 4
• Study type: Interventional
• Enrollment: 50 (estimated)
• Ages: 18 Years to 90 Years
• Sex: All
• Sponsor: Bayer (Industry-sponsored)
• Locations: 10 sites (Sankt Pölten, Lower Austria and 9 other locations)
• Trial ID: NCT07298044 on ClinicalTrials.gov

What this trial studies

This Phase 4 interventional study enrolls adults with confirmed ATTR-CM who have been taking tafamidis for at least three months and are NYHA class I–II. Participants will stop tafamidis and start acoramidis, with scheduled blood draws to measure transthyretin (TTR) levels and routine cardiac and laboratory assessments including NT-proBNP and kidney function. The study aims to document the change in circulating TTR after the switch and to characterize safety and tolerability in this population. Study sites are located in Austria and the sponsor is Bayer.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18–90 with confirmed ATTR-CM who have used tafamidis for at least three months, have NYHA class I–II, eGFR ≥30 mL/min/1.73 m², and NT-proBNP between 300 and 7000 pg/mL.

Not a fit: Patients unlikely to benefit include those with advanced heart failure (NYHA III–IV), prior or planned heart/liver transplant, planned ventricular assist device, active cancer or other illness limiting life expectancy to <1 year, ischemic heart failure, or inadequate kidney function.

Why it matters

Potential benefit: If successful, switching to acoramidis may raise blood transthyretin levels and potentially slow disease progression or improve heart function for people with ATTR-CM.

How similar studies have performed: Both tafamidis and acoramidis are approved TTR stabilizers with demonstrated benefits in ATTR-CM, but direct switch studies specifically measuring changes in blood TTR are limited.

Eligibility criteria

Inclusion Criteria:

* Participants must be 18 to 90 years of age inclusive, at the time of signing the informed consent.
* Diagnosis of ATTR-CM; disease defining examination, i.e., Single Photon Emission Computed Tomography (SPECT) or SPECT/Computed Tomography (CT) or biopsy, within 24 months prior to Visit 1 (V1).
* 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.
* New York Heart Association (NYHA) class ≤ II at V1.
* Estimate glomerular filtration rate (eGFR) ≥ 30 mL/min/1.73m\^2 at V1.
* N-terminal pro-B-type Natriuretic Peptide (NT-proBNP) \> 300 and ≤ 7000 pg/mL at V1.

Exclusion Criteria:

* Prior liver or heart transplantation or planned within the next 12 months.
* Current or planned use of ventricular assist device.
* Active cancer or other disease that decreases the life expectancy to less than one year.
* Heart failure due to ischemic heart disease.
* Myocardial infarction, cardiovascular (CV) surgery, or unstable angina within the last 90 days prior to V1.
* Confirmed diagnosis of light-chain amyloidosis.
* Dialysis or severe renal impairment as reflected by Urinary Albumin Creatinine Ratio (UACR) \> 300 mg/g at V1.
* Major surgery 90 days prior to V1.
* Recent initiation of Sodium-Glucose-Cotransporter-2 inhibitors (SGLT2i) within 3 months before V1.
* Initiation of treatment with a diuretic or increase in diuretic dose within 3 months before V1.
* Treatment with calcium channel blockers (e.g., verapamil, diltiazem) or digitalis.
* Recent CV hospitalization within 3 months before V1.
* Known hypersensitivity to acoramidis or to any of the excipients.
* 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.
* Known or suspected liver disorder and bile secretion/flow (cholestasis, also history of it).
* Abnormal liver function tests at V1, defined as ALT (GPT) or AST (GOT) ≥ 3 x ULN or total bilirubin ≥ 3 x ULN at V1.

Where this trial is running

Sankt Pölten, Lower Austria and 9 other locations

• Universitätsklinikum St. Pölten - Lilienfeld - Klinische Abteilung für Innere Medizin 3 — Sankt Pölten, Lower Austria, Austria (Withdrawn)
• Medizinische Universität Wien- Universitätsklinik für Innere Medizin II, Klinische Abteilung für Kardiologie — Vienna, State of Vienna, Austria (Not_yet_recruiting)
• Klinik Favoriten - 5.Medizinische Abteilung-Kardiologie — Vienna, State of Vienna, Austria (Not_yet_recruiting)
• Klinik Ottakring - 3. Medizinische Abteilung mit Kardiologie, internistischer Intensivmedizin und Ambulanz — Vienna, State of Vienna, Austria (Not_yet_recruiting)
• Medizinische Universität Graz- Klinische Abteilung für Kardiologie — Vienna, Styria, Austria (Recruiting)
• Krankenhaus St. Josef Braunau | Innere Medizin I — Braunau am Inn, Upper Austria, Austria (Not_yet_recruiting)
• AZ St-Jan Brugge-Oostende A.V. — Bruges, Belgium (Not_yet_recruiting)
• Hôpital Erasme/Erasmus Ziekenhuis — Brussels, Belgium (Not_yet_recruiting)
• KVZ Kardiovaskulaeres Zentrum Darmstadt GmbH — Darmstadt, Hesse, Germany (Not_yet_recruiting)
• Oslo Universitetssykehus HF, Rikshospitalet — Oslo, Norway (Not_yet_recruiting)

Study contacts

• Study coordinator: Bayer Clinical Trials Contact

Conditions Transthyretin Amyloid Cardiomyopathy; ATTR-CM