ClinicalTrials.gov record NCT07298044

A Study to Learn More About the Change in the Blood Levels of Transthyretin When Participants With Transthyretin Amyloidosis With Cardiomyopathy Switch From Tafamidis to Acoramidis

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 28, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 4A study of a treatment that is already approved, to learn more about its long-term effects, safety or use in everyday practice.
Age18 years old to 90 years old
SexAll
LocationAustria, Belgium, Germany, Italy, Norway
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 4, open-label study is for adults with transthyretin amyloid cardiomyopathy (ATTR-CM) who were previously treated with tafamidis. Participants continue tafamidis during screening, then take acoramidis tablets twice daily for up to 6 months. Researchers measure blood transthyretin at baseline and several follow-up times, and collect safety and heart- and kidney-related tests; the study includes two site visits, six home nurse visits, and a final phone check-in.

What it is studying. The study is testing how serum transthyretin (TTR) levels change after people with variant or wild-type ATTR-CM who were on tafamidis switch to acoramidis. It also records safety (treatment-emergent and serious adverse events) and measures other heart- and kidney-related biomarkers, signs of cardiac remodeling by echocardiogram, exercise capacity (6-minute walk), and quality-of-life questionnaires.

What the researchers hope to learn. Primary scientific goal: measure change in serum TTR from baseline to month 6 or premature discontinuation. Secondary goals: measure earlier changes in TTR (weeks 1–4, month 3), record number of treatment-emergent and serious adverse events, and assess changes from baseline to month 6 in NT-proBNP, hs-TnT, RBP4, echocardiographic signs of remodeling, 6-minute walk distance, renal markers (eGFR, creatinine, cystatin C, UACR), TSH, and quality-of-life scores (KCCQ-OS, EQ-5D-5L).

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

Transthyretin (TTR) is a protein made by the liver that helps transport thyroid hormone and vitamin A in the blood. In some people, this protein breaks down and forms harmful clumps called amyloid. TTR amyloid gets deposited in the heart wall and stops it from pumping blood properly, which may lead to heart failure. The breakage in TTR protein can be age-related (wild-type ATTR-CM), or genetic (variant ATTR-CM). The study drug, acoramidis, works by attaching itself to the TTR protein, making TTR more stable and less likely to break down and form amyloid (clumps). This helps to slow down the progression of the disease, improve heart function, and increase the TTR levels in the blood. Acoramidis is an approved treatment for wild-type or variant ATTR-CM in Europe and the United States. Tafamidis is another drug that stabilizes TTR and prevents it from breaking down. In this study, acoramidis will be studied in participants with ATTR--CM who were previously treated with tafamidis. The main purpose of this study is to assess the change in blood TTR levels after participants are switched from tafamidis to acoramidis. This will be studied to understand if acoramidis causes an increase in blood TTR levels beyond the levels achieved with tafamidis. For this, the researchers will measure the change in the levels of TTR protein in participants' blood after 6 months of the treatment with acoramidis, or earlier if a participant stops the treatment before reaching that six-month mark. All participants will continue taking tafamidis during the screening period. In the treatment period of the study, participants will take acoramidis as two tablets twice daily by mouth, for up to 6 months. At the start of this study, the study doctors will review each participant's medical history and check their overall health. The study doctors will perform electrocardiograms (ECG), and measure blood pressure and heart rate. Researchers will also take blood and urine samples from the participants to measure levels of TTr, NT-proBNP, hs-TnT, hs-CRP, RBP4, eGFR, creatinine, cystatin-C, UACR, and TSH at the start of the study, and at various time points thereafter (during the study) to assess heart, kidney and thyroid function. There will be a total of 9 study check-ins. Participants will visit the study site twice: at screening and at the end of treatment period. A study nurse will visit the participant's home 6 times, at the start of treatment, Weeks 1, 2, 3 and 4, then again at 3 months. The final check-in will be done by phone. The study doctors will monitor the health of the participants regularly for any medical problems during follow-up visits. Participants will know the treatment they will receive during the study. Each participant could be in the study for about 8 months.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Acoramidis

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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 within 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.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

You will stop tafamidis after screening and take acoramidis (two tablets twice daily) for up to 6 months; tafamidis is continued during the screening period. There are a total of nine study check-ins: two site visits (screening and end of treatment), six home visits by a study nurse (start of treatment, Weeks 1–4, and at 3 months), and a final phone check-in; total time in the study is about 8 months. At visits the study team will review your health, do ECGs and vital-sign checks, take blood and urine samples to measure transthyretin and other heart, kidney, and thyroid markers, perform an echocardiogram (to assess heart remodeling), a 6-minute walk test, and ask you to complete health questionnaires; transthyretin (TTR) will be measured at baseline, weekly during the first month, at month 3, and at month 6 or earlier if you stop treatment. The registry requires that you already have a disease-defining exam for ATTR-CM (SPECT or SPECT/CT or biopsy) within 24 months before screening; the record does not state that the study will perform that exam.

How the study is structured. Open-label, single-group Phase 4 trial. All participants receive acoramidis 712 mg (two 356 mg tablets) by mouth twice daily for up to 6 months. Blood and urine samples, ECGs, echocardiogram, 6-minute walk test, and quality-of-life questionnaires are done at baseline and at specified follow-up times (weeks 1–4, month 3, and month 6). The study has two site visits (screening and end of treatment), six home nurse visits (start of treatment, weeks 1–4, and month 3), and a final phone check-in; safety events are recorded from first dose until 30 days after last dose or end of study.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The listed sites are in Austria, Belgium, Germany, Italy, Norway. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on wild-type ATTR amyloidosis

This trial is also listed on this site under ATTRv amyloidosis.

Clinical trials121 trials listed for wild-type ATTR amyloidosisEvery trial the registry lists for wild-type ATTR amyloidosis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in wild-type ATTR amyloidosis researchLatest monthly update: September 2026. Includes: Frailty and support needs. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Feb 13, 2026
Primary completion
Jul 15, 2027 (estimated)
Study completion
Jul 15, 2027 (estimated)
Target enrollment
50 participants (estimated)
Last updated on registry
Sep 25, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Bayer

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (26)

  • Universitätsklinikum St. Pölten - Lilienfeld - Klinische Abteilung für Innere Medizin 3Withdrawn

    Sankt Pölten, Lower Austria, 1210 · Austria

  • Medizinische Universität Wien- Universitätsklinik für Innere Medizin II, Klinische Abteilung für KardiologieRecruiting

    Vienna, State of Vienna, 1090 · Austria

  • Klinik Favoriten - 5.Medizinische Abteilung-KardiologieRecruiting

    Vienna, State of Vienna, 1100 · Austria

  • Klinik Ottakring - 3. Medizinische Abteilung mit Kardiologie, internistischer Intensivmedizin und AmbulanzNot Yet Recruiting

    Vienna, State of Vienna, 3500 · Austria

  • Medizinische Universität Graz- Klinische Abteilung für KardiologieRecruiting

    Vienna, Styria, 1030 · Austria

  • Krankenhaus St. Josef Braunau | Innere Medizin IRecruiting

    Braunau am Inn, Upper Austria, 5280 · Austria

  • VIVIT - Landeskrankenhaus Feldkirch - Innere Medizin I, Kardiologie, Angiologie, Endokrinologie, Diabetologie, IntensivmedizinWithdrawn

    Feldkirch, Vorarlberg, 6800 · Austria

  • Hôpital Erasme/Erasmus ZiekenhuisNot Yet Recruiting

    Brussels, Brussels Capital, 1070 · Belgium

  • AZ St-Jan Brugge-Oostende A.V.Not Yet Recruiting

    Bruges, West Flanders, 8000 · Belgium

  • Universitaetsklinikum Ulm | Klinik fuer Innere Medizin IIWithdrawn

    Ulm, Baden-Wurttemberg, 89081 · Germany

Terms used on this page · explanations by MedicalUpdates.ai

study drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
screening period
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
ECG
A recording of the heart's electrical activity. QTc is one measurement from it; some drugs are avoided if it is prolonged.
eGFR
A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
Computed Tomography
An X-ray scan that builds detailed cross-section images of the body. 'High-resolution' CT (HRCT) is used to look closely at the lungs.
biopsy
Removal of a small piece of tissue so it can be examined under a microscope. 'Biopsy-proven' or 'histologically confirmed' means the diagnosis was confirmed by examining tissue under a microscope.
life expectancy
Some studies require that, in the investigator's judgement, a participant is likely to live long enough to complete the study period. It is a study requirement, not a prediction about any individual.
x ULN
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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