ClinicalTrials.gov record NCT07306949

Real-Life Clinical Efficacy of Acoramidis in Participants With ATTR-CM and Association With Cardiac Biomarkers

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

A Multicenter, Open-Label, Single Arm, Post-Marketing Clinical Study to Evaluate the Therapeutic Effects of Acoramidis Hydrochlorideon Cardiac Biomarkers and Current Clinical Features in Patients With Transthyretin-type Cardiac Amyloidosis: The CARE ATTR Study

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

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 100 years old

SexAll

LocationJapan

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 open-label, single-group phase 4 study gives oral acoramidis to people with transthyretin-type cardiac amyloidosis. Participants receive 800 mg twice daily and are followed with clinical assessments and blood tests through 18 months. The study measures cardiac biomarkers and clinical indexes to evaluate disease progression.

What it is studying. The study is testing whether treatment with acoramidis affects disease progression indexes in transthyretin-type cardiac amyloidosis and how those changes relate to cardiac biomarkers. Main measures include NT-proBNP and outpatient diuretic intensification (ODI); other outcomes include high-sensitivity cardiac troponin T (hs-cTnT), serum transthyretin (TTR), symptom questionnaire scores (KCCQ-12), and kidney function (eGFR).

What the researchers hope to learn. To evaluate whether treatment with acoramidis prevents deterioration of an ATTR-CM disease progression index and to test whether those indexes can be used as surrogate markers of disease progression. The study will also assess associations between treatment and cardiac biomarkers (NT-proBNP, ODI, hs-cTnT), serum TTR, patient-reported heart-failure symptoms, and kidney function.

What this study is about

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

The purpose of this study is to confirm that the treatment with acoramidis prevents the deterioration of the ATTR-CM disease progression index and that these indexes are surrogate markers of disease progression.

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

Treatment history of ATTR-CM is one of the following:
Naive participants: newly diagnosed with ATTR-CM and no prior treatment with drugs for ATTR-CM
Switch participants: Participants who are using tafamidis, a TTR stabilizer, as treatment for ATTR-CM and who, in the judgment of the post-marketing clinical trial investigator (co-principal investigator), can be expected to benefit from switching to acoramidis.
Naive participants must meet the following requirements:
History of hospitalization for heart failure or heart failure symptoms requiring treatment, including diuretics
Echocardiographic end-diastolic ventricular septal thickness greater than 12 millimeters (mm)
Confirmed diagnosis of ATTR-CM (wild type or mutant) by one of the following diagnostic methods
Tissue biopsy shows amyloid deposition and TTR precursor protein is identified by immunohistochemistry or mass spectrometry.
Bone scintigraphy showing strong accumulation ** (Perugini score ≥ 2) consistent with myocardium and no M protein, negating the possibility of AL amyloidosis

Exclusion criteria

Have confirmed diagnosis of AL amyloidosis
Switch participants: prior treatment with gene silencing agents (pachysilane sodium, butrisilane sodium) as treatment for ATTR-CM (including when specifically scheduled to start treatment with a gene silencing agent)
Likelihood of receiving a heart transplant within 1 year from the time screening begins
Hypersensitivity to acoramidis, its metabolites, or additives in the formulation has been confirmed.
Pregnant or lactating women
Has a clinically significant medical condition, an abnormal laboratory test result, or a condition that may jeopardize the safety of the study participant, increase the risk of participation in the post-marketing clinical trial, or affect the study
Participating in an interventional study other than this study, including a clinical trial
In the opinion of the responsible (sub)physician for the post-marketing clinical trial, has a history of drug abuse, alcoholism, or psychiatric disorder that would preclude compliance with this Post-Marketing Clinical Study Protocol

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.

Participants will take oral acoramidis 800 mg twice daily for 18 months. They will have clinic visits with blood tests to measure cardiac biomarkers (NT‑proBNP and high‑sensitivity troponin T), serum transthyretin, kidney function, and will complete quality‑of‑life questionnaires (KCCQ‑12) at specified time points (including baseline, 28 days, 12 months and 18 months). Screening will use or require tests to confirm ATTR‑CM (for example, review of echocardiogram septal thickness, tissue biopsy showing TTR, or bone scintigraphy with Perugini score ≥2 and no M protein). Investigators will record changes in heart‑failure treatment such as outpatient diuretic intensification.

How the study is structured. Multicenter, open-label, single-arm post-marketing (phase 4) study. All participants receive oral acoramidis 800 mg twice daily; investigators collect blood tests, symptom questionnaires, and clinical data at baseline and during follow-up (notably at 12 and 18 months).

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 Japan. 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

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: Oct 1, 2025
Primary completion: Mar 31, 2028 (estimated)
Study completion: Mar 31, 2028 (estimated)
Target enrollment: 200 participants (estimated)
Last updated on registry: Jan 7, 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: Alexion Pharmaceuticals, Inc.

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.

Alexion Pharmaceuticals, Inc. (Sponsor) 📞 1-855-752-2356 ✉ clinicaltrials@alexion.com

Participating sites (16)

Research SiteNot Yet Recruiting
Bunkyō City, 113-8431 · Japan
Research SiteNot Yet Recruiting
Bunkyō City, 113-8603 · Japan
Research SiteNot Yet Recruiting
Kumamoto, 860-8556 · Japan
Research SiteRecruiting
Kurume-shi, 830-0011 · Japan
Research SiteNot Yet Recruiting
Kyoto, 606-8507 · Japan
Research SiteNot Yet Recruiting
Mitaka-shi, 181-8611 · Japan
Research SiteNot Yet Recruiting
Nagoya, 466-8560 · Japan
Research SiteNot Yet Recruiting
Nankoku-shi, 783-8505 · Japan
Research SiteNot Yet Recruiting
Okayama, 700-8558 · Japan
Research SiteNot Yet Recruiting
Ōtsu, 520-2192 · Japan

Terms used on this page · explanations by MedicalUpdates.ai

prior treatment: Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
principal investigator: The doctor or scientist responsible for running the study at a site or overall.
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.
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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